ANZCTR - Registration

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02332668

Registration number

NCT02332668

Ethics application status

Date submitted

6/01/2015

Date registered

7/01/2015

Titles & IDs

Public title

A Study of Pembrolizumab (MK-3475) in Pediatric Participants With an Advanced Solid Tumor or Lymphoma (MK-3475-051/KEYNOTE-051)

Scientific title

A Phase I/II Study of Pembrolizumab (MK-3475) in Children With Advanced Melanoma or a PD-L1 Positive Advanced, Relapsed or Refractory Solid Tumor or Lymphoma (KEYNOTE-051)

Secondary ID [1]

MK-3475-051

Secondary ID [2]

3475-051

Universal Trial Number (UTN)

Trial acronym

Linked study record

Health condition

Health condition(s) or problem(s) studied:

Diabetes Mellitus

Melanoma

Lymphoma

Solid Tumor

Classical Hodgkin Lymphoma

Microsatellite-instability-high Solid Tumor

Mitral Valve Insufficiency

Heart Failure

Secondary Progressive Multiple Sclerosis

Condition category

Condition code

Cancer

Lymphoma (non Hodgkin's lymphoma) - High grade lymphoma

Cancer

Lymphoma (non Hodgkin's lymphoma) - Low grade lymphoma

Cancer

Malignant melanoma

Human Genetics and Inherited Disorders

Other human genetics and inherited disorders

Oral and Gastrointestinal

Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon

Neurological

Other neurological disorders

Metabolic and Endocrine

Diabetes

Cardiovascular

Coronary heart disease

Cardiovascular

Other cardiovascular diseases

Cardiovascular

Diseases of the vasculature and circulation including the lymphatic system

Skin

Dermatological conditions

Skin

Other skin conditions

Infection

Studies of infection and infectious agents

Intervention/exposure

Study type

Interventional

Description of intervention(s) / exposure

Treatment: Other - Pembrolizumab
Treatment: Drugs - Sitagliptin
Treatment: Drugs - Placebo
Treatment: Devices - Carillon Mitral Contour System
Treatment: Drugs - ZGN-440 for Injectable Suspension
Treatment: Drugs - ZGN-440 Placebo for Injectable Suspension
Treatment: Surgery - Blood Draw
Treatment: Surgery - CSF collection by lumbar puncture (Optional)
Treatment: Other - recombinant influenza hemagglutinin
Treatment: Other - Advax1
Treatment: Other - Advax2
Other interventions - Home
Other interventions - Ward
Treatment: Drugs - ceftriaxone
Treatment: Drugs - flucloxacillin

Experimental: Melanoma - Participants aged 6 months to \<18 years with melanoma receive pembrolizumab, starting dose 2 mg/kg (maximum dose 200 mg), intravenously (IV) once every 3 weeks (Q3W). Enrollment of participants aged 6 months to \<12 years with melanoma was closed with Amendment 8. Enrollment of participants aged =12 years to =18 years with melanoma continues.

Experimental: Solid Tumors and Other Lymphomas - Participants aged 6 months to \<18 years with solid tumors and other lymphomas receive pembrolizumab, starting dose 2 mg/kg (maximum dose 200 mg), IV Q3W. Initial enrollment limited to programmed death-ligand 1 (PD-L1)-positive participants. PD-L1-negative participants may enroll if responses are observed. Enrollment of participants with solid tumors and other lymphomas was closed with Amendment 8.

Experimental: rrcHL - Participants aged 3 years to \<18 years with rrcHL receive pembrolizumab, starting dose 2 mg/kg (maximum dose 200 mg), IV Q3W.

Experimental: MSI-H - Participants aged 6 months to \<18 years with microsatellite-instability-high (MSI-H) solid tumors receive pembrolizumab, starting dose 2 mg/kg (maximum dose 200 mg), IV Q3W.

Experimental: TMB-H - Participants aged 6 months to \<18 years with tumor-mutational burden-high =10 mutation/Mb (TMB-H) solid tumors receive pembrolizumab, starting dose 2 mg/kg (maximum dose 200 mg), IV Q3W.

Experimental: Adjuvant Melanoma - Participants aged 12 years to \<18 years with resected high-risk Stage IIB, IIC, III, or IV melanoma receive pembrolizumab, starting dose 2 mg/kg (maximum dose 200 mg), intravenously (IV) once every 3 weeks (Q3W).

Experimental: Sitaglipltin (100mg) - Active drug (sitagliptin)

Placebo comparator: Placebo (sugar pill) - Inactive drug (placebo)

Experimental: Treatment Group - Implantation of the Carillon Mitral Contour System

No intervention: Control Group - Optimized stable medical therapy

Subjects Assigned to BAF312 - Patients with secondary progressive multiple sclerosis (SPMS) randomized to receive BAF312 (siponimod). Refer to ClinicalTrials.gov record NCT01665144 for more information.

Subjects Assigned to Placebo (Controls) - Patients with secondary progressive multiple sclerosis (SPMS) randomized to receive placebo. Refer to ClinicalTrials.gov record NCT01665144 for more information.

Experimental: gevokizumab - Solution for subcutaneous injection (Part 1, Group B)

Placebo comparator: Placebo - Solution for subcutaneous injection (Part 1, Group A)

Experimental: gevokizumab open-label - Solution for subcutaneous injection (Part 2, Open-label)

Experimental: HA 15ug+Advax2 - recombinant influenza hemagglutinin (H5) 15ug, Advax2 adjuvant 20mg, i.m. injection, 2 doses

Experimental: HA 5ug+Advax1 - recombinant influenza hemagglutinin (H5) 5ug, Advax1 adjuvant 20mg, i.m. injection, 2 doses

Experimental: HA 5ug+Advax2 - recombinant influenza hemagglutinin (H5) 5ug, Advax2 adjuvant 20mg, i.m. injection, 2 doses

Experimental: HA 2.5ug+Advax2 - recombinant influenza hemagglutinin (H5) 2.5ug, Advax2 adjuvant 20mg, i.m. injection, 2 doses

Experimental: HA 15ug - recombinant influenza hemagglutinin (H5) 15ug, i.m. injection, 2 doses

Treatment: Other: Pembrolizumab
IV infusion

Treatment: Drugs: Sitagliptin
100mg mane for 2 days

Treatment: Drugs: Placebo
Inactive drug (Placebo)

Treatment: Devices: Carillon Mitral Contour System
Percutaneous mitral valve repair

Treatment: Drugs: ZGN-440 for Injectable Suspension
Subjects will receive ZGN-440 twice weekly subcutaneous injections for up to 52 weeks.

Treatment: Drugs: ZGN-440 Placebo for Injectable Suspension
Subjects will receive placebo twice weekly subcutaneous injections for up to 52 weeks.

Treatment: Surgery: Blood Draw
Blood draws (65 mLs \[\~4 tablespoons\] per blood draw) at 4 time points: Prior to study medication initiation, and at +6, +12 and+24 months post treatment initiation.

Treatment: Surgery: CSF collection by lumbar puncture (Optional)
For participants who volunteer to donate CSF samples: up to 25 mLs (\<2 tablespoons): prior to study medication initiation, and at month 24 post treatment initiation.

Treatment: Other: recombinant influenza hemagglutinin
recombinant influenza hemagglutinin

Treatment: Other: Advax1
Delta inulin adjuvant formulation 1

Treatment: Other: Advax2
Delta inulin adjuvant formulation 2

Other interventions: Home
The main intervention is for children with uncomplicated cellulitis to remain at home throughout the period of intravenous treatment but as it is not feasible to administer flucloxacillin four times a day by the Hospital-In-The-Home team, once daily ceftriaxone is the most ideal antibiotic to be given to this group

Other interventions: Ward
Admission to a hospital based ward

Treatment: Drugs: ceftriaxone
50mg/kg once daily

Treatment: Drugs: flucloxacillin
50mg/kg 6 hourly

Intervention code [1]

Treatment: Other

Intervention code [2]

Treatment: Drugs

Intervention code [3]

Treatment: Devices

Intervention code [4]

Treatment: Surgery

Intervention code [5]

Other interventions

Comparator / control treatment

Control group

Outcomes

Primary outcome [1]

Objective Response Rate (ORR) by Response Evaluation Criteria in Solid Tumors and Other Lymphoma Version 1.1 (RECIST 1.1) per Site Assessment (Each Disease Indication Evaluated Separately)

Timepoint [1]

Up to 2 years

Primary outcome [2]

ORR by RECIST 1.1 per Site Assessment for MSI-H or TMBH Solid Tumors (Each Cohort Evaluated Separately)

Timepoint [2]

Up to 2 years

Primary outcome [3]

ORR by International Working Group (IWG) Response Criteria (Cheson, 2007) per BICR Assessment for rrcHL Cohort

Timepoint [3]

Up to approximately 2 years

Primary outcome [4]

Number of Participants with Dose-Limiting Toxicities (DLTs)

Timepoint [4]

Cycle 1 (Up to 21 days)

Primary outcome [5]

Number of Participants Experiencing Adverse Events (AEs)

Timepoint [5]

Up to 27 months

Primary outcome [6]

Number of Participants Discontinuing Study Drug Due to AEs

Timepoint [6]

Up to 2 years

Primary outcome [7]

Gastric emptying

Timepoint [7]

3 hours per gastric emptying study (i.e. 6 hours)

Primary outcome [8]

Change in Baseline Regurgitant Volume Associated With the Carillon Device Relative to the Control Population at 12 Months

Timepoint [8]

Baseline and 12 months

Primary outcome [9]

Difference in the Rate of Major Adverse Events Between Treatment (Carillon) and Control Groups

Timepoint [9]

Baseline and 12 months

Primary outcome [10]

Change in body weight

Timepoint [10]

Baseline to Week 26

Primary outcome [11]

Safety and tolerability assessed by adverse events, laboratory evaluations, ECGs, vital signs, physical examinations

Timepoint [11]

Baseline to Week 26 and Week 52

Primary outcome [12]

Change in frequency of MBP-reactive Th17 cells

Timepoint [12]

From baseline to the follow-up time points (Open label phase + 6 months and OLP +12 months).

Primary outcome [13]

Adverse Events

Timepoint [13]

30 days

Primary outcome [14]

The proportion of subjects at Day 126 with complete closure of the PG target ulcer confirmed 2 weeks later (at Day 140) and without the need for rescue treatment

Timepoint [14]

Day 126

Primary outcome [15]

The incidence of adverse events

Timepoint [15]

12 months

Primary outcome [16]

Treatment failure (inadequate clinical improvement, adverse event)

Timepoint [16]

Within 2 days of commencing empiric antibiotic

Secondary outcome [1]

ORR by IWG Response Criteria (Cheson, 2007) per Site Assessment (rrcHL Cohort)

Timepoint [1]

Up to 2 years

Secondary outcome [2]

DOR per RECIST 1.1 by Site Assessment (Advanced Melanoma, Solid Tumors and Other Lymphoma, Each Disease Indication Is Evaluated Separately)

Timepoint [2]

Up to approximately 2 years

Secondary outcome [3]

DOR per RECIST 1.1 by Site Assessment (MSI-H and TMBH, Each Cohort Is Evaluated Separately)

Timepoint [3]

Up to approximately 2 years

Secondary outcome [4]

DOR per IWG 2007 (Cheson, 2007) Response by BICR Assessment (rrcHL Cohort)

Timepoint [4]

Up to approximately 2 years

Secondary outcome [5]

DOR per IWG 2007 (Cheson, 2007) Response by Site Assessment (rrcHL Cohort)

Timepoint [5]

Up to approximately 2 years

Secondary outcome [6]

DOR per Immune-related Response Evaluation Criteria in Solid Tumors (irRECIST) by Site Assessment (Solid Tumors and Other Lymphoma, Each Disease Indication Evaluated Separately)

Timepoint [6]

Up to approximately 2 years

Secondary outcome [7]

DOR per Immune-related Response Evaluation Criteria in Solid Tumors (irRECIST) by Site Assessment (MSI-H and TMB-H, Each Cohort Is Evaluated Separately)

Timepoint [7]

Up to approximately 2 years

Secondary outcome [8]

Progression-free Survival (PFS) Using RECIST 1.1 Criteria by Site Assessment (Solid Tumors and Other Lymphoma, Each Disease Indication Evaluated Separately)

Timepoint [8]

Up to approximately 2 years

Secondary outcome [9]

PFS using RECIST 1.1 Criteria by Site Assessment (MSI-H and TMB-H, Each Cohort Evaluated Separately)

Timepoint [9]

Up to approximately 2 years

Secondary outcome [10]

PFS using IWG 2007 Criteria (Chesson 2007) by BICR Assessment (rrcHL Cohort)

Timepoint [10]

Up to approximately 2 years

Secondary outcome [11]

PFS using IWG 2007 Criteria (Chesson, 2007) by Site Assessment (rrcHL Cohort)

Timepoint [11]

Up to approximately 2 years

Secondary outcome [12]

PFS Using irRECIST Criteria by Site Assessment

Timepoint [12]

Up to approximately 2 years

Secondary outcome [13]

Disease Control Rate by RECIST 1.1 Using Site Assessment (Solid Tumors and Other Lymphoma, Each Disease Indication Evaluated Separately)

Timepoint [13]

Up to approximately 2 years

Secondary outcome [14]

Disease Control Rate by RECIST 1.1 Using Site Assessment (MSIH and TMB-H, Each Cohort Evaluated Separately)

Timepoint [14]

Up to approximately 2 years

Secondary outcome [15]

Disease Control Rate by irRECIST Using Site Assessment (Solid Tumors and Other Lymphomas, Each Disease Indication Evaluated Separately)

Timepoint [15]

Up to approximately 2 years

Secondary outcome [16]

Overall Survival

Timepoint [16]

Up to approximately 2 years

Secondary outcome [17]

Objective irRECIST Response Rate by Site Assessment (Each Disease Indication Evaluated Separately)

Timepoint [17]

Up to approximately 2 years

Secondary outcome [18]

Area Under the Concentration Curve (AUC) for Pembrolizumab

Timepoint [18]

Predose Cycles 1, 2, 4, 8 and every 4 cycles thereafter, and within 30 minutes post infusion at Cycles 1 and 8. Additional single pharmacokinetic samples obtained in Cycle 1 between 72 to 168 hours post-dose, and Cycle 1 at 336 hours post-dose

Secondary outcome [19]

Glycaemia

Timepoint [19]

4 hours during each gastric empty study (i.e. 8 hours)

Secondary outcome [20]

Gastrointestinal hormone release

Timepoint [20]

4 hours during each gastric empty study (i.e. 8 hours)

Secondary outcome [21]

Intragastric meal distribution

Timepoint [21]

3 hours during each gastric empty study (i.e. 6 hours)

Secondary outcome [22]

Blood pressure

Timepoint [22]

4.5 hours during each gastric empty study (i.e. 9 hours)

Secondary outcome [23]

Heart rate

Timepoint [23]

4.5 hours during each gastric empty study (i.e. 9 hours)

Secondary outcome [24]

Splanchnic blood flow

Timepoint [24]

4 hours during each gastric empty study (i.e. 8 hours)

Secondary outcome [25]

Cardiac output

Timepoint [25]

4 hours during each gastric empty study (i.e. 8 hours)

Secondary outcome [26]

Stroke volume

Timepoint [26]

4 hours during each gastric empty study (i.e. 8 hours)

Secondary outcome [27]

Appetite

Timepoint [27]

4 hours during each gastric empty study (i.e. 8 hours)

Secondary outcome [28]

Rate of Heart Failure Hospitalizations Between Treatment (Carillon) and Control Groups

Timepoint [28]

Baseline and 12 months

Secondary outcome [29]

Change in Six-minute Walk Distance Between Treatment (Carillon) and Control Groups

Timepoint [29]

Baseline and 12 Months

Secondary outcome [30]

Change in Left Ventricular Volumes Between Treatment (Carillon) and Control Groups

Timepoint [30]

Baseline and 12 Months

Secondary outcome [31]

Peak aortic valve pressure gradient as measured by echocardiography and assessed by an independent core laboratory

Timepoint [31]

At discharge from hospital or at 7 days post-procedure (whichever comes first), 30 days, 6 months, and 12 months post procedure

Secondary outcome [32]

Peak aortic velocity as measured by echocardiography and assessed by an independent core laboratory

Timepoint [32]

At discharge from hospital or at 7 days post-procedure (whichever comes first), 30 days, 6 months, and 12 months post procedure

Secondary outcome [33]

Mortality: all-cause, cardiovascular, and non-cardiovascular

Timepoint [33]

At discharge from hospital or 7 days post-procedure (whichever comes first), 30 days, 6 months, and 12 months post procedure

Secondary outcome [34]

Stroke: disabling and non-disabling

Timepoint [34]

At discharge from hospital or 7 days post-procedure (whichever comes first), 30 days, 6 months, and 12 months post procedure

Secondary outcome [35]

Myocardial infarction (MI): periprocedural (=72 hours post index procedure) and spontaneous (>72 hours post index procedure)

Timepoint [35]

At discharge from hospital or 7 days post-procedure (whichever comes first), 30 days, 6 months, and 12 months post procedure

Secondary outcome [36]

Bleeding: life-threatening (or disabling) and major

Timepoint [36]

At discharge from hospital or 7 days post-procedure (whichever comes first), 30 days, 6 months, and 12 months post procedure

Secondary outcome [37]

Acute kidney injury based on the Acute Kidney Injury Network (AKIN) System Stage 3 (including renal replacement therapy) or Stage 2

Timepoint [37]

=7 days post index procedure

Secondary outcome [38]

Major vascular complications major

Timepoint [38]

At discharge from hospital or 7 days post-procedure (whichever comes first), 30 days, 6 months, and 12 months post procedure

Secondary outcome [39]

Repeat procedure for valve-related dysfunction (surgical or interventional therapy)

Timepoint [39]

At discharge from hospital or 7 days post-procedure (whichever comes first), 30 days, 6 months, and 12 months post procedure

Secondary outcome [40]

Hospitalization for valve-related symptoms or worsening congestive heart failure (NYHA class III or IV)

Timepoint [40]

At discharge from hospital or 7 days post-procedure (whichever comes first), 30 days, 6 months, and 12 months post procedure

Secondary outcome [41]

New permanent pacemaker implantation resulting from new or worsened conduction disturbances

Timepoint [41]

At discharge from hospital or 7 days post-procedure (whichever comes first), 30 days, 6 months, and 12 months post procedure

Secondary outcome [42]

New onset of atrial fibrillation or atrial flutter

Timepoint [42]

At discharge from hospital or 7 days post-procedure (whichever comes first), 30 days, 6 months, and 12 months post procedure

Secondary outcome [43]

Coronary obstruction

Timepoint [43]

=72 hours post index procedure

Secondary outcome [44]

Ventricular septal perforation

Timepoint [44]

=72 hours post index procedure

Secondary outcome [45]

Mitral apparatus damage

Timepoint [45]

=72 hours post index procedure

Secondary outcome [46]

Cardiac tamponade

Timepoint [46]

=72 hours post index procedure

Secondary outcome [47]

Prosthetic aortic valve malpositioning, including valve migration, valve embolization, or ectopic valve deployment

Timepoint [47]

At discharge from hospital or 7 days post-procedure (whichever comes first), 30 days, 6 months, and 12 months post procedure

Secondary outcome [48]

Transcatheter aortic valve (TAV)-in-TAV deployment

Timepoint [48]

At discharge from hospital or 7 days post-procedure (whichever comes first), 30 days, 6 months, and 12 months post procedure

Secondary outcome [49]

Prosthetic aortic valve thrombosis

Timepoint [49]

At discharge from hospital or 7 days post-procedure (whichever comes first), 30 days, 6 months, and 12 months post procedure

Secondary outcome [50]

Prosthetic aortic valve endocarditis

Timepoint [50]

At discharge from hospital or 7 days post-procedure (whichever comes first), 30 days, 6 months, and 12 months post procedure

Secondary outcome [51]

Neurological status per modified Rankin Scale score

Timepoint [51]

At discharge from hospital or 7 days post-procedure (whichever comes first), 30 days, 6 months, and 12 months post procedure

Secondary outcome [52]

Neurological status per National Institutes of Health Stroke Scale

Timepoint [52]

At discharge from hospital or 7 days post-procedure (whichever comes first), 30 days, 6 months, and 12 months post procedure

Secondary outcome [53]

Functional Improvement from baseline per NYHA functional classification

Timepoint [53]

At discharge from hospital or 7 days post-procedure (whichever comes first), 30 days, 6 months, and 12 months post procedure

Secondary outcome [54]

Change in body weight

Timepoint [54]

Baseline to Week 52

Secondary outcome [55]

Change in fasting glycemic parameters

Timepoint [55]

Baseline to Week 26 and Week 52

Secondary outcome [56]

Change in cardiometabolic parameters

Timepoint [56]

Baseline to Week 26 and Week 52

Secondary outcome [57]

Change in Patient Reported Outcomes (PRO) scores

Timepoint [57]

Baseline to Week 26 and Week 52

Secondary outcome [58]

Change in frequency of polyclonal CD4+ Th17, Th1, Th2, and Treg cells

Timepoint [58]

From baseline to the follow-up time points (Open label phase + 6 months and OLP +12 months).

Secondary outcome [59]

Change in chemokine and cytokines levels

Timepoint [59]

From baseline to the follow-up time points (Open label phase + 6 months and OLP +12 months).

Secondary outcome [60]

Change in Regulatory B Cells

Timepoint [60]

From baseline to the follow-up time points (Open label phase + 6 months and OLP +12 months).

Secondary outcome [61]

Changes of clinical status and lymphocyte subgroups

Timepoint [61]

From baseline to the follow-up time points (Open label phase + 6 months and OLP +12 months).

Secondary outcome [62]

Procedural Success (Number of patients with successful TMVR placement)

Timepoint [62]

Through 5 years

Secondary outcome [63]

Reduction of MR (Number of patients with a reduction of MR Grade)

Timepoint [63]

Through 5 years

Secondary outcome [64]

The proportions of subjects at Day 126 with a reduction in the target ulcer area of = 75% or = 90% from baseline.

Timepoint [64]

Day 126

Secondary outcome [65]

Hemagglutination inhibition assay

Timepoint [65]

1 month post each immunization and 11 months post final immunization

Secondary outcome [66]

Time to no progression

Timepoint [66]

Within 3 days

Secondary outcome [67]

Time to discharge

Timepoint [67]

14 days

Secondary outcome [68]

Readmission rate

Timepoint [68]

28 days

Secondary outcome [69]

Representation to ED

Timepoint [69]

28 days

Secondary outcome [70]

ED Length of stay

Timepoint [70]

2 days

Secondary outcome [71]

Duration of iv antibiotics

Timepoint [71]

14 days

Secondary outcome [72]

IV cannula resiting (Rates of iv cannula needing at least one resiting)

Timepoint [72]

14 days

Secondary outcome [73]

Complications of cellulitis (Development of abscess requiring drainage)

Timepoint [73]

14 days

Secondary outcome [74]

Adverse events

Timepoint [74]

14 days

Secondary outcome [75]

Comparing patient costs

Timepoint [75]

14 days

Secondary outcome [76]

Quality of life (QOL) indicators

Timepoint [76]

1 year

Secondary outcome [77]

Cellulitis clinical score

Timepoint [77]

14 days

Secondary outcome [78]

Microbiology

Timepoint [78]

1 year

Eligibility

Key inclusion criteria

* Between 6 months and <18 years of age on day of signing informed consent is documented.
* Histologically- or cytologically-documented, locally-advanced, or metastatic solid malignancy or lymphoma that is incurable and has failed prior standard therapy, or for which no standard therapy exists, or for which no standard therapy is considered appropriate
* Any number of prior treatment regimens
* Tissue (or lymph node biopsy for rrcHL participants) available from an archival tissue sample or, if appropriate, a newly obtained core or excisional biopsy of a tumor lesion not previously irradiated
* Advanced melanoma or PD-L1-positive advanced, relapsed, or refractory solid tumor or lymphoma
* Measurable disease based on RECIST 1.1 (Or based on IWG [Cheson, 2007] [i.e., measurement must be >15 mm in longest diameter or >10 mm in short axis] for rrcHL participants)
* Participants with neuroblastoma with only metaiodobenzylguanidine (MIBG)-positive evaluable disease may be enrolled
* Lansky Play Scale =50 for participants from 6 months up to and including 16 years of age; or Karnofsky score =50 for participants >16 years of age
* Adequate organ function
* Female participants of childbearing potential should have a negative urine or serum pregnancy test within 72 hours before the first dose of study medication
* Female participant is not a woman of childbearing potential (WOCBP) or is a WOCBP who is abstinent from heterosexual intercourse or using contraception during the intervention period and for at least 120 days after the last dose of study intervention
* Contraceptive use by men should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
* Demonstrate adequate organ function.

Minimum age

6 Months

Maximum age

17 Years

Sex

Both males and females

Can healthy volunteers participate?

Key exclusion criteria

* Currently participating and receiving study therapy in, or has participated in a study of an investigational agent and received study therapy or used an investigational device within 4 weeks of the date of allocation/randomization
* Diagnosis of immunodeficiency or receiving systemic steroid therapy or any other form of immunosuppressive therapy within 7 days prior to the date of allocation/randomization
* Prior systemic anti-cancer therapy including investigational agent within 2 weeks prior to study Day 1 or not recovered from adverse events due to a previously administered agent
* Prior radiotherapy within 2 weeks of start of study treatment
* Known additional malignancy that is progressing or requires active treatment with the exception of basal cell carcinoma of the skin, squamous cell carcinoma of the skin or carcinoma in situ (eg, breast carcinoma, cervical carcinoma in situ) with potentially curative therapy, or in situ cervical cancer
* Known active central nervous system (CNS) metastases and/or carcinomatous meningitis
* Tumor(s) involving the brain stem
* Severe hypersensitivity (= Grade 3) to pembrolizumab and/or any of its excipients
* Active autoimmune disease that has required systemic treatment in past 2 years; replacement therapy (such as thyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency) is acceptable
* Has a history of (non-infectious) pneumonitis that required steroids or current pneumonitis.
* Active infection requiring systemic therapy
* Pregnant or breastfeeding, or expecting to conceive or father children within the projected duration of the trial through 120 days after the last dose of study medication
* Prior therapy with an anti-programmed cell death (PD)-1, anti-PD-ligand 1 (anti-PD-L1), anti-PD-L2 agent, or any agent directed to another stimulatory or inhibitory T-cell receptor (eg, cytotoxic lymphocyte associated protein-4 [CTLA-4], OX-40, CD137)
* Human immunodeficiency virus (HIV)
* Hepatitis B or C
* Known history of active tuberculosis (TB; Bacillus tuberculosis)
* Received a live vaccine within 30 days of planned start of study medication
* Has undergone solid organ transplant at any time, or prior allogeneic hematopoietic stem cell transplantation within the last 5 years. (Participants who have had an allogeneic hematopoietic transplant >5 years ago are eligible as long as there are no symptoms of Graft Versus Host Disease [GVHD].)
* History or current evidence of any condition, therapy, or laboratory abnormality, or known severe hypersensitivity to any component or analog of the trial treatment, that might confound the results of the trial, or interfere with the participant's participation for the full duration of the study
* Known psychiatric or substance abuse disorders that would interfere with the requirements of the study

Study design

Purpose of the study

Treatment

Allocation to intervention

Non-randomised trial

Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)

Methods used to generate the sequence in which subjects will be randomised (sequence generation)

Masking / blinding

Open (masking not used)

Who is / are masked / blinded?

Intervention assignment

Single group

Other design features

Phase

Phase 1

Type of endpoint/s

Statistical methods / analysis

Recruitment

Recruitment status

Recruiting

Data analysis

Reason for early stopping/withdrawal

Other reasons

Date of first participant enrolment

Anticipated

Actual

18/03/2015

Date of last participant enrolment

Anticipated

Actual

Date of last data collection

Anticipated

25/10/2027

Actual

Sample size

Target

370

Accrual to date

Final

Recruitment in Australia

Recruitment state(s)

SA,NSW,VIC,QLD,WA

Recruitment hospital [1]

University of Adelaide, Discipline of Medicine, Royal Adelaide Hospital - Adelaide

Recruitment hospital [2]

Royal North Shore Hospital - St. Leonards

Recruitment hospital [3]

Royal Prince Alfred - Sydney

Recruitment hospital [4]

Flinders Medical Centre - Adelaide

Recruitment hospital [5]

Monash Health - Clayton

Recruitment hospital [6]

Alfred Health - Prahran

Recruitment hospital [7]

Prince Charles - Brisbane

Recruitment hospital [8]

Prince Charles Hospital - Chermside

Recruitment hospital [9]

Monash Heart - Clayton

Recruitment hospital [10]

Royal Prince Alfred Hospital - Camperdown

Recruitment hospital [11]

Pendlebury Research - Cardiff

Recruitment hospital [12]

Australian Clinical Research Network - Maroubra

Recruitment hospital [13]

Illawara Diabetes Service - Wollongong

Recruitment hospital [14]

Q-Pharm - Herston

Recruitment hospital [15]

Ipswich Research Institute - Ipswich

Recruitment hospital [16]

AusTrials - Sherwood

Recruitment hospital [17]

CMAX - Adelaide

Recruitment hospital [18]

Box Hill Hospital - Box Hill

Recruitment hospital [19]

St. Vincent's Hospital - Fitzroy

Recruitment hospital [20]

Barwon Health - Geelong

Recruitment hospital [21]

Austin Health - Heidelberg West

Recruitment hospital [22]

Emeritus Research - Malvern East

Recruitment hospital [23]

Baker IDI Heart and Diabetes Institute - Melbourne

Recruitment hospital [24]

Keogh Institute for Medical Research - Nedlands

Recruitment hospital [25]

Royal Prince Alfred Hospital - Sydney

Recruitment hospital [26]

- Benowa

Recruitment hospital [27]

- Woolloongabba

Recruitment hospital [28]

- Parkville

Recruitment hospital [29]

- Fremantle

Recruitment hospital [30]

- St. Leonards

Recruitment hospital [31]

- Sydney

Recruitment hospital [32]

- Westmead

Recruitment hospital [33]

Flinders University - Adelaide

Recruitment hospital [34]

The Royal Children's Hospital Melbourne - Parkville

Recruitment postcode(s) [1]

5000 - Adelaide

Recruitment postcode(s) [2]

2065 - St. Leonards

Recruitment postcode(s) [3]

- Sydney

Recruitment postcode(s) [4]

5042 - Adelaide

Recruitment postcode(s) [5]

3168 - Clayton

Recruitment postcode(s) [6]

3181 - Prahran

Recruitment postcode(s) [7]

- Brisbane

Recruitment postcode(s) [8]

4032 - Chermside

Recruitment postcode(s) [9]

2050 - Camperdown

Recruitment postcode(s) [10]

2285 - Cardiff

Recruitment postcode(s) [11]

2035 - Maroubra

Recruitment postcode(s) [12]

2500 - Wollongong

Recruitment postcode(s) [13]

4006 - Herston

Recruitment postcode(s) [14]

4305 - Ipswich

Recruitment postcode(s) [15]

4075 - Sherwood

Recruitment postcode(s) [16]

3128 - Box Hill

Recruitment postcode(s) [17]

3065 - Fitzroy

Recruitment postcode(s) [18]

3220 - Geelong

Recruitment postcode(s) [19]

3081 - Heidelberg West

Recruitment postcode(s) [20]

3145 - Malvern East

Recruitment postcode(s) [21]

3004 - Melbourne

Recruitment postcode(s) [22]

6009 - Nedlands

Recruitment postcode(s) [23]

- Benowa

Recruitment postcode(s) [24]

- Woolloongabba

Recruitment postcode(s) [25]

- Parkville

Recruitment postcode(s) [26]

- Fremantle

Recruitment postcode(s) [27]

- St. Leonards

Recruitment postcode(s) [28]

- Westmead

Recruitment postcode(s) [29]

3072 - Parkville

Recruitment outside Australia

Country [1]

United States of America

State/province [1]

Colorado

Country [2]

United States of America

State/province [2]

Indiana

Country [3]

United States of America

State/province [3]

Massachusetts

Country [4]

United States of America

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New York

Country [5]

United States of America

State/province [5]

Texas

Country [6]

Brazil

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Sao Paulo

Country [7]

France

State/province [7]

Paris

Country [8]

Germany

State/province [8]

Haar

Country [9]

Israel

State/province [9]

Hod Hasharon

Country [10]

Italy

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Rome

Country [11]

Korea, Republic of

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Seoul

Country [12]

Netherlands

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Haarlem

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Sweden

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Stockholm

Country [14]

United Kingdom

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Hoddesdon

Country [15]

Czechia

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Olomouc

Country [16]

Czechia

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Prague

Country [17]

France

State/province [17]

Auvergne

Country [18]

France

State/province [18]

Caen

Country [19]

France

State/province [19]

Montpellier

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France

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Rouen

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France

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Toulouse

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Germany

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North Rhine-Westphalia

Country [23]

Germany

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Berlin

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Germany

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Bochum

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Germany

State/province [25]

Frankfurt am Main

Country [26]

Germany

State/province [26]

Frankfurt

Country [27]

Germany

State/province [27]

Freiburg

Country [28]

Germany

State/province [28]

Lübeck

Country [29]

Germany

State/province [29]

Recklinghausen

Country [30]

Netherlands

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Maastricht

Country [31]

New Zealand

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Grafton

Country [32]

Poland

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Poznan

Country [33]

United Kingdom

State/province [33]

Middlesex

Country [34]

United Kingdom

State/province [34]

Yorkshire

Country [35]

United Kingdom

State/province [35]

Newcastle upon Tyne

Country [36]

United States of America

State/province [36]

California

Country [37]

United States of America

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Michigan

Country [38]

United States of America

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Minnesota

Country [39]

United States of America

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New Mexico

Country [40]

United States of America

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North Carolina

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United States of America

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Ohio

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United States of America

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Oregon

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United States of America

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Washington

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United States of America

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Arizona

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United States of America

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Georgia

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United States of America

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Illinois

Country [47]

United States of America

State/province [47]

Pennsylvania

Country [48]

United States of America

State/province [48]

Virginia

Country [49]

United States of America

State/province [49]

Wisconsin

Country [50]

Denmark

State/province [50]

Copenhagen

Country [51]

France

State/province [51]

Lille

Country [52]

United Kingdom

State/province [52]

London

Country [53]

United States of America

State/province [53]

Florida

Country [54]

United States of America

State/province [54]

Missouri

Country [55]

United States of America

State/province [55]

Nevada

Country [56]

Canada

State/province [56]

Ontario

Funding & Sponsors

Primary sponsor type

Commercial sector/industry

Name

Merck Sharp & Dohme LLC

Address

Country

Other collaborator category [1]

Other

Name [1]

Menzies Institute for Medical Research

Address [1]

Country [1]

Other collaborator category [2]

Other

Name [2]

Autoimmunity Centers of Excellence

Address [2]

Country [2]

Other collaborator category [3]

Commercial sector/industry

Name [3]

Novartis Pharmaceuticals

Address [3]

Country [3]

Other collaborator category [4]

Commercial sector/industry

Name [4]

Medtronic

Address [4]

Country [4]

Other collaborator category [5]

Other

Name [5]

Flinders University

Address [5]

Country [5]

Other collaborator category [6]

Other

Name [6]

Australian Respiratory and Sleep Medicine Institute

Address [6]

Country [6]

Ethics approval

Ethics application status

Summary

Brief summary

Researchers are looking for new ways to treat children with different types of melanoma (skin cancer), solid tumors, and lymphomas (blood cancers) that are any of these:

* Advanced, which means cancer spread in the body or cannot be removed with surgery
* Relapsed, which means cancer has come back after it had responded to previous treatment (responded means it stopped growing, gets smaller, or disappeared)
* Refractory, which means cancer did not respond to previous treatment

Pembrolizumab is an immunotherapy, which is a treatment that helps the immune system fight cancer. Researchers want to learn if different doses of pembrolizumab can cause at least 1 of the types of cancer to get smaller or go away.

With Amendment 8, enrolment of participants with solid tumours and participants 6 months to under 12 years old with melanoma were closed. Enrolment of participants 12-18 years old with melanoma continues. Enrolment of participants who have tumours with specific traits (microsatellite-instability-high (MSI-H), and tumour-mutational burden-high =10 mutation/Mb (TMB-H)) also continues.

Trial website

https://clinicaltrials.gov/study/NCT02332668

Trial related presentations / publications

Geoerger B, Kang HJ, Yalon-Oren M, Marshall LV, Vezina C, Pappo A, Laetsch TW, Petrilli AS, Ebinger M, Toporski J, Glade-Bender J, Nicholls W, Fox E, DuBois SG, Macy ME, Cohn SL, Pathiraja K, Diede SJ, Ebbinghaus S, Pinto N. Pembrolizumab in paediatric patients with advanced melanoma or a PD-L1-positive, advanced, relapsed, or refractory solid tumour or lymphoma (KEYNOTE-051): interim analysis of an open-label, single-arm, phase 1-2 trial. Lancet Oncol. 2020 Jan;21(1):121-133. doi: 10.1016/S1470-2045(19)30671-0. Epub 2019 Dec 4.
Wu T, Rayner CK, Young RL, Horowitz M. Gut motility and enteroendocrine secretion. Curr Opin Pharmacol. 2013 Dec;13(6):928-34. doi: 10.1016/j.coph.2013.09.002. Epub 2013 Sep 20.
Chaikomin R, Rayner CK, Jones KL, Horowitz M. Upper gastrointestinal function and glycemic control in diabetes mellitus. World J Gastroenterol. 2006 Sep 21;12(35):5611-21. doi: 10.3748/wjg.v12.i35.5611.
Horowitz M, Edelbroek MA, Wishart JM, Straathof JW. Relationship between oral glucose tolerance and gastric emptying in normal healthy subjects. Diabetologia. 1993 Sep;36(9):857-62. doi: 10.1007/BF00400362.
Jones KL, Horowitz M, Carney BI, Wishart JM, Guha S, Green L. Gastric emptying in early noninsulin-dependent diabetes mellitus. J Nucl Med. 1996 Oct;37(10):1643-8.
Horowitz M, Rayner CK, Jones KL. Mechanisms and clinical efficacy of lixisenatide for the management of type 2 diabetes. Adv Ther. 2013 Feb;30(2):81-101. doi: 10.1007/s12325-013-0009-4. Epub 2013 Feb 13.
Baggio LL, Drucker DJ. Biology of incretins: GLP-1 and GIP. Gastroenterology. 2007 May;132(6):2131-57. doi: 10.1053/j.gastro.2007.03.054.
ELRICK H, STIMMLER L, HLAD CJ Jr, ARAI Y. PLASMA INSULIN RESPONSE TO ORAL AND INTRAVENOUS GLUCOSE ADMINISTRATION. J Clin Endocrinol Metab. 1964 Oct;24:1076-82. doi: 10.1210/jcem-24-10-1076. No abstract available.
Nauck M, Stockmann F, Ebert R, Creutzfeldt W. Reduced incretin effect in type 2 (non-insulin-dependent) diabetes. Diabetologia. 1986 Jan;29(1):46-52. doi: 10.1007/BF02427280.
Nauck MA, Heimesaat MM, Orskov C, Holst JJ, Ebert R, Creutzfeldt W. Preserved incretin activity of glucagon-like peptide 1 [7-36 amide] but not of synthetic human gastric inhibitory polypeptide in patients with type-2 diabetes mellitus. J Clin Invest. 1993 Jan;91(1):301-7. doi: 10.1172/JCI116186.
Nauck MA, Niedereichholz U, Ettler R, Holst JJ, Orskov C, Ritzel R, Schmiegel WH. Glucagon-like peptide 1 inhibition of gastric emptying outweighs its insulinotropic effects in healthy humans. Am J Physiol. 1997 Nov;273(5):E981-8. doi: 10.1152/ajpendo.1997.273.5.E981.
Khoo J, Rayner CK, Jones KL, Horowitz M. Incretin-based therapies: new treatments for type 2 diabetes in the new millennium. Ther Clin Risk Manag. 2009 Jun;5(3):683-98. doi: 10.2147/tcrm.s4975. Epub 2009 Aug 20.
Stevens JE, Horowitz M, Deacon CF, Nauck M, Rayner CK, Jones KL. The effects of sitagliptin on gastric emptying in healthy humans - a randomised, controlled study. Aliment Pharmacol Ther. 2012 Aug;36(4):379-90. doi: 10.1111/j.1365-2036.2012.05198.x. Epub 2012 Jun 28.
Vella A, Bock G, Giesler PD, Burton DB, Serra DB, Saylan ML, Dunning BE, Foley JE, Rizza RA, Camilleri M. Effects of dipeptidyl peptidase-4 inhibition on gastrointestinal function, meal appearance, and glucose metabolism in type 2 diabetes. Diabetes. 2007 May;56(5):1475-80. doi: 10.2337/db07-0136. Epub 2007 Feb 15.
Woerle H, Lindenberger T, Linke R, Foley JE, Ligueros-Sayalan AA, ZhangY, He Y-L, BelingerC, Goeke B, Schirra J. A single dose of vidagliptin (VILDA) decelerates gastric emptying (GE) in patients with type 2 diabetes (T2DM). American Diabetes Association, 67th Scientific Sessions 500-p (abstract), 2007.
Wu T, Bound MJ, Zhao BR, Standfield SD, Bellon M, Jones KL, Horowitz M, Rayner CK. Effects of a D-xylose preload with or without sitagliptin on gastric emptying, glucagon-like peptide-1, and postprandial glycemia in type 2 diabetes. Diabetes Care. 2013 Jul;36(7):1913-8. doi: 10.2337/dc12-2294. Epub 2013 Jan 28.
Rayner CK, Samsom M, Jones KL, Horowitz M. Relationships of upper gastrointestinal motor and sensory function with glycemic control. Diabetes Care. 2001 Feb;24(2):371-81. doi: 10.2337/diacare.24.2.371.
Pilichiewicz AN, Chaikomin R, Brennan IM, Wishart JM, Rayner CK, Jones KL, Smout AJ, Horowitz M, Feinle-Bisset C. Load-dependent effects of duodenal glucose on glycemia, gastrointestinal hormones, antropyloroduodenal motility, and energy intake in healthy men. Am J Physiol Endocrinol Metab. 2007 Sep;293(3):E743-53. doi: 10.1152/ajpendo.00159.2007. Epub 2007 Jul 3.
Ma J, Pilichiewicz AN, Feinle-Bisset C, Wishart JM, Jones KL, Horowitz M, Rayner CK. Effects of variations in duodenal glucose load on glycaemic, insulin, and incretin responses in type 2 diabetes. Diabet Med. 2012 May;29(5):604-8. doi: 10.1111/j.1464-5491.2011.03496.x.
Jansen RW, Lipsitz LA. Postprandial hypotension: epidemiology, pathophysiology, and clinical management. Ann Intern Med. 1995 Feb 15;122(4):286-95. doi: 10.7326/0003-4819-122-4-199502150-00009.
Jones KL, Tonkin A, Horowitz M, Wishart JM, Carney BI, Guha S, Green L. Rate of gastric emptying is a determinant of postprandial hypotension in non-insulin-dependent diabetes mellitus. Clin Sci (Lond). 1998 Jan;94(1):65-70. doi: 10.1042/cs0940065.
Russo A, Stevens JE, Wilson T, Wells F, Tonkin A, Horowitz M, Jones KL. Guar attenuates fall in postprandial blood pressure and slows gastric emptying of oral glucose in type 2 diabetes. Dig Dis Sci. 2003 Jul;48(7):1221-9. doi: 10.1023/a:1024182403984.
Vanis L, Gentilcore D, Rayner CK, Wishart JM, Horowitz M, Feinle-Bisset C, Jones KL. Effects of small intestinal glucose load on blood pressure, splanchnic blood flow, glycemia, and GLP-1 release in healthy older subjects. Am J Physiol Regul Integr Comp Physiol. 2011 Jun;300(6):R1524-31. doi: 10.1152/ajpregu.00378.2010. Epub 2011 Mar 9.
Jian ZJ, Zhou BY. Efficacy and safety of acarbose in the treatment of elderly patients with postprandial hypotension. Chin Med J (Engl). 2008 Oct 20;121(20):2054-9.
Sasaki E, Goda K, Nagata K, Kitaoka H, Ohsawa N, Hanafusa T. Acarbose improved severe postprandial hypotension in a patient with diabetes mellitus. J Diabetes Complications. 2001 May-Jun;15(3):158-61. doi: 10.1016/s1056-8727(01)00138-6.
Gentilcore D, Bryant B, Wishart JM, Morris HA, Horowitz M, Jones KL. Acarbose attenuates the hypotensive response to sucrose and slows gastric emptying in the elderly. Am J Med. 2005 Nov;118(11):1289. doi: 10.1016/j.amjmed.2005.05.019. No abstract available.
Yonenaga A, Ota H, Honda M, Koshiyama D, Yagi T, Hanaoka Y, Yamamoto H, Yamaguchi Y, Iijima K, Akishita M, Ouchi Y. Marked improvement of elderly postprandial hypotension by dipeptidyl peptidase IV inhibitor. Geriatr Gerontol Int. 2013 Jan;13(1):227-9. doi: 10.1111/j.1447-0594.2012.00903.x. No abstract available.
Cockcroft DW, Gault MH. Prediction of creatinine clearance from serum creatinine. Nephron. 1976;16(1):31-41. doi: 10.1159/000180580.
Trahair LG, Vanis L, Gentilcore D, Lange K, Rayner CK, Horowitz M, Jones KL. Effects of variations in duodenal glucose load on blood pressure, heart rate, superior mesenteric artery blood flow and plasma noradrenaline in healthy young and older subjects. Clin Sci (Lond). 2012 Mar;122(6):271-9. doi: 10.1042/CS20110270.
Parker BA, Sturm K, MacIntosh CG, Feinle C, Horowitz M, Chapman IM. Relation between food intake and visual analogue scale ratings of appetite and other sensations in healthy older and young subjects. Eur J Clin Nutr. 2004 Feb;58(2):212-8. doi: 10.1038/sj.ejcn.1601768.
Ahren B. DPP-4 inhibitors. Best Pract Res Clin Endocrinol Metab. 2007 Dec;21(4):517-33. doi: 10.1016/j.beem.2007.07.005.
Information JsP: Merck Sharp & Dohme (Australia) Pty Ltd. South Granville, NSW, Australia, 2008.
Deacon CF. Dipeptidyl peptidase-4 inhibitors in the treatment of type 2 diabetes: a comparative review. Diabetes Obes Metab. 2011 Jan;13(1):7-18. doi: 10.1111/j.1463-1326.2010.01306.x.
Schofer J, Siminiak T, Haude M, Herrman JP, Vainer J, Wu JC, Levy WC, Mauri L, Feldman T, Kwong RY, Kaye DM, Duffy SJ, Tubler T, Degen H, Brandt MC, Van Bibber R, Goldberg S, Reuter DG, Hoppe UC. Percutaneous mitral annuloplasty for functional mitral regurgitation: results of the CARILLON Mitral Annuloplasty Device European Union Study. Circulation. 2009 Jul 28;120(4):326-33. doi: 10.1161/CIRCULATIONAHA.109.849885. Epub 2009 Jul 13.
Siminiak T, Wu JC, Haude M, Hoppe UC, Sadowski J, Lipiecki J, Fajadet J, Shah AM, Feldman T, Kaye DM, Goldberg SL, Levy WC, Solomon SD, Reuter DG. Treatment of functional mitral regurgitation by percutaneous annuloplasty: results of the TITAN Trial. Eur J Heart Fail. 2012 Aug;14(8):931-8. doi: 10.1093/eurjhf/hfs076. Epub 2012 May 21.
Siminiak T, Hoppe UC, Schofer J, Haude M, Herrman JP, Vainer J, Firek L, Reuter DG, Goldberg SL, Van Bibber R. Effectiveness and safety of percutaneous coronary sinus-based mitral valve repair in patients with dilated cardiomyopathy (from the AMADEUS trial). Am J Cardiol. 2009 Aug 15;104(4):565-70. doi: 10.1016/j.amjcard.2009.04.021. Epub 2009 May 29.
Witte KK, Lipiecki J, Siminiak T, Meredith IT, Malkin CJ, Goldberg SL, Stark MA, von Bardeleben RS, Cremer PC, Jaber WA, Celermajer DS, Kaye DM, Sievert H. The REDUCE FMR Trial: A Randomized Sham-Controlled Study of Percutaneous Mitral Annuloplasty in Functional Mitral Regurgitation. JACC Heart Fail. 2019 Nov;7(11):945-955. doi: 10.1016/j.jchf.2019.06.011. Epub 2019 Sep 11.
Proietto J, Malloy J, Zhuang D, Arya M, Cohen ND, de Looze FJ, Gilfillan C, Griffin P, Hall S, Nathow T, Oldfield GS, O'Neal DN, Roberts A, Stuckey BGA, Yue D, Taylor K, Kim D. Efficacy and safety of methionine aminopeptidase 2 inhibition in type 2 diabetes: a randomised, placebo-controlled clinical trial. Diabetologia. 2018 Sep;61(9):1918-1922. doi: 10.1007/s00125-018-4677-0. Epub 2018 Jul 11.
Wu Q, Mills EA, Wang Q, Dowling CA, Fisher C, Kirch B, Lundy SK, Fox DA, Mao-Draayer Y; AMS04 Study Group. Siponimod enriches regulatory T and B lymphocytes in secondary progressive multiple sclerosis. JCI Insight. 2020 Feb 13;5(3):e134251. doi: 10.1172/jci.insight.134251.
Zahr F, Song HK, Chadderdon SM, Gada H, Mumtaz M, Byrne T, Kirshner M, Bajwa T, Weiss E, Kodali S, George I, Heiser J, Merhi WM, Thaden JJ, Zhang A, Lim DS, Reardon MJ, Adams DH, Mack MJ, Leon MB. 30-Day Outcomes Following Transfemoral Transseptal Transcatheter Mitral Valve Replacement: Intrepid TMVR Early Feasibility Study Results. JACC Cardiovasc Interv. 2022 Jan 10;15(1):80-89. doi: 10.1016/j.jcin.2021.10.018. Epub 2021 Nov 6.
Bapat V, Rajagopal V, Meduri C, Farivar RS, Walton A, Duffy SJ, Gooley R, Almeida A, Reardon MJ, Kleiman NS, Spargias K, Pattakos S, Ng MK, Wilson M, Adams DH, Leon M, Mack MJ, Chenoweth S, Sorajja P; Intrepid Global Pilot Study Investigators. Early Experience With New Transcatheter Mitral Valve Replacement. J Am Coll Cardiol. 2018 Jan 2;71(1):12-21. doi: 10.1016/j.jacc.2017.10.061. Epub 2017 Nov 16.
Ibrahim LF, Huang L, Hopper SM, Dalziel K, Babl FE, Bryant PA. Intravenous ceftriaxone at home versus intravenous flucloxacillin in hospital for children with cellulitis: a cost-effectiveness analysis. Lancet Infect Dis. 2019 Oct;19(10):1101-1108. doi: 10.1016/S1473-3099(19)30288-9. Epub 2019 Aug 13.
Ibrahim LF, Hopper SM, Orsini F, Daley AJ, Babl FE, Bryant PA. Efficacy and safety of intravenous ceftriaxone at home versus intravenous flucloxacillin in hospital for children with cellulitis (CHOICE): a single-centre, open-label, randomised, controlled, non-inferiority trial. Lancet Infect Dis. 2019 May;19(5):477-486. doi: 10.1016/S1473-3099(18)30729-1. Epub 2019 Mar 7.
Ibrahim LF, Babl FE, Orsini F, Hopper SM, Bryant PA. Cellulitis: Home Or Inpatient in Children from the Emergency Department (CHOICE): protocol for a randomised controlled trial. BMJ Open. 2016 Jan 11;6(1):e009606. doi: 10.1136/bmjopen-2015-009606.

Public notes

Contacts

Principal investigator

Name

Medical Director

Address

Merck Sharp & Dohme LLC

Country

Phone

Fax

Contact person for public queries

Name

Toll Free Number

Address

Country

Phone

1-888-577-8839

Fax

Contact person for scientific queries

Data sharing statement

Will individual participant data (IPD) for this trial be available (including data dictionaries)?

Yes

What data in particular will be shared?

http://engagezone.msd.com/doc/ProcedureAccessClinicalTrialData.pdf

When will data be available (start and end dates)?

Available to whom?

Available for what types of analyses?

How or where can data be obtained?

IPD available at link: http://engagezone.msd.com/ds_documentation.php

What supporting documents are/will be available?

No Supporting Document Provided

Results publications and other study-related documents

Type	Citations or Other Details
Journal	Wu Q, Mills EA, Wang Q, Dowling CA, Fisher C, Kirc... [More Details]

Results not provided in https://clinicaltrials.gov/study/NCT02332668

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For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02332668