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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05233397


Additional trial details provided through ANZCTR are available at the end of this record.


Registration number
NCT05233397
Ethics application status
Date submitted
25/01/2022
Date registered
10/02/2022

Titles & IDs
Public title
ACTEMRA® for the Treatment of Pediatric Adamantinomatous Craniopharyngioma
Scientific title
Phase 2 Study of Systemic IL-6 Receptor Antagonist ACTEMRA® (Tocilizumab) for the Treatment of Progressive/Recurrent Pediatric Adamantinomatous Craniopharyngioma
Secondary ID [1] 0 0
CONNECT1905
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Adamantinomatous Craniopharyngioma 0 0
Recurrent Adamantinomatous Craniopharyngioma 0 0
Condition category
Condition code
Cancer 0 0 0 0
Brain
Cancer 0 0 0 0
Bone

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Tocilizumab

Experimental: Stratum 1 and Stratum 2 - Stratum 1: Patients with progressive or recurrent adamantinomatous craniopharyngiomas following radiation therapy.

Stratum 2: Patients with measurable adamantinomatous craniopharyngioma who have undergone surgery but have not previously received radiation therapy. Progressive disease is allowed but not required


Treatment: Drugs: Tocilizumab
For \< 30 kg: 12 mg/kg IV every 2 weeks; For =30 kg: 8 mg/kg IV every 2 weeks
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Sustained objective response rate of patients with recurrent/progressive previously irradiated ACP to treatment with systemic tocilizumab
Timepoint [1] 0 0
From Day 1 of treatment through 30 days following end of protocol treatment
Primary outcome [2] 0 0
Sustained objective response rate of patients with measurable ACP who have undergone surgery but have not been previously treated with radiation to treatment with systemic tocilizumab
Timepoint [2] 0 0
From Day 1 of treatment through 30 days following end of protocol treatment
Secondary outcome [1] 0 0
Biological effects of tocilizumab on ACP tumor tissue and cyst fluid.
Timepoint [1] 0 0
From Day 1 of treatment through 30 days following end of protocol treatment
Secondary outcome [2] 0 0
Toxicities associated with tocilizumab in children with ACP
Timepoint [2] 0 0
From Day 1 of treatment through 30 days following end of protocol treatment
Secondary outcome [3] 0 0
PFS of ACP patients treated with tocilizumab after radiation
Timepoint [3] 0 0
12 months
Secondary outcome [4] 0 0
PFS of ACP patients treated with tocilizumab who have not received radiation
Timepoint [4] 0 0
12 months

Eligibility
Key inclusion criteria
1. Age: Patients must be = 12 months and = 25 years of age at the time of study enrollment.
2. Diagnosis: Patients with histologically-confirmed adamantinomatous craniopharyngioma (ACP) Histologic confirmation of ACP may be made on solid tumor or, if no solid tumor can be safely obtained, cyst fluid with classic ACP characteristics of thick, cholesterol-rich, greenish-brown liquid in the context of imaging features consistent with craniopharyngioma, including lobulated, cystic/solid mass with calcifications that originates in the sellar/suprasellar region.
3. Disease Status: Patients must have measurable disease.

* Stratum 1: Patients with progressive or recurrent ACP who demonstrate cystic and/or solid recurrence or progression at least 6 months post completion of radiation therapy
* Stratum 2: Patients with measurable ACP who have undergone surgery but have NOT previously undergone irradiation (but may have received prior systemic or intracystic therapy). Progressive disease is allowed but not required.
4. Performance Level: Karnofsky = 50% for patients > 16 years of age and Lansky = 50 for patients = 16 years of age (See Appendix I). Note: Neurologic deficits in patients with CNS tumors must have been stable for at least 7 days prior to study enrollment. Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.
5. Prior Therapy: Patients must have recovered or stabilized from the acute toxic effects of prior treatments

* Biologic (anti-neoplastic agent): At least 7 days must have elapsed after the last (systemic or intracystic) dose of a biologic agent. For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur. The duration of this interval must be discussed with the study chair
* Immunotherapy: At least 42 days after the completion of any type of systemic immunotherapy, e.g. tumor vaccines.
* Monoclonal antibodies: At least 21 days after the last dose of a monoclonal antibody.
* Radiation therapy: Patients must have had their last (conventional or hypofractionated) fraction of: a) Focal irradiation > 6 months prior to enrollment and b) No prior craniospinal irradiation is permitted.
* Corticosteroids: Patients receiving dexamethasone must be on a stable or decreasing dose for at least 1 week prior to enrollment
* Myelosuppressive systemic therapy: At least 21 days must have elapsed after the last systemic myelosuppressive therapy.
* Surgery: At least 6 weeks must have elapsed since surgery.
6. Organ Function Requirements

Adequate Bone Marrow Function Defined as:
* Peripheral absolute neutrophil count (ANC) =1000/mm3
* Platelet count =100,000/mm3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment)
* Hemoglobin >8 g/dL (may be transfused)

Adequate Renal Function Defined as:
* Creatinine clearance or radioisotope GFR > 70ml/min/1.73 m2 or
* A serum creatinine based on (Schwartz et al. J. Peds, 106:522, 1985) age/gender as follows:

1 to < 2 years: maximum serum creatinine 0.6 mg/dL for males and females. 2 to < 6 years: maximum serum creatinine 0.8 mg/dL for males and females. 6 to < 10 years: maximum serum creatinine 1.0 mg/dL for males and females. 10 to < 13 years: maximum serum creatinine 1.2 mg/dL for males and females. 13 to < 16 years: maximum serum creatinine 1.5 mg/dL for males and 1.4 mg/dL for females.

= 16 years: maximum serum creatinine 1.7 mg/dL for males and 1.4 mg/dL for females.

Adequate Liver Function Defined as:
* Total bilirubin within normal institutional limits
* AST (SGOT) = 2.5 × institutional upper limit of normal
* ALT (SGPT) = 2.5 × institutional upper limit of normal

Adequate Neurologic Function Defined as:
* Patients with neurological deficits should have deficits that are stable for a minimum of 1 week prior to enrollment.
* Patients with current seizure disorders may be enrolled if seizures are well-controlled on antiepileptic therapies.
7. Informed Consent: All patients and/or their parents or legally authorized representatives must sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines.
Minimum age
1 Year
Maximum age
25 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Pregnancy or Breast-Feeding: Pregnant or breast-feeding women will not be entered on this study due to unknown risks of fetal and teratogenic adverse events as seen in animal/human studies. Pregnancy tests must be obtained in girls who are post-menarchal. Males or females of reproductive potential may not participate unless they have agreed to use an effective contraceptive method for at least 90 days after discontinuation of drug for females and at least 60 days for males. For females of childbearing potential, agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive methods (bilateral tubal ligation, male sterilization, hormonal contraceptives that inhibit ovulation, hormone-releasing intrauterine devices, and copper intrauterine devices; hormonal contraceptive methods must be supplemented by a barrier method) and agreement to refrain from donating eggs are required. For males of reproductive potential, agreement to remain abstinent (refrain from heterosexual intercourse) or use a condom, and agreement to refrain from donating sperm.
2. Gastrointestinal Disease: Patients with a history of serious gastrointestinal disease, including inflammatory bowel disease or gastrointestinal perforation
3. Concomitant Medications

* Corticosteroids: Patients receiving corticosteroids who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment are not eligible.
* Investigational Drugs: Patients who are currently receiving another investigational drug are not eligible.
* Anti-cancer Agents: Patients who are currently receiving other anti-cancer agents are not eligible.
4. Study Specific:

* Patients who have an uncontrolled infection are not eligible.
* Patients who have received any live or attenuated vaccinations within three months prior to start of therapy are not eligible.
* Any significant concurrent medical or surgical condition that would jeopardize the patient's safety or ability to complete the study, including, but not limited to, disease of the nervous, renal, hepatic, cardiac (such as symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia), pulmonary, or endocrine system
* Patients who have a history of Human Immunodeficiency Virus, Hepatitis B Virus, Hepatitis C Virus or Tuberculosis infection are not eligible.
* Patients who have received a prior solid organ transplantation are not eligible.
* Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible.
* Patients who have a history of alcohol, drug, or chemical abuse within 6 months of screening.
* Patients who have had surgery within the last 6 weeks or who have concerns for poor postsurgical wound healing.
* Patients who have a history of allergic reactions attributed to compounds of similar chemical or biologic composition to tocilizumab and its excipients are not eligible.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
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 2
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
16/12/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/12/2027
Actual
Sample size
Target
38
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,WA
Recruitment hospital [1] 0 0
Sydney Children's Hospital - Randwick
Recruitment hospital [2] 0 0
Queensland Children's Hospital - South Brisbane
Recruitment hospital [3] 0 0
Perth Children's Hospital - Perth
Recruitment postcode(s) [1] 0 0
2031 - Randwick
Recruitment postcode(s) [2] 0 0
4101 - South Brisbane
Recruitment postcode(s) [3] 0 0
6000 - Perth
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Colorado
Country [2] 0 0
United States of America
State/province [2] 0 0
District of Columbia
Country [3] 0 0
United States of America
State/province [3] 0 0
North Carolina
Country [4] 0 0
United States of America
State/province [4] 0 0
Ohio
Country [5] 0 0
Canada
State/province [5] 0 0
Quebec

Funding & Sponsors
Primary sponsor type
Other
Name
Nationwide Children's Hospital
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
Children's Hospital Colorado
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Holly Lindsay, MD
Address 0 0
Children's Hospital Colorado
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Amy K Jones, MSN
Address 0 0
Country 0 0
Phone 0 0
16147223284
Fax 0 0
Email 0 0
connect@nationwidechildrens.org
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05233397

Additional trial details provided through ANZCTR
Accrual to date
1
Recruiting in Australia
Recruitment state(s)
NSW,QLD,WA
Recruitment hospital [1] 127
Sydney Children's Hospital
Recruitment hospital [2] 128
Queensland Children's Hospital
Recruitment hospital [3] 129
Perth Children's Hospital
Recruitment postcode(s) [1] 129
2031
Recruitment postcode(s) [2] 130
4101
Recruitment postcode(s) [3] 131
6009
Funding & Sponsors
Primary sponsor
Other Collaborative groups
Primary sponsor name
Australia and New Zealand Children’s Haematology/Oncology Group (ANZCHOG
Primary sponsor address
27-31 Wright Street
Clayton, VIC, 3168
Primary sponsor country
Australia
Ethics approval
Ethics application status
Approved
Ethics committee name [1] 57
Child and Adolescent Health Service Human Research Ethics Committee
Address [1] 57
15 Hospital Avenue Nedlands, WA, 6009
Country [1] 57
Date submitted for ethics approval [1] 57
20/09/2022
Approval date [1] 57
26/10/2022
Ethics approval number [1] 57
RGS0000005636
 
Public notes

Contacts
Principal investigator
Title 369 0
Dr
Name 369 0
Neevkia Manoharan
Address 369 0
Sydney Children’s Hospital Kids Cancer Centre Level 1 South Wing, High Street Randwick 2031, NSW Australia
Country 369 0
Australia
Phone 369 0
+61 2 9382 1730
Fax 369 0
Email 369 0
 Neevika.Manoharan@health.nsw.gov.au
Contact person for public queries
Title 370 0
Ms
Name 370 0
Robyn Strong
Address 370 0
27-31 Wright Street Clayton, VIC, 3168
Country 370 0
Australia
Phone 370 0
+613 8572 2684
Fax 370 0
Email 370 0
anzchog_connect1905@anzchog.org
Contact person for scientific queries
Title 371 0
Dr
Name 371 0
Neevkia Manoharan
Address 371 0
Sydney Children’s Hospital Kids Cancer Centre Level 1 South Wing, High Street Randwick 2031, NSW Australia
Country 371 0
Australia
Phone 371 0
+61 2 9382 1730
Fax 371 0
Email 371 0
 Neevika.Manoharan@health.nsw.gov.au