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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02310763

Registration number

NCT02310763

Ethics application status

Date submitted

4/11/2014

Date registered

8/12/2014

Titles & IDs

Public title

A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy

Scientific title

A PHASE 2 RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTIPLE ASCENDING DOSE STUDY TO EVALUATE THE SAFETY, EFFICACY, PHARMACOKINETICS AND PHARMACODYNAMICS OF PF-06252616 IN AMBULATORY BOYS WITH DUCHENNE MUSCULAR DYSTROPHY

Secondary ID [1]

2014-002072-92

Secondary ID [2]

B5161002

Universal Trial Number (UTN)

Trial acronym

Linked study record

Health condition

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

Duchenne Muscular Dystrophy

Condition category

Condition code

Musculoskeletal

Other muscular and skeletal disorders

Human Genetics and Inherited Disorders

Other human genetics and inherited disorders

Neurological

Other neurological disorders

Intervention/exposure

Study type

Interventional

Description of intervention(s) / exposure

Treatment: Other - PF-06252616

Experimental: PF-06252616 - 3 dose levels (5mg/kg, 20mg/kg and 40 mg/kg) of IV infused PF-06252616 will be investigated within each subject

Placebo comparator: Placebo - Matching Placebo

Treatment: Other: PF-06252616
PF-06252616 IV Infusion, 3 dose levels (5mg/kg, 20 mg/kg and 40 mg/kg) will be investigated within each subject

Intervention code [1]

Treatment: Other

Comparator / control treatment

Control group

Outcomes

Primary outcome [1]

Number of Participants With Treatment-emergent Adverse Events (TEAEs) by Week 49

Timepoint [1]

Study Day 1 to Week 49 visit

Primary outcome [2]

Number of Participants Who Discontinued From the Study Due to TEAEs by Week 49

Timepoint [2]

Study Day 1 to Week 49 visit

Primary outcome [3]

Number of Participants With Dose Reduced or Temporary Discontinuation Due to TEAEs by Week 49

Timepoint [3]

Study Day 1 to Week 49 visit

Primary outcome [4]

Number of Participants With Laboratory Test Abnormalities (Without Regard to Baseline Abnormality) by Week 49 - Hematology

Timepoint [4]

Baseline to Week 49 visit

Primary outcome [5]

Number of Participants With Laboratory Test Abnormalities (Without Regard to Baseline Abnormality) by Week 49 - Coagulation

Timepoint [5]

Baseline to Week 49 visit

Primary outcome [6]

Number of Participants With Laboratory Test Abnormalities (Without Regard to Baseline Abnormality) by Week 49 - Liver Function

Timepoint [6]

Baseline to Week 49 visit

Primary outcome [7]

Number of Participants With Laboratory Test Abnormalities (Without Regard to Baseline Abnormality) by Week 49 - Renal Function

Timepoint [7]

Baseline to Week 49 visit

Primary outcome [8]

Number of Participants With Laboratory Test Abnormalities (Without Regard to Baseline Abnormality) by Week 49 - Electrolytes

Timepoint [8]

Baseline to Week 49 visit

Primary outcome [9]

Number of Participants With Laboratory Test Abnormalities (Without Regard to Baseline Abnormality) by Week 49 - Hormones

Timepoint [9]

Baseline to Week 49 visit

Primary outcome [10]

Number of Participants With Laboratory Test Abnormalities (Without Regard to Baseline Abnormality) by Week 49 - Clinical Chemistry

Timepoint [10]

Baseline to Week 49 visit

Primary outcome [11]

Number of Participants With Laboratory Test Abnormalities (Without Regard to Baseline Abnormality) by Week 49 - Urinalysis

Timepoint [11]

Baseline to Week 49 visit

Primary outcome [12]

Number of Participants With Laboratory Test Abnormalities (Without Regard to Baseline Abnormality) by Week 49 - Fecal

Timepoint [12]

Baseline to Week 49 visit

Primary outcome [13]

Categorical Summary of Liver Iron Accumulation by Week 49

Timepoint [13]

Screening, Weeks 13, 29 and 45

Primary outcome [14]

Number of Participants With Physical Examination Findings Reported as SAEs by Week 49

Timepoint [14]

Baseline to Week 49 visit

Primary outcome [15]

Summary of Tanner Stage Rating by Week 49

Timepoint [15]

Baseline, Weeks 17, 33 and 49

Primary outcome [16]

Number of Participants With Vital Signs Findings Reported as SAEs by Week 49

Timepoint [16]

Baseline to Week 49 visit

Primary outcome [17]

Number of Participants With Electrocardiogram (ECG) Data Meeting Pre-specified Criteria by Week 49

Timepoint [17]

Baseline to Week 49 visit

Primary outcome [18]

Change From Baseline in Left Ventricular Ejection Fraction (LVEF) as Compared to Placebo by Week 49

Timepoint [18]

Baseline to Week 49 visit

Primary outcome [19]

Height-adjusted Z-score of Lumbar Spine Bone Mineral Density Over Time by Week 49

Timepoint [19]

Screening and Week 49

Primary outcome [20]

Bone Age to Chronological Age Ratio by Week 49

Timepoint [20]

Screening, Weeks 17, 33 and 49

Primary outcome [21]

Number of Participants With Suicidal Ideation and Suicidal Behavior Reported as AEs by Week 49

Timepoint [21]

Baseline to Week 49 visit

Primary outcome [22]

Change From Baseline on the 4 Stair Climb (4SC) as Compared to Placebo at Weeks 17, 33 and 49

Timepoint [22]

Baseline, Weeks 17, 33 and 49

Secondary outcome [1]

Change From Baseline as Compared to Placebo on Forced Vital Capacity (FVC) at Weeks 17, 33 and 49

Timepoint [1]

Baseline, Weeks 17, 33 and 49

Secondary outcome [2]

Change From Baseline as Compared to Placebo on the Northstar Ambulatory Assessment (NSAA) at Weeks 17, 33 and 49

Timepoint [2]

Baseline, Weeks 17, 33 and 49

Secondary outcome [3]

Change From Baseline as Compared to Placebo on the Ankle Range of Motion (ROM) at Weeks 17, 33 and 49

Timepoint [3]

Baseline, Weeks 17, 33 and 49

Secondary outcome [4]

Change From Baseline as Compared to Placebo on the Performance of Upper Limb (PUL) Overall Score at Weeks 17, 33 and 49

Timepoint [4]

Baseline, Weeks 17, 33 and 49

Secondary outcome [5]

Change From Baseline as Compared to Placebo on the Six Minute Walk Distance (6MWD) Score at Weeks 17, 33 and 49

Timepoint [5]

Baseline, Weeks 17, 33 and 49

Secondary outcome [6]

Change From Baseline as Compared to Placebo on Muscle Strength of Elbow Extension at Weeks 17, 33 and 49

Timepoint [6]

Baseline, Weeks 17, 33 and 49

Secondary outcome [7]

Change From Baseline as Compared to Placebo on Muscle Strength of Elbow Flexion at Weeks 17, 33 and 49

Timepoint [7]

Baseline, Weeks 17, 33 and 49

Secondary outcome [8]

Change From Baseline as Compared to Placebo on Muscle Strength of Hip Abduction at Weeks 17, 33 and 49

Timepoint [8]

Baseline, Weeks 17, 33 and 49

Secondary outcome [9]

Change From Baseline as Compared to Placebo on Muscle Strength of Knee Extension at Weeks 17, 33 and 49

Timepoint [9]

Baseline, Weeks 17, 33 and 49

Secondary outcome [10]

Change From Baseline as Compared to Placebo on Muscle Strength of Shoulder Abduction at Weeks 17, 33 and 49

Timepoint [10]

Baseline, Weeks 17, 33 and 49

Secondary outcome [11]

Change From Baseline to Week 49 on 4SC for Participants in Sequence 3 Compared to the Natural History Control Group

Timepoint [11]

Baseline, Week 49

Secondary outcome [12]

Change From Baseline to Week 97 on 4SC for Participants in Sequence 1 Compared to the Natural History Control Group

Timepoint [12]

Baseline, Week 97

Secondary outcome [13]

Change From Baseline to Week 49 on FVC for Participants in Sequence 3 Compared to the Natural History Control Group

Timepoint [13]

Baseline, Week 49

Secondary outcome [14]

Change From Baseline to Week 97 on FVC for Participants in Sequence 1 Compared to the Natural History Control Group

Timepoint [14]

Baseline, Week 97

Secondary outcome [15]

Change From Baseline to Week 49 on NSAA for Participants in Sequence 3 Compared to the Natural History Control Group

Timepoint [15]

Baseline, Week 49

Secondary outcome [16]

Change From Baseline to Week 97 on NSAA for Participants in Sequence 1 Compared to the Natural History Control Group

Timepoint [16]

Baseline, Week 97

Secondary outcome [17]

Change From Baseline to Week 49 on 6MWD for Participants in Sequence 3 Compared to the Natural History Control Group

Timepoint [17]

Baseline, Week 49

Secondary outcome [18]

Change From Baseline to Week 97 on 6MWD for Participants in Sequence 1 Compared to the Natural History Control Group

Timepoint [18]

Baseline, Week 97

Secondary outcome [19]

Change From Baseline as Compared to Placebo on 4SC at Week 17 in Pre-specified Subsets

Timepoint [19]

Baseline, Week 17

Secondary outcome [20]

Change From Baseline as Compared to Placebo on 4SC at Week 33 in Pre-specified Subsets

Timepoint [20]

Baseline, Week 33

Secondary outcome [21]

Change From Baseline as Compared to Placebo on 4SC at Week 49 in Pre-specified Subsets

Timepoint [21]

Baseline, Week 49

Secondary outcome [22]

Change From Baseline as Compared to Placebo on FVC at Week 17 in Pre-specified Subsets

Timepoint [22]

Baseline, Week 17

Secondary outcome [23]

Change From Baseline as Compared to Placebo on FVC at Week 33 in Pre-specified Subsets

Timepoint [23]

Baseline, Week 33

Secondary outcome [24]

Change From Baseline as Compared to Placebo on FVC at Week 49 in Pre-specified Subsets

Timepoint [24]

Baseline, Week 49

Secondary outcome [25]

Change From Baseline as Compared to Placebo on NSAA at Week 17 in Pre-specified Subsets

Timepoint [25]

Baseline, Week 17

Secondary outcome [26]

Change From Baseline as Compared to Placebo on NSAA at Week 33 in Pre-specified Subsets

Timepoint [26]

Baseline, Week 33

Secondary outcome [27]

Change From Baseline as Compared to Placebo on NSAA at Week 49 in Pre-specified Subsets

Timepoint [27]

Baseline, Week 49

Secondary outcome [28]

Change From Baseline as Compared to Placebo on PUL Overall Scores at Week 17 in Pre-specified Subsets

Timepoint [28]

Baseline, Week 17

Secondary outcome [29]

Change From Baseline as Compared to Placebo on PUL Overall Score at Week 33 in Pre-specified Subsets

Timepoint [29]

Baseline, Week 33

Secondary outcome [30]

Change From Baseline as Compared to Placebo on PUL Overall Score at Week 49 in Pre-specified Subsets

Timepoint [30]

Baseline, Week 49

Secondary outcome [31]

Change From Baseline as Compared to Placebo on 6MWD at Week 17 in Pre-specified Subsets

Timepoint [31]

Baseline, Week 17

Secondary outcome [32]

Change From Baseline as Compared to Placebo on 6MWD at Week 33 in Pre-specified Subsets

Timepoint [32]

Baseline, Week 33

Secondary outcome [33]

Change From Baseline as Compared to Placebo on 6MWD at Week 49 in Pre-specified Subsets

Timepoint [33]

Baseline, Week 49

Secondary outcome [34]

Change From Baseline on Muscle Strength at Weeks 17, 33 and 49 in Pre-specified Subset (Baseline 4SC <3.5 Seconds)

Timepoint [34]

Baseline, Weeks 17, 33 and 49

Secondary outcome [35]

Change From Baseline on Muscle Strength at Weeks 17, 33 and 49 in Pre-specified Subset (Baseline 4SC >=3.5 Seconds and <=8 Seconds)

Timepoint [35]

Baseline, Weeks 17, 33 and 49

Secondary outcome [36]

Change From Baseline on Muscle Strength at Weeks 17, 33 and 49 in Pre-specified Subset (Baseline 4SC >8 Seconds)

Timepoint [36]

Baseline, Weeks 17, 33 and 49

Secondary outcome [37]

Percent Change From Baseline in Whole Thigh Muscle Volume as Compared to Placebo by Weeks 17, 33 and 49

Timepoint [37]

Baseline, Weeks 17, 33 and 49

Secondary outcome [38]

Percent Change From Baseline as Compared to Placebo in Whole Thigh Muscle Volume Index by Weeks 17, 33 and 49

Timepoint [38]

Baseline, Weeks 17, 33 and 49

Secondary outcome [39]

Change From Baseline in Whole Thigh Muscle Volume Through Week 97

Timepoint [39]

Baseline, Weeks 17, 33, 49 and 97

Secondary outcome [40]

Change From Baseline in Whole Thigh Muscle Volume Index Through Week 97

Timepoint [40]

Baseline, Weeks 17, 33, 49 and 97

Secondary outcome [41]

Concentration of Growth Differentiation Factor 8 (GDF-8) at Time 0 (Pre-dose),(C0(GDF-8) )

Timepoint [41]

Predose on Day 1 of Week 1

Secondary outcome [42]

Trough Serum Concentration of GDF-8 (Ctrough,(GDF-8)) for Participants Receiving Domagrozumab in Period 1

Timepoint [42]

Every 4 weeks on dosing day (at predose, end of 2-hour infusion and 6 hours since start of infusion) from Week 1 to Week 48

Secondary outcome [43]

Ctrough,(GDF-8) for Participants of Sequence 3 in Period 2

Timepoint [43]

Every 4 weeks on dosing day (predose, end of 2-hour infusion and 6 hours since start of infusion) from Week 49 to Week 96

Secondary outcome [44]

Trough (Pre-dose) Serum Concentration (Ctrough) of Domagrozumab

Timepoint [44]

Every 4 weeks on dosing day (predose, end of 2-hour infusion and 6 hours since start of infusion) from Week 1 to Week 96 for Sequence 1; from Week 1 to Week 48 for Sequence 2; from Week 49 to Week 96 for Sequence 3

Secondary outcome [45]

Maximum Serum Concentration (Cmax) of Domagrozumab

Timepoint [45]

Secondary outcome [46]

Time for Cmax (Tmax) of Domagrozumab

Timepoint [46]

Secondary outcome [47]

Terminal Half-life (t1/2) of Domagrozumab for Participants in Sequence 2 After the Last Dose of Domagrozumab

Timepoint [47]

At predose, end of 2-hour infusion and 6 hours since start of infusion at Week 45

Secondary outcome [48]

Area Under the Serum Concentration-time Curve Over the Dosing Interval Tau (AUCtau) of Domagrozumab

Timepoint [48]

At predose, end of 2-hour infusion,6 hours and 168 hours since start of infusion on Weeks 1, 13, 17, 29, 33 and 45

Secondary outcome [49]

Average Serum Concentration Over the Dosing Interval (Cav) of Domagrozumab

Timepoint [49]

At predose, end of 2-hour infusion, 6 hours and 168 hours since start of infusion on Weeks 1, 13, 17, 29, 33 and 45

Secondary outcome [50]

Clearance (CL) of Domagrozumab

Timepoint [50]

At predose, end of 2-hour infusion, 6 hours and 168 hours since start of infusion on Weeks 13, 29 and 45

Secondary outcome [51]

Volume of Distribution at Steady State (Vss) of Domagrozumab for Participants in Sequence 2 Required for Additional PK Assessment

Timepoint [51]

At predose, end of 2-hour infusion, 6 hours and 168 hours since start of infusion on Week 45

Secondary outcome [52]

Number of Participants With Anti-drug Antibodies (ADA) Development by Week 97

Timepoint [52]

Baseline, every 4 weeks from Week 5 to Week 97 visit or early termination

Eligibility

Key inclusion criteria

1. Ambulatory boys age 6 to <16 years old (at the time of randomization), diagnosed with DMD. Diagnosis must be confirmed in subject's medical history and by genetic testing obtained during routine clinical care for diagnostic purposes as reported from an appropriate regulated laboratory using a clinically validated genetic test (genetic testing is not provided by the sponsor).
2. Subjects who are able to perform the 4 stair climb in > or = 0.33 but < or =1.6 stairs/second.
3. Subjects must be receiving glucocorticosteroids for a minimum of 6 months prior to signing informed consent. There should be no significant change (>0.2 mg/kg) in dosage or dose regimen (not related to body weight change) for at least 3 months immediately prior to signing the informed consent and a reasonable expectation that dosage and dosing regimen will not change significantly for the duration of the study.
4. Adequate hepatic and renal function on screening laboratory assessments.
5. No underlying disposition for iron accumulation on screening laboratory assessments.
6. Iron content estimate on the screening liver MRI is within the normal range.

Minimum age

6 Years

Maximum age

15 Years

Sex

Males

Can healthy volunteers participate?

Key exclusion criteria

1. Subjects with known cognitive impairment or behavioral issues that would impede the ability to follow instructions.
2. History of major surgical procedure within 6 weeks of signing the informed consent or planned surgery during the study.
3. Any injury which may impact functional testing. Previous injuries must be fully healed prior to consenting. Prior lower limb fractures must be fully healed and at least 3 months from injury date.
4. Presence or history of other musculoskeletal or neurologic disease or somatic disorder not related to DMD including pulmonary and cardiac disease.
5. Compromised cardiac function (left ventricular ejection fraction <55% as determined on a screening cardiac MRI or echocardiogram). Subjects may be receiving ACE (angiotensin converting enzyme) inhibitors or beta blockers, ARB (angiotensin II receptor antagonist) or aldosterone blocker/thiazide diuretic; however they must have initiated treatment more than 3 months prior to screening to ensure stable therapy.
6. Evidence or history of clinically significant hematological, renal, endocrine, pulmonary, gastrointestinal, cardiovascular (including uncontrolled hypertension), hepatic, neurologic, or allergic disease (including drug allergies, but excluding untreated, asymptomatic, seasonal allergies at time of dosing).
7. Documented history of iron overload including hemochromatosis, beta thalassemia major, beta thalassemia intermedia or hemolytic anemia.
8. Unwilling or unable (eg, metal implants, requires sedation) to undergo examination with closed MRI without sedation.
9. Participation in other studies involving investigational drug(s) for a minimum of 30 days or within 5 half lives (whichever is longer) prior to signing the informed consent and/or during study participation.
10. Current or prior treatment with anti-myostatin, exon skipping, nonsense mutation targeted therapies ever or more than 30 days of treatment with utrophin modifiers and treatment with utrophin modifiers within 30 days prior ot signing the informed consent and/or during study participation.
11. Current or prior treatment within the past 3 months with androgens or human growth hormone.
12. Current treatment with immunosuppressant therapies (other than glucocorticoid steroids), aminoglycosides (eg, gentamicin), multi vitamins with iron and iron supplements and other investigational therapies (including idebenone).

Study design

Purpose of the study

Treatment

Allocation to intervention

Randomised controlled 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

Blinded (masking used)

Who is / are masked / blinded?

The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data

Intervention assignment

Other design features

Phase

Phase 2

Type of endpoint/s

Statistical methods / analysis

Recruitment

Recruitment status

Stopped early

Data analysis

Reason for early stopping/withdrawal

Other reasons

Date of first participant enrolment

Anticipated

Actual

24/11/2014

Date of last participant enrolment

Anticipated

Actual

Date of last data collection

Anticipated

Actual

23/11/2018

Sample size

Target

Accrual to date

Final

121

Recruitment in Australia

Recruitment state(s)

QLD

Recruitment hospital [1]

Lady Cilento Children's Hospital - South Brisbane

Recruitment postcode(s) [1]

4101 - South Brisbane

Recruitment outside Australia

Country [1]

United States of America

State/province [1]

California

Country [2]

United States of America

State/province [2]

Colorado

Country [3]

United States of America

State/province [3]

Florida

Country [4]

United States of America

State/province [4]

Illinois

Country [5]

United States of America

State/province [5]

Iowa

Country [6]

United States of America

State/province [6]

Kansas

Country [7]

United States of America

State/province [7]

Maryland

Country [8]

United States of America

State/province [8]

Massachusetts

Country [9]

United States of America

State/province [9]

Minnesota

Country [10]

United States of America

State/province [10]

Missouri

Country [11]

United States of America

State/province [11]

North Carolina

Country [12]

United States of America

State/province [12]

Ohio

Country [13]

United States of America

State/province [13]

Pennsylvania

Country [14]

United States of America

State/province [14]

Utah

Country [15]

Bulgaria

State/province [15]

Sofia

Country [16]

Canada

State/province [16]

Alberta

Country [17]

Canada

State/province [17]

British Columbia

Country [18]

Canada

State/province [18]

Ontario

Country [19]

Canada

State/province [19]

Quebec

Country [20]

Italy

State/province [20]

Genova

Country [21]

Italy

State/province [21]

Rome

Country [22]

Japan

State/province [22]

Hyogo

Country [23]

Japan

State/province [23]

Tokyo

Country [24]

Poland

State/province [24]

Warszawa

Country [25]

United Kingdom

State/province [25]

Liverpool

Country [26]

United Kingdom

State/province [26]

London

Country [27]

United Kingdom

State/province [27]

Newcastle upon Tyne

Country [28]

United Kingdom

State/province [28]

Newcastle-upon-Tyne

Funding & Sponsors

Primary sponsor type

Commercial sector/industry

Name

Pfizer

Address

Country

Ethics approval

Ethics application status

Summary

Brief summary

This is a Phase 2 randomized, 2-period, double-blind, placebo-controlled, multiple ascending dose study to evaluate the safety, efficacy, PK and PD of PF-06252616 administered to ambulatory boys diagnosed with Duchenne Muscular Dystrophy. Three intravenous (IV) dose levels will be investigated in a within subject dose escalating fashion. Subjects will be randomly assigned to 1 of 3 sequence groups for approximately 96 weeks (2 periods of 48 weeks each). In period 1, two of the sequence groups will receive PF-06252616 and one sequence group will receive placebo. In period 2, the placebo group will switch to PF-06252616 and the two remaining sequence groups will either receive placebo or PF-06252616. Efficacy will be based on an observed mean change from baseline on function (4 stair climb) of PF-06252616 as compared to the placebo at the end of period 1. Period 2 provides an opportunity to evaluate PK. Subjects will receive monthly IV infused doses of either PF-06252616 or placebo and will undergo safety evaluations (Laboratory, cardiac monitoring, physical exams, x-ray, MRI), functional evaluations (pulmonary function testing, 4 stair climb, range of motion, strength testing, Northstar Ambulatory Assessment, upper limb functional testing and the six minute walk test), pharmacokinetic testing and pharmacodynamic testing to evaluate changes in muscle volume (MRI).

Trial website

https://clinicaltrials.gov/study/NCT02310763

Trial related presentations / publications

Sherlock SP, Palmer J, Wagner KR, Abdel-Hamid HZ, Tian C, Mah JK, Muntoni F, Guglieri M, Butterfield RJ, Charnas L, Marraffino S. Dual-energy X-ray absorptiometry measures of lean body mass as a biomarker for progression in boys with Duchenne muscular dystrophy. Sci Rep. 2022 Nov 5;12(1):18762. doi: 10.1038/s41598-022-23072-5.
Wojciechowski J, Purohit VS, Harnisch LO, Dua P, Tan B, Nicholas T. Population PK and PD Analysis of Domagrozumab in Pediatric Patients with Duchenne Muscular Dystrophy. Clin Pharmacol Ther. 2022 Dec;112(6):1291-1302. doi: 10.1002/cpt.2747. Epub 2022 Oct 4.
Muntoni F, Guglieri M, Mah JK, Wagner KR, Brandsema JF, Butterfield RJ, McDonald CM, Mayhew AG, Palmer JP, Marraffino S, Charnas L, Mercuri E. Novel approaches to analysis of the North Star Ambulatory Assessment (NSAA) in Duchenne muscular dystrophy (DMD): Observations from a phase 2 trial. PLoS One. 2022 Aug 23;17(8):e0272858. doi: 10.1371/journal.pone.0272858. eCollection 2022.
Sherlock SP, Palmer J, Wagner KR, Abdel-Hamid HZ, Bertini E, Tian C, Mah JK, Kostera-Pruszczyk A, Muntoni F, Guglieri M, Brandsema JF, Mercuri E, Butterfield RJ, McDonald CM, Charnas L, Marraffino S. Quantitative magnetic resonance imaging measures as biomarkers of disease progression in boys with Duchenne muscular dystrophy: a phase 2 trial of domagrozumab. J Neurol. 2022 Aug;269(8):4421-4435. doi: 10.1007/s00415-022-11084-0. Epub 2022 Apr 8.
Wagner KR, Guglieri M, Ramaiah SK, Charnas L, Marraffino S, Binks M, Vaidya VS, Palmer J, Goldstein R, Muntoni F. Safety and disease monitoring biomarkers in Duchenne muscular dystrophy: results from a Phase II trial. Biomark Med. 2021 Oct;15(15):1389-1396. doi: 10.2217/bmm-2021-0222. Epub 2021 Sep 17.
Sherlock SP, Zhang Y, Binks M, Marraffino S. Quantitative muscle MRI biomarkers in Duchenne muscular dystrophy: cross-sectional correlations with age and functional tests. Biomark Med. 2021 Jun;15(10):761-773. doi: 10.2217/bmm-2020-0801. Epub 2021 Jun 22.
Wagner KR, Abdel-Hamid HZ, Mah JK, Campbell C, Guglieri M, Muntoni F, Takeshima Y, McDonald CM, Kostera-Pruszczyk A, Karachunski P, Butterfield RJ, Mercuri E, Fiorillo C, Bertini ES, Tian C, Statland J, Sadosky AB, Purohit VS, Sherlock SP, Palmer JP, Binks M, Charnas L, Marraffino S, Wong BL. Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophy. Neuromuscul Disord. 2020 Jun;30(6):492-502. doi: 10.1016/j.nmd.2020.05.002. Epub 2020 May 19. Erratum In: Neuromuscul Disord. 2021 Feb;31(2):167-168. doi: 10.1016/j.nmd.2021.01.001.

Public notes

Contacts

Principal investigator

Name

Pfizer CT.gov Call Center

Address

Pfizer

Country

Phone

Fax

Contact person for public queries

Name

Address

Country

Phone

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?

Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

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: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests

What supporting documents are/will be available?

Type	Other Details	Attachment
Study protocol		https://cdn.clinicaltrials.gov/large-docs/63/NCT02310763/Prot_000.pdf
Statistical analysis plan		https://cdn.clinicaltrials.gov/large-docs/63/NCT02310763/SAP_001.pdf
Study protocol		https://cdn.clinicaltrials.gov/large-docs/63/NCT02310763/Prot_000.pdf
Statistical analysis plan		https://cdn.clinicaltrials.gov/large-docs/63/NCT02310763/SAP_001.pdf

Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results are available at https://clinicaltrials.gov/study/NCT02310763

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For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02310763