An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02907619
• Date of registration: 14/09/2016
• Prospective Registration: Yes
• Primary sponsor: Pfizer
• Public title: An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy
• Scientific title: A MULTICENTER, OPEN-LABEL EXTENSION STUDY TO EVALUATE THE LONG TERM SAFETY OF PF-06252616 IN BOYS WITH DUCHENNE MUSCULAR DYSTROPHY
• Date of first enrolment: October 13, 2016
• Target sample size: 59
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT02907619
• Study type: Interventional
• Study design: Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Canada, Italy, Japan, United Kingdom, United States

Contacts

• Name: Pfizer CT.gov Call Center
• Affiliation: Pfizer

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Subjects with Duchenne muscular dystrophy who enrolled and completed study B5161002.

2. Signed and dated informed consent document (ICD) indicating that the subject's parent
or legal guardian/caregiver has been informed of all pertinent aspects of the study.

3. Subjects and their legal guardians/caregivers who are willing and able to comply with
scheduled visits, treatment plan, laboratory tests, and other study procedures.

4. Subject have;

1. Adequate hepatic function on screening laboratory assessments

2. GLDH less than 20 units/liter (2 x upper limit of normal [ULN])

3. Iron content estimate on the liver MRI within the normal range.

Exclusion Criteria:

1. Unwilling or unable (eg, metal implants) to undergo examination with closed MRI.

2. All male subjects who are able to father children and are sexually active and at risk
for impregnating a female partner, who are unwilling or unable to use a highly
effective method of contraception. In addition, all sexually active male subjects who
are unwilling or unable to prevent potential transfer of and exposure to drug through
semen to their partners by using a condom consistently and correctly. .

3. Subjects who are investigational site staff members directly involved in the conduct
of the study and their family members, site staff members otherwise supervised by the
Investigator, or subjects who are related to Pfizer employees directly involved in the
conduct of the study.

4. Other severe acute or chronic medical or psychiatric condition or laboratory
abnormality that may increase the risk associated with study participation.

5. Participation in other studies involving investigational drug(s), with the exception
of B5161002.

6. History of allergic or anaphylactic reaction to a therapeutic or diagnostic protein or
additives of this investigational product.



Age minimum: 6 Years
Age maximum: 18 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Duchenne Muscular Dystrophy

Intervention(s)

Biological: PF-06252616

Primary Outcome(s)

Number of Participants With Laboratory Test Abnormalities (Without Regard to B5161004 Baseline Abnormality) - Electrolytes [Time Frame: 2 Years]

Number of Participants With Significant Results of Physical Examinations Including Nose and Throat Mucosal Examinations [Time Frame: 2 Years]

Number of Participants With Laboratory Test Abnormalities (Without Regard to B5161004 Baseline Abnormality) - Hematology [Time Frame: 2 Years]

Number of Participants With Laboratory Test Abnormalities (Without Regard to B5161004 Baseline Abnormality) - Liver Function [Time Frame: 2 Years]

Number of Participants With Post-Baseline Vital Signs Data Meeting Categorical Summarization Criteria - B5161004 Baseline [Time Frame: 2 Years]

Number of Participants With Iron Accumulation Data Meeting Categorical Summarization Criteria [Time Frame: Screening and Week 49.]

Number of Participants With Laboratory Test Abnormalities (Without Regard to B5161004 Baseline Abnormality) - Fecal Blood [Time Frame: 2 Years]

Number of Participants With Laboratory Test Abnormalities (Without Regard to B5161004 Baseline Abnormality) - Urinalysis [Time Frame: 2 Years]

Number of Participants With Post-Baseline ECG Data Meeting Categorical Summarization Criteria - B5161004 Baseline [Time Frame: 2 Years]

Change From Baseline in Left Ventricular Ejection Fraction (LVEF) by Cardiac MRI - Overall Baseline [Time Frame: Baseline, Weeks 49, 97, 146.]

Number of Participants Who Discontinued From the Study Due to TEAEs [Time Frame: 2 Years]

Number of Participants With Dose Reduced or Temporary Discontinuation Due to AEs [Time Frame: 2 Years]

Change From Baseline in Left Ventricular Ejection Fraction (LVEF) by Echocardiogram - B5161004 Baseline [Time Frame: Baseline, Week 49.]

Summary of Pubertal Development by Tanner Stage [Time Frame: Screening, Baseline, Week 49.]

Bone Age to Chronological Age Ratio [Time Frame: Baseline and Week 49.]

Number of Participants With Laboratory Test Abnormalities (Without Regard to B5161004 Baseline Abnormality) - Clinical Chemistry [Time Frame: 2 Years]

Change From Baseline in Left Ventricular Ejection Fraction (LVEF) by Cardiac MRI - B5161004 Baseline [Time Frame: Baseline and Week 49.]

Number of Participants With Post-Baseline Vital Signs Data Meeting Categorical Summarization Criteria - Overall Baseline [Time Frame: 2 Years]

Change From Baseline in Left Ventricular Ejection Fraction (LVEF) by Echocardiogram - Overall Baseline [Time Frame: Baseline, Weeks 49, 97, 146.]

Number of Participants With Severe Treatment-Emergent Adverse Events (TEAEs) [Time Frame: 2 Years]

Number of Participants With Suicidal Ideation or Suicidal Behavior [Time Frame: 2 Years]

Whole Body and Spine DXA: Bone Mineral Density Z-Score, Height Adjusted Over Time [Time Frame: Screening (Week 97 visit within parent study B5161002) and Week 49]

Number of Participants With Data of Serum Ferritin, Serum Iron and % Transferrin Saturation Meeting Categorical Summarization Criteria - B5161004 Baseline [Time Frame: Baseline, Weeks 13, 25, 37, 49, 61, 73 and 85.]

Number of Participants With Laboratory Test Abnormalities (Without Regard to B5161004 Baseline Abnormality) - Hormones [Time Frame: 2 Years]

Number of Participants With Laboratory Test Abnormalities (Without Regard to B5161004 Baseline Abnormality) - Renal Function [Time Frame: 2 Years]

Number of Participants With Post-Baseline ECG Data Meeting Categorical Summarization Criteria - Overall Baseline [Time Frame: 2 Years]

Summary of Testicular Volume [Time Frame: Screening, Baseline, Week 49.]

Number of Participants With Laboratory Test Abnormalities (Without Regard to B5161004 Baseline Abnormality) - Coagulation [Time Frame: 2 Years]

Secondary Outcome(s)

Change From Baseline on the Northstar Ambulatory Assessment (NSAA) Score - B5161004 Baseline [Time Frame: Baseline, Weeks 13, 25, 49, 73.]

Change From Baseline on the 6MWD - Overall Baseline [Time Frame: Baseline,Weeks 9,17,25,33,41,49,57,65,73,81,89,97,110,122,146,170.]

Change From Baseline on the Ankle Range of Motion (ROM) - B5161004 Baseline [Time Frame: Baseline, Weeks 13, 25, 49 and 73.]

Change From Baseline on the Forced Expiratory Volume in One Second (FEV1) - B5161004 Baseline [Time Frame: Baseline, Weeks 13, 25, 49 and 73.]

Change From Baseline on the NSAA - Time to Stand From Supine - B5161004 Baseline [Time Frame: Baseline, Weeks 13, 25, 49, 73.]

Change From Baseline on the NSAA - Time to Complete 10 m Run/Walk - Overall Baseline [Time Frame: Baseline, Weeks 9,17,25,33,41,49,57,65,73,81,89,97,110,122,146,170.]

Change From Baseline on the NSAA Score - Overall Baseline [Time Frame: Baseline, Weeks 9,17,25,33,41,49,57,65,73,81,89,97,110,122,146,170.]

Change From Baseline on the Peak Expiratory Flow Rate (PEFR)- B5161004 Baseline [Time Frame: Baseline, Weeks 13, 25, 49 and 73.]

Change From Baseline on the 4SC - Overall Baseline [Time Frame: Baseline, Weeks 9,17,25,33,41,49,57,65,73,81,89,97,110,122,146,170.]

Change From Baseline on the FVC - Overall Baseline [Time Frame: Baseline, Weeks 9,17,25,33,41,49,57,65,73,81,89,97,110,122,146,170.]

Change From Baseline on the PUL Overall Score - Overall Baseline [Time Frame: Baseline, Weeks 9,17,25,33,41,49,57,65,73,81,89,97,110,122,146,170.]

Change From Baseline on the Myometry Based Muscle Strength - Overall Baseline [Time Frame: Baseline,Weeks 9,17,25,33,41,49,57,65,73,81,89,97,110,122,146,170.]

Change From Baseline on the Performance of Upper Limb (PUL) Overall Score - B5161004 Baseline [Time Frame: Baseline, Weeks 13, 25, 49 and 73.]

Change From Baseline on the NSAA - Time to Complete 10 m Run/Walk - B5161004 Baseline [Time Frame: Baseline, Weeks 13, 25, 49, 73.]

Change From Baseline on the Six Minute Walk Distance (6MWD) - B5161004 Baseline [Time Frame: Baseline, Weeks 13, 25, 49 and 73.]

Number of Participants With Anti-drug Antibodies (ADA) Development [Time Frame: Weeks 1, 25, 49, 73 and Early Termination]

Change From Baseline on the 4 Stair Climb (4SC) - B5161004 Baseline [Time Frame: Baseline, Weeks 13, 25, 49, 73.]

Change From Baseline on the Ankle ROM - Overall Baseline [Time Frame: Baseline, Weeks 9,17,25,33,41,49,57,65,73,81,89,97,110,122,146,170.]

Change From Baseline on the Forced Vital Capacity (FVC) - B5161004 Baseline [Time Frame: Baseline, Weeks 13, 25, 49 and 73.]

Change From Baseline on the Myometry Based Muscle Strength - B5161004 Baseline [Time Frame: Baseline, Weeks 13, 25, 49, 73.]

Change From Baseline on the NSAA - Time to Stand From Supine - Overall Baseline [Time Frame: Baseline, Weeks 9,17,25,33,41,49,57,65,73,81,89,97,110,122,146,170.]

Serum PF-06252616 (Domagrozumab) Concentration Versus Time Summary [Time Frame: Weeks 1, 25, 49 and 73]

Secondary ID(s)

2016-001615-21

B5161004

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 25/11/2019
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02907619