A Phase 3 Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy

JRCT ID: jRCT2051210011

Registered date:22/04/2021

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: Duchenne Muscular Dystrophy
• Date of first enrollment: 21/05/2021
• Target sample size: 99
• Countries of recruitment: Israel,Japan,Italy,Japan,Spain,Japan,Belgium,Japan,Canada,Japan,France,Japan,Korea,Japan,Russian Federation,Japan,Switzerland,Japan,Taiwan,Japan,United States,Japan,United Kingdom,Japan
• Study type: Interventional

Intervention(s)

Genetic: PF-06939926
PF-06939926 will be administered as a single IV infusion at Year 1 for Cohort 1.

Other: Placebo
Placebo will be administered as a single IV infusion at Year 2 for Cohort 1.

Other: Placebo
Placebo will be administered as a single IV infusion at Year 1 for Cohort 2.

Genetic: PF-06939926
PF-06939926 will be administered as a single IV infusion at Year 2 for Cohort 2

Outcome(s)

Primary Outcome

Primary Outcome Measures:
1.Change from Baseline in North Star Ambulatory Assessment (NSAA) [ Time Frame: Week 52 ]
The NSAA is a 17-item test that measures gross motor function in children with Duchenne.

Secondary Outcome

Secondary Outcome Measures:
1.Change from Baseline in mini-dystrophin expression level in muscle [ Time Frame: Week 52 ]
Mini-dystrophin expression level from a muscle biopsy will be assessed by liquid chromatography mass spectrometry (LC-MS).

2.Change from Baseline in distribution of mini-dystrophin expression in the muscle [ Time Frame: Week 52 ]
Mini-dystrophin distribution from a muscle biopsy will be assessed by immunofluorescence.

3.Change from Baseline in serum creatine kinase (CK) [ Time Frame: Week 52 ]
Changes in the circulating levels of CK.

4.Number of skills gained based on the individual items of the NSAA. [ Time Frame: Week 52 ]
To count the skills that each child gained, based on the individual items of the NSAA.

5.Number of skills improved or maintained based on the individual items of the NSAA [ Time Frame: Week 52 ]
To count the skills that each child improved or maintained, based on the individual items of the NSAA.

6.Change from Baseline in the 10-meter run/walk test velocity [ Time Frame: Week 52 ]
Velocity is calculated based on the time that it takes to complete the 10-meter run/walk test.

7.Change from Baseline in the rise from floor velocity [ Time Frame: Week 52 ]
Velocity is calculated based on the time that it takes to the rise from floor.

8.Change from Baseline in the Modified Pediatric Outcomes Data Collection Instrument (PODCI): Transfer and Basic Mobility Core Scale [ Time Frame: Week 52 ]
The PODCI contains a list of questions to assess how each caregiver/child evaluates the child's ability to to walk, stand, and perform activities of daily living.

9.Change from Baseline in the Modified Pediatric Outcomes Data Collection Instrucment (PODCI): Sports and Physical Functioning Core Scale [ Time Frame: Week 52 ]
The PODCI contains a list of questions to assess how each caregiver/child evaluates the child's ability to perform recreational activities.

Key inclusion & exclusion criteria

• Age minimum: >= 4age old
• Age maximum: <= 7age old
• Gender: Male

Include criteria

Key inclusion criteria:
1.Confirmed diagnosis of Duchenne muscular dystrophy by prior genetic testing
2.Receiving a stable daily dose (at least 0.5 mg/kg/day prednisone or prednisolone, or at least 0.75 mg/kg/day deflazacort) for at least 3 months prior to Screening
3.Ambulatory, as assessed by protocol-specified criteria

Exclude criteria

Key exclusion criteria:
1.Positive test performed by Pfizer for neutralizing antibodies to AAV9
2.Any treatment designed to increase dystrophin expression within 6 months prior to screening (e.g., Translarna, EXONDYS 51, VYONDYS 53)
3.Any prior treatment with gene therapy
4.Any non-healed injury that may impact functional testing (eg NSAA)
5.Abnormality in specified laboratory tests, including blood counts, liver and kidney function

Related Information

• Primary Sponsor: Kawai Norisuke
• Secondary ID(s): NCT04281485