E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Duchenne Muscular Dystrophy |
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E.1.1.1 | Medical condition in easily understood language |
Duchenne muscular dystrophy is a rare, serious, life-threatening, X-linked recessive, degenerative neuromuscular disease caused by mutations in the dystrophin gene. |
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E.1.1.2 | Therapeutic area | Diseases [C] - Musculoskeletal Diseases [C05] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10052655 |
E.1.2 | Term | Duchenne muscular dystrophy gene carrier |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The primary objectives of this study are the assessments of the safety and efficacy of intravenous administration of SRP-9001 in DMD participants. |
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E.2.2 | Secondary objectives of the trial |
The secondary objectives include the effects of SRP-9001 on physical functional assessments and micro-dystrophin expression as measured by immunofluorescence (IF) and IF percent dystrophin positive fibers (PDPF) of biopsied muscle tissue. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Established clinical diagnosis of DMD and documented dystrophin gene mutation of DMD phenotype. • Indication of symptomatic muscular dystrophy by protocol-specified criteria. • Ability to cooperate with motor assessment testing. • Stable dose equivalent of oral corticosteroids for at least 12 weeks. • A frameshift mutation contained between exons 18 and 58 (inclusive).
Other inclusion criteria apply. |
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E.4 | Principal exclusion criteria |
• Impaired cardiovascular function on echocardiogram. • Prior or ongoing medical condition on physical examination, electrocardiogram, or laboratory findings that could adversely affect participant safety, compromise completion of follow-up, or impair assessment of study results. • Exposure to another investigational drug or exon skipping medication within 6 months of screening. • Exposure to an investigational or commercial gene therapy product. • Abnormal liver or renal function by protocol-specified criteria.
Other exclusion criteria apply. |
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E.5 End points |
E.5.1 | Primary end point(s) |
• Change From Baseline in Quantity of Micro-dystrophin Protein Expression as Measured by Western Blot • Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
• Western Blot: Baseline up to Week 12 (Part 1) • NSAA Total Score: Baseline up to Week 48 (Part 1) |
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E.5.2 | Secondary end point(s) |
• Change From Baseline in Time to Rise From the Floor • Change From Baseline in Time to Ascend 4 Steps • Change From Baseline in Time of 10 Meter Timed Test • Change From Baseline in Time of 100 Meter Timed Test • Change From Baseline in Quantity of Micro-dystrophin Expression Measured by IF Fiber Intensity • Change From Baseline in Quantity of Micro-dystrophin Expression Measured by IF PDPF |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
• Rise From the Floor: Baseline up to Week 48 (Part 1) • Ascend 4 Steps: Baseline up to Week 48 (Part 1) • 10 Meter Timed Test: Baseline up to Week 48 (Part 1) • 100 Meter Timed Test: Baseline up to Week 48 (Part 1) • IF Fiber Intensity: Baseline up to Week 12 (Part 1) • IF PDPF: Baseline up to Week 12 (Part 1) |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
Parallel up to the measurement of the primary outcomes at Week 48. |
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E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
E.8.3 |
Will this trial be conducted at a single site globally?
| No |
E.8.4 | Will this trial be conducted at multiple sites globally? | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.2 | Trial being conducted completely outside of the EEA | Yes |
E.8.6.3 | Specify the countries outside of the EEA in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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Last participant last visit |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.2 | In all countries concerned by the trial years | 5 |