A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (ENVISION)

Conditions

Duchenne Muscular Dystrophy

Brief summary

Part 1: Change From Baseline in the Total Score of Performance of Upper Limb (PUL) (Version 2.0) at Week 72

Detailed description

Part 1: Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 72, Part 1: Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 72, Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot, Part 1: Change From Baseline in Patient-Reported Outcomes Measurement Information (PROMIS) Score in Upper Extremity Function to Week 72, Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE), Part 1 (For Cohort 2 Only): Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 72, Part 1: Change From Baseline in Global Circumferential Strain as Measured by Cardiac MRI at Week 72, Part 1: Change From Baseline in PUL (Version 2.0) Middle Domain Score at Week 72.

Related papers

No related papers found yet.

Interventions

DRUGSaline

DRUG0.9% sodium chloride solution IV infusion marketed authorized will be used as the placebo and will be supplied by the site. the SmPC previously authorized are Country specific. for Belgium

DRUGSpain and Sweden per local requirements under CTDs no SmPCs were submitted.

DRUGDelandistrogene moxeparvovec-rokl

Eligibility

Sex/Gender

Male

Age

0 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
Part 1: Change From Baseline in the Total Score of Performance of Upper Limb (PUL) (Version 2.0) at Week 72	—

Secondary

Measure	Time frame
Part 1: Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 72, Part 1: Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 72, Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot, Part 1: Change From Baseline in Patient-Reported Outcomes Measurement Information (PROMIS) Score in Upper Extremity Function to Week 72, Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE), Part 1 (For Cohort 2 Only): Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 72, Part 1: Change From Baseline in Global Circumferential Strain as Measured by Cardiac MRI at Week 72, Part 1: Change From Baseline in PUL (Version 2.0) Middle Domain Score at Week 72.	—

Measure

Time frame

Part 1: Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 72, Part 1: Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 72, Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot, Part 1: Change From Baseline in Patient-Reported Outcomes Measurement Information (PROMIS) Score in Upper Extremity Function to Week 72, Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE), Part 1 (For Cohort 2 Only): Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 72, Part 1: Change From Baseline in Global Circumferential Strain as Measured by Cardiac MRI at Week 72, Part 1: Change From Baseline in PUL (Version 2.0) Middle Domain Score at Week 72.

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Countries

Belgium, France, Germany, Italy, Spain, Sweden

Outcome results

None listed