Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy

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

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2019-003374-91-FR

Enrollment

120

Registered

2021-09-30

Start date

2022-09-12

Completion date

Unknown

Last updated

2025-01-27

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Duchenne Muscular Dystrophy MedDRA version: 20.1 Level: PT Classification code 10052655 Term: Duchenne muscular dystrophy gene carrier System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Product Code: SRP-9001 Pharmaceutical Form: Solution for infusion INN or Proposed INN: Not available CAS Number: 2305040-16-6 Current Sponsor code: SRP-9001 Other descriptive name: ADENO-ASSOCIATED VI

Eligibility

Sex/Gender

Male

Inclusion criteria

Inclusion criteria: A subject must meet all of the following criteria to be eligible to participate in this study: 1. Is male at birth, ambulatory, and = 4 to 16 and =65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: A subject who meets any of the following criteria will be excluded from this study: 1. Has left ventricular ejection fraction 2× upper limit of normal (ULN) • Glutamate dehydrogenase (GLDH) > 15 U/L • Total bilirubin > ULN. Note; elevations in total bilirubin confirmed to be due to Gilbert’s syndrome are not exclusionary. • White blood cell count > 18,500 per µl • Platelets = 150,000 per µL 10. Family does not want to disclose subject’s study participation with general practitioner/primary care physician and other medical providers. 11. In the opinion of the Investigator, the subject is not likely to be compliant with the study protocol.

Design outcomes

Primary

Measure	Time frame
Main Objective: To evaluate the effect of SRP-9001 on physical function as assessed by the North Star Ambulatory Assessment (NSAA) score ;Secondary Objective: • To evaluate the effect of SRP-9001 on physical function as assessed by: o Number of skills gained or improved on the NSAA • To evaluate micro-dystrophin expression from SRP-9001 at 12 weeks (Part 1) as measured by Western blot of biopsied muscle tissue • To evaluate the effect of SRP-9001 on timed function tests as assessed by measuring: o Time to rise from the floor o 100-meter walk/run (100MWR) o Time to ascend 4 steps o 10-meter walk/run (10MWR) • To evaluate the effect of SRP-9001 on stride velocity 95th centile (SV95C) as measured by a wearable device • To evaluate subject (parent/caregiver proxy) reported Mobility and Upper Extremity Function using the Patient Reported Outcomes Measurement Information System (PROMIS®) tool • To evaluate the safety of SRP-9001 ;Primary end point(s): • Change in NSAA total score from Baseline to Week 52 (Part 1) ;Timepoint(s) of evaluation of this end point: From Baseline to Week 52 (Part 1)	—

Measure

Time frame

Main Objective: To evaluate the effect of SRP-9001 on physical function as assessed by the North Star Ambulatory Assessment (NSAA) score ;Secondary Objective: • To evaluate the effect of SRP-9001 on physical function as assessed by: o Number of skills gained or improved on the NSAA • To evaluate micro-dystrophin expression from SRP-9001 at 12 weeks (Part 1) as measured by Western blot of biopsied muscle tissue • To evaluate the effect of SRP-9001 on timed function tests as assessed by measuring: o Time to rise from the floor o 100-meter walk/run (100MWR) o Time to ascend 4 steps o 10-meter walk/run (10MWR) • To evaluate the effect of SRP-9001 on stride velocity 95th centile (SV95C) as measured by a wearable device • To evaluate subject (parent/caregiver proxy) reported Mobility and Upper Extremity Function using the Patient Reported Outcomes Measurement Information System (PROMIS®) tool • To evaluate the safety of SRP-9001 ;Primary end point(s): • Change in NSAA total score from Baseline to Week 52 (Part 1) ;Timepoint(s) of evaluation of this end point: From Baseline to Week 52 (Part 1)

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Secondary

Measure	Time frame
Secondary end point(s): • Number of skills gained or improved at Week 52 (Part 1) as measured by the NSAA • Quantity of micro-dystrophin protein expression at Week 12 (Part 1) as measured by Western Blot • Change in time to rise from the floor from Baseline to Week 52 (Part 1) • Change in time of 100MWR from Baseline to Week 52 (Part 1) • Change in time to ascend 4 steps from Baseline to Week 52 (Part 1) • Change in time of 10MWR from Baseline to Week 52 (Part 1) • Change in SV95C from Baseline to Week 52 (Part 1) • Change in PROMIS score per domain from Baseline over 52 weeks (Part 1) • Incidence of treatment-emergent adverse events • Incidence of serious adverse events • Incidence of adverse events of special interest • Clinically significant changes in vital signs and physical examination findings • Clinically significant changes in safety laboratory assessments, electrocardiograms (ECGs), and echocardiograms (ECHOs) ;Timepoint(s) of evaluation of this end point: • Number of skills gained or improved at Week 52 (Part 1) as measured by the NSAA • Quantity of micro-dystrophin protein expression at Week 12 (Part 1) as measured by Western Blot • Change in time to rise from the floor from Baseline to Week 52 (Part 1) • Change in time of 100MWR from Baseline to Week 52 (Part 1) • Change in time to ascend 4 steps from Baseline to Week 52 (Part 1) • Change in time of 10MWR from Baseline to Week 52 (Part 1) • Change in SV95C from Baseline to Week 52 (Part 1) • Change in PROMIS score per domain from Baseline over 52 weeks (Part 1) • For other secondary endpoints, full duration of the study	—

Measure

Time frame

Secondary end point(s): • Number of skills gained or improved at Week 52 (Part 1) as measured by the NSAA • Quantity of micro-dystrophin protein expression at Week 12 (Part 1) as measured by Western Blot • Change in time to rise from the floor from Baseline to Week 52 (Part 1) • Change in time of 100MWR from Baseline to Week 52 (Part 1) • Change in time to ascend 4 steps from Baseline to Week 52 (Part 1) • Change in time of 10MWR from Baseline to Week 52 (Part 1) • Change in SV95C from Baseline to Week 52 (Part 1) • Change in PROMIS score per domain from Baseline over 52 weeks (Part 1) • Incidence of treatment-emergent adverse events • Incidence of serious adverse events • Incidence of adverse events of special interest • Clinically significant changes in vital signs and physical examination findings • Clinically significant changes in safety laboratory assessments, electrocardiograms (ECGs), and echocardiograms (ECHOs) ;Timepoint(s) of evaluation of this end point: • Number of skills gained or improved at Week 52 (Part 1) as measured by the NSAA • Quantity of micro-dystrophin protein expression at Week 12 (Part 1) as measured by Western Blot • Change in time to rise from the floor from Baseline to Week 52 (Part 1) • Change in time of 100MWR from Baseline to Week 52 (Part 1) • Change in time to ascend 4 steps from Baseline to Week 52 (Part 1) • Change in time of 10MWR from Baseline to Week 52 (Part 1) • Change in SV95C from Baseline to Week 52 (Part 1) • Change in PROMIS score per domain from Baseline over 52 weeks (Part 1) • For other secondary endpoints, full duration of the study

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Countries

Belgium, France, Germany, Hong Kong, Italy, Japan, Spain, Taiwan, United Kingdom, United States

Contacts

Public ContactSarepta Clinical Trial Inquiries

Sarepta Therapeutics, Inc.

sareptally@sarepta.com

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026