Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy
- Conditions
- Duchenne Muscular DystrophyMedDRA version: 20.0Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]MedDRA version: 20.1Level: PTClassification code 10052655Term: Duchenne muscular dystrophy gene carrierSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
- Registration Number
- EUCTR2019-003374-91-BE
- Lead Sponsor
- Sarepta Therapeutics, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- Male
- Target Recruitment
- 120
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 < 8 years of age at the time of randomization.
2. Has a definitive diagnosis of DMD prior to Screening based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test. Genetic report must describe a frameshift deletion, frameshift duplication, premature stop (nonsense), canonical splice site mutation, or other pathogenic variant in the DMD gene fully contained between exons 18 to 79 (inclusive) that is expected to lead to absence of dystrophin protein.
a. Mutations between or including exons 1 to 17 are not eligible.
b. In-frame deletions, in-frame duplications, and variants of uncertain significance (VUS”) are not eligible.
c. Mutations fully contained within exon 45 (inclusive) are not eligible.
3. Is able to cooperate with motor assessment testing.
4. Has a NSAA score > 16 and < 29 at the Screening visit.
5. Has a time to rise from floor < 5 seconds at the Screening visit.
6. Is on a stable daily dose of oral corticosteroids for at least 12 weeks before Screening and the dose and regimen are expected to remain constant (except for modifications to accommodate changes in weight) throughout the study.
7. Has rAAVrh74 antibody titers < 1:400 (ie, not elevated) as determined by an ELISA.
8. Subjects who are sexually active must agree to use, for the entire duration of the study, a condom and the female sexual partner must also use a highly effective form of birth control (eg, oral contraceptive). Refer to Appendix 1.
9. Has (a) parent(s) or legal guardian(s) who is (are) able to understand and comply with the study visit schedule and all other protocol requirements.
10. Is willing to provide informed assent (if applicable) and has (a) parent(s) or legal guardian(s) who is (are) willing to provide informed consent for the subject to participate in the study.
Are the trial subjects under 18? yes
Number of subjects for this age range: 120
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
A subject who meets any of the following criteria will be excluded from this study:
1. Has left ventricular ejection fraction < 40% on the screening ECHO or clinical signs and/or symptoms of cardiomyopathy.
2. Has had major surgery within 3 months prior to Day 1 or planned surgery or procedures that would interfere with the conduct of the study for any time during this study.
3. Has had presence of any other clinically significant illness, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic, or behavioral disease, or infection or malignancy or concomitant illness or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risks for gene transfer or a medical condition or extenuating circumstance that, in the opinion of the Investigator, might compromise the subject’s ability to comply with the protocol required testing or procedures or compromise the subject’s wellbeing, safety, or clinical interpretability.
4. Has serological evidence of current, chronic, or active human immunodeficiency virus, hepatitis C, or hepatitis B infection.
5. Has a symptomatic infection (eg, upper respiratory tract infection, pneumonia, pyelonephritis, meningitis) within 4 weeks prior to Day 1.
6. Demonstrates cognitive delay or impairment that could confound motor development in the opinion of the Investigator.
7. Has had treatment with any of the following therapies according to the time frames specified:
• Any time:
- Gene therapy
- Cell based therapy (eg, stem cell transplantation)
- CRISPR/Cas9, or any other form of gene editing
• Within 12 weeks of Day 1 and any time during the study:
- Use of human growth factor or givinostat
• Within 6 months of Day 1 and any time during the study:
- Any investigational medication
- Any treatment designed to increase dystrophin expression (eg, Translarna™, EXONDYS 51™, VILTEPSO™)
8. Has received a live virus vaccine within 4 weeks or inactive vaccine within 2 weeks of the Day 1 visit or expects to receive a vaccination during the first 3 months after Day 1.
9. Has abnormal laboratory values considered clinically significant including but not limited to:
• GGT > 2× 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. Has known hypersensitivity to delandistrogene moxeparvovec or any excipients of delandistrogene moxeparvovec.
11. Family does not want to disclose subject’s study participation with general practitioner/primary care physician and other medical providers.
12. In the opinion of the Investigator, the subject is not likely to be compliant with the study protocol.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method