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
Conditions
Interventions
Product Code: SRP-9001
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Delandistrogene moxeparvovec
CAS Number: 2305040-16-6
Current Sponsor code: SRP-9001
Other descriptive name: AD
Sponsors
Sarepta Therapeutics, Inc.
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: A subject must meet the following criteria to be eligible to participate in this study: 1. Is male at birth and 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 mutation, or other pathogenic variant in the DMD gene fully contained between exons 18 to 79 (inclusive) that is expected to lead to complete absence of dystrophin protein. • Mutations fully or partially contained within exons 1-17 (inclusive) are not eligible. 2. Cohort 1 only (non-ambulatory): a. Has been non-ambulatory for a minimum of 6 months with onset of non-ambulatory status defined as participant- or caregiver-reported age at continuous wheelchair use, approximated to the nearest month, with an NSAA walk score of "0" and inability to perform the 10MWR at the Screening visit. b. Has a PUL entry item score = 2 at the Screening visit. 3. Cohort 2 only (ambulatory = 8 years to 3 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 × ULN • Glutamate dehydrogenase (GLDH) >15 U/L • Total bilirubin > ULN. Note that elevations in total bilirubin confirmed to be due toGilbert's syndrome are not exclusionary. • White blood cell count > 18,500 per µL • Platelets = 150,000 per µL 11. Has a known hypersensitivity to SRP-9001 or its excipients 12. Subject or family does not want to disclose subject’s study participation with general practitioner/primary care physician and other medical providers. 13. 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 in Part 1 as assessed by the Performance Upper Limb (PUL) (Version 2.0 [V2.0]);Secondary Objective: To evaluate the effect of SRP-9001 on respiratory function in Part 1 as assessed by: o Forced vital capacity (FVC) percent predicted o Peak expiratory flow (PEF) percent predicted To evaluate micro-dystrophin expression from SRP-9001 at 12 weeks (Part 1) as measured by western blot of biopsied muscle tissue To evaluate subject and parent/caregiver proxy reported 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 PUL (V2.0) total score ;Timepoint(s) of evaluation of this end point: From Baseline to Week 72 (Part 1) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): •Change in PEF% predicted from Baseline to Week 72 (Part 1) •Change in FVC% predicted from Baseline to Week 72 (Part 1) •The quantity of micro-dystrophin protein expression at Week 12 (Part 1) as measured by western blot •Change in PROMIS score per domain from Baseline to 72 weeks (Part 1) •Change in the NSAA score from Baseline to Week 72 (Part 1) and Week 52 (Part 2) •Incidence of treatment-emergent adverse events (TEAEs) •Incidence of adverse events of special interest (AESIs) •Clinically significant changes in vital signs and physical examination findings •Incidence of serious adverse events (SAEs) •Clinically significant changes in safety laboratory assessments, electrocardiograms (ECGs), echocardiograms (ECHOs) ;Timepoint(s) of evaluation of this end point: •Change in PEF% predicted from Baseline to Week 72 (Part 1) •Change in FVC% predicted from Baseline to Week 72 (Part 1) •The quantity of micro-dystrophin protein expression at Week 12 (Part 1) as measured by western blot •Change in PROMIS score per domain from Baseline to 72 weeks (Part 1) •Change in the NSAA score from Baseline to Week 72 (Part 1) and Week 52 (Part 2) •Incidence of treatment-emergent adverse events (TEAEs) •Incidence of adverse events of special interest (AESIs) •Clinically significant changes in vital signs and physical examination findings •Incidence of serious adverse events (SAEs) •Clinically significant changes in safety laboratory assessments, electrocardiograms (ECGs), echocardiograms (ECHOs) | — |
Countries
Australia, Belgium, France, Germany, Israel, Italy, Japan, Spain, Sweden, United Kingdom, United States
Contacts
Public ContactSarepta Clinical Trial Inquiries
Sarepta Therapeutics, Inc.
Outcome results
None listed