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Also known as SRP-9001, ELEVIDYS, RO7494222, delandistrogene moxeparvovec-rokl
delandistrogene moxeparvovec · 7 trials · 2 indications
The NSAA is a healthcare provider administered scale that rates performance of various motor abilities in ambulant children with Duchenne Muscular Dystrophy and is used to monitor disease progression and treatment effects. During assessment, participants are asked to perform 17 different functional activities that are graded as: 2 - "Normal" - no obvious modification of activity; 1 - Modified method but achieves goal independent of assistance; 0 - Unable to achieve independently. The NSAA total score is defined as the sum of all 17 items, ranging from 0 (worst) to 34 (best). The response vector consists of the change from baseline in NSAA total score at the post-baseline visit. The model includes the covariates of treatment group, visit, treatment group by visit interaction, age group, baseline NSAA total score, and baseline NSAA total score by visit interaction. All covariates are fixed effects in this analysis. An increase in score indicates an improvement in motor function.
Baseline muscle biopsies with ultrasound guidance were performed pre-treatment and post-treatment (Week 12) on all participants. The change from baseline in delandistrogene moxeparvovec dystrophin expression in these muscle biopsy samples was determined by Western Blot. Dystrophin protein was measured and then adjusted based on the percentage of muscle content in the biopsy sample. An increase in protein expression indicates production of the delandistrogene moxeparvovec dystrophin protein.
The NSAA is a healthcare provider administered scale that rates performance of various motor abilities in ambulant children with Duchenne Muscular Dystrophy and is used to monitor disease progression and treatment effects. During assessment, participants are asked to perform 17 different functional activities that are graded as: 2 - "Normal" - no obvious modification of activity; 1 - Modified method but achieves goal independent of assistance; 0 - Unable to achieve independently. The NSAA total score is defined as the sum of all 17 items, ranging from 0 (worst) to 34 (best). The response vector consists of the change from baseline in NSAA total score at the post-baseline visit. The model includes the covariates of treatment group, visit, treatment group by visit interaction, age group, baseline NSAA total score, and baseline NSAA total score by visit interaction. All covariates are fixed effects in this analysis. An increase in score indicates an improvement in motor function.
An AE is any untoward medical occurrence in a clinical study participant that does not necessarily have a causal relationship with the study drug. An AE can, therefore, be any unfavorable and unintended symptom, sign, disease, condition, or test abnormality that occurs during or after administration of a study drug, whether or not considered related to the study drug. A summary of serious and all other non-serious adverse events regardless of causality is located in the Reported Adverse Events module.
| Arm | Type | Description |
|---|---|---|
| Delandistrogene Moxeparvovec | EXPERIMENTAL | Participants received delandistrogene moxeparvovec in a previous clinical study. |
| Delandistrogene Moxeparvovec followed by Placebo | EXPERIMENTAL | Participants will receive single IV infusion of delandistrogene moxeparvovec on Day 1. Then, participants will receive a single IV infusion of matching placebo at approximately 72 weeks. |
| Placebo followed by Delandistrogene Moxeparvovec | PLACEBO_COMPARATOR | Participants will receive matching placebo IV infusion on Day 1. Then, participants will have the opportunity to receive a single IV infusion of delandistrogene moxeparvovec at approximately 72 weeks. |
| Delandistrogene Moxeparvovec in Part 1 Followed by Placebo in Part 2 | EXPERIMENTAL | Participant will receive delandistrogene moxeparvovec at Part 1 followed by matching placebo at Part 2 followed by an open-label extension at Part 3. |
| Placebo in Part 1 Followed by Delandistrogene Moxeparvovec in Part 2 | EXPERIMENTAL | Participant will receive matching placebo at Part 1 followed by delandistrogene moxeparvovec at Part 2 followed by an open-label extension at Part 3. |
| Cohort A: Delandistrogene Moxeparvovec | EXPERIMENTAL | Participants will receive a Single IV infusion of delandistrogene moxeparvovec on Day 1. |
| Cohort B: Delandistrogene Moxeparvovec | EXPERIMENTAL | Participants will receive a Single IV infusion of delandistrogene moxeparvovec on Day 1. |
| Name | Type | Description |
|---|---|---|
| delandistrogene moxeparvovec | GENETIC | No study drug will be administered as part of this study. Eligible participants who received treatment with delandistrogene moxeparvovec during a previous clinical study will be included. |
| placebo | GENETIC | Single IV infusion of matching placebo |
Inclusion Criteria: * Received delandistrogene moxeparvovec for Duchenne muscular dystrophy in a previous clinical study. * Has (a) parent(s) or legal caregiver(s) or is ≥18 years of age and able to understand and comply with the study visit schedule and all other protocol requirements. Exclusion ...
Delandistrogene moxeparvovec is an investigational gene therapy being developed for Duchenne Muscular Dystrophy, a genetic muscle-wasting disorder. It is designed as an exogenous gene therapy intended to address the underlying cause of DMD. Sarepta Therapeutics, Inc. (SRPT) is developing the therapy, which is in Phase 3 clinical development.
Delandistrogene moxeparvovec is being developed to treat Duchenne Muscular Dystrophy, including both ambulatory and non-ambulatory patients. Clinical studies have enrolled males with DMD across a range of ages, from children under four years old to older participants. It remains investigational and is not yet approved for commercial use.
Delandistrogene moxeparvovec targets the DMD gene, delivering an exogenous gene to muscle cells. The goal is to address the genetic defect underlying Duchenne Muscular Dystrophy. By providing a functional copy of the gene, the therapy aims to produce a shortened form of dystrophin protein. It is classified as a gene therapy.
Sarepta Therapeutics, Inc. (ticker SRPT) is developing delandistrogene moxeparvovec. The company is the sponsor of the clinical program, which includes Phase 3 trials and a long-term follow-up study. Sarepta is a biopharmaceutical company focused on genetic medicines for rare neuromuscular diseases.
Delandistrogene moxeparvovec is in Phase 3 clinical development. It is an investigational gene therapy and has not been approved by the FDA. The program includes completed and ongoing Phase 3 studies, as well as a Phase 2 study in participants under four years of age.
Delandistrogene moxeparvovec has been studied in several clinical trials, including NCT05096221, a completed Phase 3 study in DMD; NCT05881408, a Phase 3 study in non-ambulatory and ambulatory participants; NCT05967351, a Phase 3 long-term follow-up study; and NCT06128564, a Phase 2 study in children under four years old.