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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 (DMD), a progressive neuromuscular disease. It is designed to deliver a gene to help produce a shortened form of dystrophin, the protein missing in DMD. The drug is currently in Phase 3 clinical development and is not yet approved.
Delandistrogene moxeparvovec is a gene therapy that targets the underlying cause of Duchenne muscular dystrophy by delivering a gene that encodes a functional form of dystrophin. This micro-dystrophin is intended to compensate for the defective dystrophin gene in patients with DMD, potentially slowing muscle degeneration.
Delandistrogene moxeparvovec is being developed by Sarepta Therapeutics, Inc., a biopharmaceutical company traded on NASDAQ under the ticker SRPT. Sarepta is conducting multiple clinical trials to evaluate the safety and efficacy of this gene therapy for Duchenne muscular dystrophy.
Delandistrogene moxeparvovec is in Phase 3 clinical development for Duchenne muscular dystrophy. It is an investigational drug, meaning it has not been approved by regulatory authorities. Several trials are ongoing, including a completed Phase 3 study and an active Phase 3 trial enrolling non-ambulatory and ambulatory participants.
Delandistrogene moxeparvovec is being studied in multiple trials, including NCT05096221, a completed Phase 3 study in ambulatory DMD patients, and NCT05881408, an active Phase 3 trial in non-ambulatory and ambulatory participants. Other trials include NCT03375164 (Phase 1, completed) and NCT06128564 (Phase 2, active).
Yes, delandistrogene moxeparvovec is also known as SRP-9001. Clinical trial records for this gene therapy use both names interchangeably. This investigational treatment is being developed by Sarepta Therapeutics for Duchenne muscular dystrophy and is currently in Phase 3 trials.