Delandistrogene moxeparvovec in Muscular Dystrophy, Duchenne: NCT03769116 Clinical Landscape Report 2026
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This Clinical Landscape Report was built with PatSnap Life Sciences MCP workflows. Clinical Trials MCP supplies protocol and result records, Drug & Asset MCP adds asset context, and Company & Deal Intelligence MCP resolves sponsor background. Use the same structured MCP building blocks in your research workflow.
Data snapshot: 16 October 2026. This strategic research report is not medical, regulatory or investment advice. Trial status and dates can change.
Phase 1/2
Clinical phase
Completed
Recruitment status
not reported
Planned enrollment
2020-12-08
Primary-completion proxy
Executive view
NCT03769116 evaluates Delandistrogene moxeparvovec in Muscular Dystrophy, Duchenne. The disclosed sponsor is Sarepta Therapeutics, Inc., the design is Interventional, and the geographic footprint is United States. The first listed primary endpoint is Change From Baseline at Week 12 in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression as Measured by Western Blot Adjusted by Muscle Content, assessed over Baseline, Week 12 (Part 1).
The key landscape question is whether this protocol can generate a clinically interpretable signal relative to nearby programs. Phase alone is not a measure of evidence quality. Endpoint relevance, comparator choice, masking, enrollment feasibility, patient selection, country coverage and follow-up must be considered together.
PatSnap MCP Servers make this assessment reproducible by keeping protocol facts, result evidence, asset attributes and sponsor identity in separate structured calls.
How the MCP evidence stack was assembled
Clinical Trials MCP clinical_trial_fetch retrieved the design, outcomes, phase, status, enrollment, sponsor, countries and timing for NCT03769116 and selected peers. clinical_trial_result_fetch supplied detailed result records from the wider Muscular Dystrophy, Duchenne landscape. Drug & Asset MCP drug_fetch was queried for Delandistrogene moxeparvovec, while Company & Deal Intelligence MCP organization_fetch was queried for Sarepta Therapeutics, Inc..
This separation reduces a common diligence error: treating a registry label, a company description or a result excerpt as if each represented the complete evidence package. Explore the source workflow at the PatSnap MCP marketplace.
Trial landscape table
| Trial | Asset / intervention | Phase / status | Sponsor | Geography | Primary endpoint | Readout proxy |
|---|---|---|---|---|---|---|
| NCT03769116 | Delandistrogene moxeparvovec | Phase 1/2 / Completed | Sarepta Therapeutics, Inc. | United States | Change From Baseline at Week 12 in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression as Measured b… Baseline, Week 12 (Part 1) | 2020-12-08 |
| NCT04240314 | AT-702 | Phase 1/2 / Completed | Nationwide Children's Hospital | United States | Number of Participants With Unacceptable Toxicity. 2 years | 2023-11-13 |
| NCT04184882 | Bocidelpar | Phase 1 / Terminated | Astellas Pharma, Inc. | United States | Number of participants with Treatment Emergent Adverse Events (TEAEs) Up to Week 28 | 2022-09-04 |
| NCT04179409 | Casimersen | Phase 2 / Completed | Nationwide Children's Hospital | United States | Change in Dystrophin Expression From Baseline Following Treatment With Either AMONDYS 45 (Previously Casimersen), EXOND… Baseline, 1 year | 2021-09-01 |
| NCT04129294 | Brogidirsen | Phase 1/2 / Completed | National Center of Neurology & Psychiatry | Japan | Adverse event and adverse drug reaction [Safety and Tolerability] At the end of Part 2 (24 weeks treatment period and 12 weeks follow u… | 2022-05-31 |
The table aligns endpoints, sponsors, phases, geographies and readout proxies. It is descriptive, not a head-to-head efficacy comparison. Differences in population, baseline risk, intervention schedule and follow-up can dominate apparent cross-trial differences.
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Protocol design and endpoint interpretation
NCT03769116 is a Phase 1/2, completed study with not reported planned participants. Allocation is Randomized, masking is Quadruple, and the intervention model is Parallel Assignment.
The primary endpoint is “Change From Baseline at Week 12 in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression as Measured by Western Blot Adjusted by Muscle Content” over “Baseline, Week 12 (Part 1).” The retrieved endpoint description is: 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..
Interpretation should test whether the endpoint captures a clinically meaningful change, whether its time horizon matches the proposed biology, and whether treatment discontinuation or missing data can bias the estimate. The planned enrollment of not reported should be assessed against expected effect size, event frequency, multiplicity, subgroup plans and attrition.
No named control drug was returned in the protocol field set. A placebo comparator can strengthen internal efficacy assessment, while an active comparator may better test clinical differentiation. Single-arm and open-label programs require greater weight on objective outcomes, independent assessment and external benchmarks.
Indexed readouts in the surrounding landscape
0 recent result records were selected as contextual evidence for Muscular Dystrophy, Duchenne. These records do not establish direct evidence for NCT03769116 unless the registration number matches.
Indexed evidence status
No directly comparable result record was retrieved for this disease query. This is a monitoring gap, not evidence of absence.
Result fields should be reconciled with the source record before quantitative comparison. Population definitions, analysis sets, dose cohorts, estimands, confidence intervals, rescue therapy and follow-up can materially change the meaning of a numerical endpoint. Clinical Trials MCP supports repeatable refreshes as result records change.
Asset and sponsor context
Delandistrogene moxeparvovec is indexed as AAV based gene therapy with micro-dystrophin biology and a global stage of Approved. The asset profile lists Nationwide Children's Hospital as an originator or developer.
Sarepta Therapeutics, Inc. is indexed in an unreported country with the website https://www.sarepta.com. Sarepta Therapeutics focuses on the discovery and development of precision genetic medicine to treat rare diseases. The record lists 27 development-stage drug assets.
For execution diligence, monitor sponsor ownership, collaborator additions, site expansion, protocol amendments and enrollment revisions. A change in ownership or geography can alter operational confidence as well as the commercial meaning of a future readout.
Development white space
- Endpoint white space. Determine whether a more patient-relevant outcome, longer durability window or blinded central assessment would resolve uncertainty left by the current endpoint.
- Population white space. Test biomarker-defined, treatment-line or risk-stratified subgroups where effect size and unmet need could be clearer.
- Comparator white space. Identify whether the study can support differentiation against the current standard of care rather than only activity against baseline or placebo.
- Geographic white space. Assess whether the disclosed footprint supports recruitment, regulatory transferability and commercial generalizability.
- Sequencing white space. Clarify whether Delandistrogene moxeparvovec is intended for monotherapy, combination, maintenance, rescue or an earlier treatment line.
White space should be framed as an unanswered development question, not merely an unoccupied mechanism label. A credible program closes a measurable clinical uncertainty with a design that can be executed and interpreted.
Strategic implications and next readouts
For sponsors, the endpoint hierarchy, safety window, enrollment pace and protocol amendment history should all support the same target product profile. Advancement criteria should be set before the readout and tied to clinical effect, uncertainty, tolerability and operational feasibility.
For business-development teams, differentiation may come from a sharper population, stronger comparator, more durable benefit, simpler delivery or clearer sequencing role. For investors, the central risk is evidence quality relative to time and capital, not the phase label in isolation.
Track recruitment status, enrollment changes, primary-completion timing, endpoint revisions, new result records, sponsor ownership and collaborator changes. Re-run the PatSnap MCP workflow when a surrogate becomes a clinical outcome, a single-country study expands, the comparator changes or a new result materially shifts the competitive benchmark.
Source trail and bottom line
Anchor trial: NCT03769116
Protocol source: https://clinicaltrials.gov/study/NCT03769116
MCP sources: Clinical Trials MCP (clinical_trial_fetch and clinical_trial_result_fetch); Drug & Asset MCP (drug_fetch); Company & Deal Intelligence MCP (organization_fetch).
Data snapshot: 16 October 2026.
Delandistrogene moxeparvovec in Muscular Dystrophy, Duchenne is best understood through the interaction of protocol design, surrounding readouts, asset context and sponsor execution. The current record makes Change From Baseline at Week 12 in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression as Measured by Western Blot Adjusted by Muscle Content and 2020-12-08 the leading decision points.
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