Deramiocel in Muscular Dystrophy, Duchenne: NCT03406780 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 2
Clinical phase
Completed
Recruitment status
not reported
Planned enrollment
2020-03-10
Primary-completion proxy
Executive view
NCT03406780 evaluates Deramiocel in Muscular Dystrophy, Duchenne. The disclosed sponsor is Capricor, Inc., the design is Interventional, and the geographic footprint is United States. The first listed primary endpoint is Change From Baseline in Functional Capacity as Assessed by the Mid-level (Elbow) Dimension Score of the Performance of Upper Limb (PUL) Version 1.2 at Month 12, assessed over Baseline, Month 12.
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 NCT03406780 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 Deramiocel, while Company & Deal Intelligence MCP organization_fetch was queried for Capricor, 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 |
|---|---|---|---|---|---|---|
| NCT03406780 | Deramiocel | Phase 2 / Completed | Capricor, Inc. | United States | Change From Baseline in Functional Capacity as Assessed by the Mid-level (Elbow) Dimension Score of the Performance of… Baseline, Month 12 | 2020-03-10 |
| 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
NCT03406780 is a Phase 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 in Functional Capacity as Assessed by the Mid-level (Elbow) Dimension Score of the Performance of Upper Limb (PUL) Version 1.2 at Month 12” over “Baseline, Month 12.” The retrieved endpoint description is: Change from baseline in functional capacity as assessed by the mid-level (elbow) dimension of PUL version 1.2 at Month 12 expressed as percentile ranked change. PUL 1.2 scale assesses motor performance in the upper limb. PUL 1.2 included 22 items. One entry item to define the starting functional level, and 21 items subdivided into: Shoulder Level (score 0 to 16); Elbow Level (score 0 to 34); Distal Level Dimension (score 0 to 24). The total score range was from 0 to 74. For all items, the higher the score, the better the outcome. A negative change indicates worst the outcome. Change from baseline and baseline va….
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 NCT03406780 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
Deramiocel is indexed as Stem cell therapy with target not reported biology and a global stage of NDA/BLA. The asset profile lists Capricor Therapeutics, Inc. as an originator or developer.
Capricor, Inc. is indexed in an unreported country. Provides cardiac stem cell therapeutics biotechnology services The record lists 2 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 Deramiocel 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: NCT03406780
Protocol source: https://clinicaltrials.gov/study/NCT03406780
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.
Deramiocel 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 in Functional Capacity as Assessed by the Mid-level (Elbow) Dimension Score of the Performance of Upper Limb (PUL) Version 1.2 at Month 12 and 2020-03-10 the leading decision points.
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