CRD-TMH-001 in Muscular Dystrophy, Duchenne: NCT05514249 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: 28 September 2026. This strategic research report is not medical, regulatory or investment advice. Trial status and dates can change.
Phase 1
Clinical phase
Active, not recruiting
Recruitment status
1
Planned enrollment
2023-09-01
Primary-completion proxy
Executive view
NCT05514249 evaluates CRD-TMH-001 in Muscular Dystrophy, Duchenne. The disclosed sponsor is Cure Rare Disease, Inc., the design is Interventional, and the geographic footprint is United States. The first listed primary endpoint is To assess the safety of CRD-TMH-001, assessed over 1 year.
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 NCT05514249 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 CRD-TMH-001, while Company & Deal Intelligence MCP organization_fetch was queried for Cure Rare Disease, 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 |
|---|---|---|---|---|---|---|
| NCT05514249 | CRD-TMH-001 | Phase 1 / Active, not recruiting | Cure Rare Disease, Inc. | United States | To assess the safety of CRD-TMH-001 1 year | 2023-09-01 |
| NCT05670730 | Delpacibart zotadirsen | Phase 1/2 / Completed | Avidity Biosciences, Inc. | United States | Incidence of treatment-emergent adverse events (TEAEs) Through study completion, up to Day 85 (Part A) or Day 169 (Part B) | 2024-11-25 |
| NCT05540860 | Sevasemten | Phase 2 / Active, not recruiting | Edgewise Therapeutics, Inc. | United States | Number of adverse events during treatment with sevasemten or placebo 48 months | 2027-01-01 |
| NCT05524883 | Zeleciment Rostudirsen | Phase 1/2 / Active, not recruiting | Dyne Therapeutics, Inc. | Canada, South Korea, Belgium, United States, Ireland, United Kingdom, Italy, Australia, Spain | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Through study completion, up to Week 337 | 2031-09-01 |
| NCT05429372 | Fordadistrogene movaparvovec | Phase 2 / Terminated | Pfizer Inc. | United States, Australia | Incidence and severity of Treatment-Emergent Adverse Events and Serious Adverse Events Through Week 52 | 2025-10-03 |
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
NCT05514249 is a Phase 1, active, not recruiting study with 1 planned participants. Allocation is N/A, masking is None (Open Label), and the intervention model is Single Group Assignment.
The primary endpoint is “To assess the safety of CRD-TMH-001” over “1 year.” The retrieved endpoint description is: To assess the safety and tolerability of the therapeutic by measuring both serious and non-serious adverse events..
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 1 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
5 recent result records were selected as contextual evidence for Muscular Dystrophy, Duchenne. These records do not establish direct evidence for NCT05514249 unless the registration number matches.
Vamorolone for Duchenne Muscular Dystrophy
Phase 2; n=133; TTSTAND velocity(12-month): Difference (LS Mean) = 0.004(95.0% CI, -0.025 to 0.032); Difference (LS Mean) = 0.001(95.0% CI, -0.027 to 0.028); TTSTAND velocity(12-month): Difference (LS Mean) = 0.004(95.0% CI, -0.025 to 0.032); Difference (LS Mean) = 0.001(95.0% CI, -0.027 to 0.028) Source: https://pubmed.ncbi.nlm.nih.gov/42202243/
Lowering pre-existing immunity to adeno-associated virus-based gene therapy: Pre treatment with imlifidase or plasmapheresis prior to administration of delandistrogene moxeparvove…
Phase 1; n=8; Micro-dystrophin Expression(Week 12) = 21.5 % ( 0.96 - 42.03); Micro-dystrophin Expression(Week 12) = 1.72 % ( 1.46 - 2.11) Source: https://download.asgct.org/2026ASGCTAbstractPublication.pdf
Family experience data with delandistrogene moxeparvovec gene therapy treatment for Duchenne muscular dystrophy.
Not Applicable; n=31; Improvement in at least one of the most impactful symptoms = 96.0 % Source: https://download.asgct.org/2026ASGCTAbstractPublication.pdf
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
CRD-TMH-001 is indexed as CRISPR/Cas9 with dystrophin biology and a global stage of Phase 1. The asset profile lists Cure Rare Disease, Inc. as an originator or developer.
Cure Rare Disease, Inc. is indexed in United States with the website https://www.cureraredisease.org. Develops and funds life-saving genetic therapies for ultra-rare diseases & transforming the lives of patients The record lists 20 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 CRD-TMH-001 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: NCT05514249
Protocol source: https://clinicaltrials.gov/study/NCT05514249
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: 28 September 2026.
CRD-TMH-001 in Muscular Dystrophy, Duchenne is best understood through the interaction of protocol design, surrounding readouts, asset context and sponsor execution. The current record makes To assess the safety of CRD-TMH-001 and 2023-09-01 the leading decision points.
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