NCT05514249 is a Phase 1 clinical trial of CRD-TMH-001 in Duchenne Muscular Dystrophy, sponsored by Cure Rare Disease, Inc.

Who is sponsoring NCT05514249?

NCT05514249 is sponsored by Cure Rare Disease, Inc.

What drugs are being tested in NCT05514249?

Interventions in NCT05514249: CRD-TMH-001.

What condition does NCT05514249 study?

NCT05514249 studies Duchenne Muscular Dystrophy.

Is NCT05514249 still recruiting?

NCT05514249 is currently status unknown. Last updated 31 August 2022.

Last reviewed 2022-08-31 · How we verify

NCT05514249

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Treatment of a Single Patient With CRD-TMH-001

Status unknown Phase 1 Last updated 31 August 2022

What this trial tests

Phase 1 trial testing CRD-TMH-001 in Duchenne Muscular Dystrophy in 1 participant. Status unknown.

Timeline

31 August 2022

Primary endpoint
1 September 2023

1 September 2023

Quick facts

Lead sponsor	Cure Rare Disease, Inc
Phase	Phase 1
Status	Status unknown
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	1
Start date	31 August 2022
Primary completion	1 September 2023
Estimated completion	1 September 2023
Sites	1 location across United States

Drugs / interventions tested

CRD-TMH-001 — full drug profile →

Conditions studied

Duchenne Muscular Dystrophy — all drugs for Duchenne Muscular Dystrophy →

Sponsor

Cure Rare Disease, Inc — full company profile →

Who can join

Adults 18 to 28, male only, with Duchenne Muscular Dystrophy. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

To assess the safety of CRD-TMH-001
Time frame: 1 year
To assess the safety and tolerability of the therapeutic by measuring both serious and non-serious adverse events.

Sponsor's own description

The study is a single patient study intended to understand the effects of a gene-editing therapeutic to treat a rare mutation of Duchenne muscular dystrophy.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

Lethal immunotoxicity in high-dose systemic AAV therapy.
Duan D. · · 2023 · cited 127× · PMID 37822079 · DOI 10.1016/j.ymthe.2023.10.015
CRISPR-Based Gene Therapies: From Preclinical to Clinical Treatments.
Laurent M, Geoffroy M, Pavani G, Guiraud S. · · 2024 · cited 45× · PMID 38786024 · DOI 10.3390/cells13100800
Current Strategies for Increasing Knock-In Efficiency in CRISPR/Cas9-Based Approaches.
Leal AF, Herreno-Pachón AM, Benincore-Flórez E, Karunathilaka A, et al · · 2024 · cited 32× · PMID 38473704 · DOI 10.3390/ijms25052456
Engineering extracellular vesicles to deliver CRISPR ribonucleoprotein for gene editing.
Whitley JA, Cai H. · · 2023 · cited 29× · PMID 37723839 · DOI 10.1002/jev2.12343
CRISPR-Cas System: The Current and Emerging Translational Landscape.
Bhokisham N, Laudermilch E, Traeger LL, Bonilla TD, et al · · 2023 · cited 25× · PMID 37190012 · DOI 10.3390/cells12081103
Revolutionizing <i>in vivo</i> therapy with CRISPR/Cas genome editing: breakthroughs, opportunities and challenges.
Macarrón Palacios A, Korus P, Wilkens BGC, Heshmatpour N, et al · · 2024 · cited 23× · PMID 38362491 · DOI 10.3389/fgeed.2024.1342193
Advancing CRISPR genome editing into gene therapy clinical trials: progress and future prospects.
Cetin B, Erendor F, Eksi YE, Sanlioglu AD, et al · · 2025 · cited 15× · PMID 40160040 · DOI 10.1017/erm.2025.10
Gene therapy for genetic diseases: challenges and future directions.
Qie B, Tuo J, Chen F, Ding H, et al · · 2025 · cited 14× · PMID 39949979 · DOI 10.1002/mco2.70091

Verify or expand the search:

Other recruiting trials for Duchenne Muscular Dystrophy

Currently open trials in the same condition.

NCT07287189 — Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients · Phase 2 · recruiting
NCT06817382 — A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Ma · Phase 1 · recruiting
NCT06402942 — Gamified Occupational Therapy for Adolescents With Duchenne Muscular Dystrophy · NA · recruiting
NCT06450639 — A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD) · Phase 2 · active not recruiting
NCT06692426 — Trial of Cell Based Therapy for DMD · Phase 1 · recruiting

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT05514249 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Cure Rare Disease, Inc
Last refreshed: 31 August 2022

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT05514249.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing