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NCT05514249 Clinical Trial

Treatment of a Single Patient With CRD-TMH-001

Duchenne Muscular Dystrophy Clinical Trial

Official title:
Treatment of a Single Patient With CRD-TMH-001

Clinical Trial Details — Status: Active, not recruiting

Administrative data
NCT number NCT05514249
Other study ID # CS-CRD-TMH-001
Secondary ID
Status Active, not recruiting
Phase Phase 1
First received
Last updated
Start date August 31, 2022
Est. completion date September 2023

Study information
Verified date August 2022
Source Cure Rare Disease, Inc
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

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.

Description:

The objective of the study is to assess the safety and preliminary efficacy of CRD-TMH-001 after intravenous administration for a period of 1 year with long-term follow-up out to 15 years.

Recruitment information / eligibility
Status Active, not recruiting
Enrollment 1
Est. completion date September 2023
Est. primary completion date September 2023
Accepts healthy volunteers No
Gender Male
Age group 18 Years to 28 Years
Eligibility Inclusion Criteria: - Completion of informed consent - Confirmation of genetic mutation - Confirmation of absence of elevated AAV9 NAbs Exclusion Criteria: - Any significant medical issue(s) (past or current) that would, in the opinion of the Principal Investigator (PI), prevent this patient from being dosed.

Study Design

Related Conditions & MeSH terms

Intervention

Drug:
• CRD-TMH-001
Participant will receive a single dose of CRD-TMH-001 administered via intravenous injection.

Locations
Country Name City State
United States UMass Chan Medical School Worcester Massachusetts

Sponsors (2)
Lead Sponsor Collaborator
Cure Rare Disease, Inc University of Massachusetts, Worcester

Country where clinical trial is conducted

United States, 

Outcome
Type Measure Description Time frame Safety issue
Primary To assess the safety of CRD-TMH-001 To assess the safety and tolerability of the therapeutic by measuring both serious and non-serious adverse events. 1 year
See also
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Active, not recruiting NCT04906460 - Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53) Phase 1/Phase 2
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