Treatment of a Single Patient With CRD-TMH-001

Status

UNKNOWN

Phases

Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT05514249

Enrollment

Registered

2022-08-24

Start date

2022-08-31

Completion date

2023-09-30

Last updated

2022-09-01

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Duchenne Muscular Dystrophy

Keywords

Duchenne, Muscular Dystrophy, CRISPR, gene-editing

Brief 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.

Detailed 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.

Interventions

DRUGCRD-TMH-001

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

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Masking description

Single patient clinical trial

Eligibility

Sex/Gender

MALE

Age

18 Years to 28 Years

Healthy volunteers

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.

Design outcomes

Primary

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

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 7, 2026