Treatment of a Single Patient With CRD-TMH-001

Cure Rare Disease, Inc

Status and phase: Active, not recruiting; Phase 1

Conditions: Duchenne Muscular Dystrophy

Treatments: Drug: CRD-TMH-001

Study type: Interventional

Funder types: Other

Identifiers

NCT05514249

CS-CRD-TMH-001

Details and patient eligibility

About

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.

Full 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.

Enrollment: 1 estimated patient

Sex: Male

Ages: 18 to 28 years old

Volunteers: No 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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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1 participants in 1 patient group

Single patient

Experimental group

Description:

Single dose of CRD-TMH-001 administered by IV

Treatment:

Drug: CRD-TMH-001