NCT05514249

Treatment of a Single Patient With CRD-TMH-001

• Status: Unknown
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 1 (estimated)
• Sponsor: Cure Rare Disease, Inc · Academic / Other
• Sex: Male
• Age: 18 Years – 28 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Duchenne Muscular Dystrophy

Interventions

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

Timeline

• Start date: 2022-08-31
• Primary completion: 2023-09-01
• Completion: 2023-09-01
• First posted: 2022-08-24
• Last updated: 2022-09-01

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study