Treatment of a Single Patient With CRD-TMH-001

Status unconfirmed · Phase 1

Conditions studied: Duchenne Muscular Dystrophy

In brief

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.

Key facts

Study ID

NCT05514249

Run by

Cure Rare Disease, Inc

People needed

1

Starts

2022-08-31

Expected to finish

2023-09-01

Last updated by the study team

2022-09-01

Who can join

Age: 18 and older, up to 28. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Completion of informed consent
• Confirmation of genetic mutation
• Confirmation of absence of elevated AAV9 NAbs

You may not qualify if…

• Any significant medical issue(s) (past or current) that would, in the opinion of the Principal Investigator (PI), prevent this patient from being dosed.

Where it is running

• UMass Chan Medical School — Worcester, Massachusetts, United States

Full record on ClinicalTrials.gov

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