Treatment of a Single Patient With CRD-TMH-001

C

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)

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

Trial contacts and locations

1

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Data sourced from clinicaltrials.gov

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