Treatment of a Single Patient With CRD-TMH-001

Cure Rare Disease, Inc1 enrolled

Overview

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.

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Duchenne Muscular Dystrophy

Interventions

CRD-TMH-001DRUG

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

Eligibility

Sex: MALEMin age: 18 YearsMax age: 28 Years

Medical Language ↔ Plain English

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.

Locations (1)

UMass Chan Medical School

Worcester, Massachusetts, United States

Outcomes

Primary Outcomes

To assess the safety of CRD-TMH-001

To assess the safety and tolerability of the therapeutic by measuring both serious and non-serious adverse events.

Time frame: 1 year

Data from ClinicalTrials.gov

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