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Treatment of a Single Patient With CRD-TMH-001

NCT05514249 · Status: UNKNOWN · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 1

Last updated 2022-09-01

No results posted yet for this study

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.

Conditions

Interventions

DRUG

CRD-TMH-001

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

Sponsors & Collaborators

  • University of Massachusetts, Worcester

    collaborator OTHER

  • Cure Rare Disease, Inc

    lead OTHER

Principal Investigators

  • Brenda Wong, MD · UMass Chan Medical School

  • Medical Affairs · Cure Rare Disease

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
28 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-08-31
Primary Completion
2023-09-30
Completion
2023-09-30
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05514249 on ClinicalTrials.gov