CRD-TMH-001 for Muscular Dystrophy, Duchenne

Phase-Based Progress Estimates
UMass Chan Medical School, Worcester, MA
Muscular Dystrophy, Duchenne+2 More
CRD-TMH-001 - Drug
18 - 65
What conditions do you have?

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.

Eligible Conditions

  • Muscular Dystrophy, Duchenne

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Muscular Dystrophy, Duchenne

Study Objectives

1 Primary · 0 Secondary · Reporting Duration: 1 year

1 year
To assess the safety of CRD-TMH-001

Trial Safety

Safety Progress

1 of 3

Other trials for Muscular Dystrophy, Duchenne

Trial Design

1 Treatment Group

Single patient
1 of 1
Experimental Treatment

1 Total Participants · 1 Treatment Group

Primary Treatment: CRD-TMH-001 · No Placebo Group · Phase 1

Single patient
Experimental Group · 1 Intervention: CRD-TMH-001 · Intervention Types: Drug

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 1 year
Closest Location: UMass Chan Medical School · Worcester, MA
Photo of massachusetts 1Photo of massachusetts 2Photo of massachusetts 3
2021First Recorded Clinical Trial
1 TrialsResearching Muscular Dystrophy, Duchenne
2 CompletedClinical Trials

Who is running the clinical trial?

University of Massachusetts, WorcesterOTHER
306 Previous Clinical Trials
741,128 Total Patients Enrolled
Cure Rare Disease, IncLead Sponsor
Brenda Wong, MDPrincipal InvestigatorUMass Chan Medical School
1 Previous Clinical Trials
22 Total Patients Enrolled
1 Trials studying Muscular Dystrophy, Duchenne
22 Patients Enrolled for Muscular Dystrophy, Duchenne
Medical AffairsStudy DirectorCure Rare Disease
34 Previous Clinical Trials
8,327 Total Patients Enrolled

Eligibility Criteria

Age 18 - 65 · Male Participants · 3 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 9th, 2021

Last Reviewed: August 12th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.