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Gene Editing Therapy

CRD-TMH-001 for Duchenne Muscular Dystrophy

Phase 1
Waitlist Available
Led By Brenda Wong, MD
Research Sponsored by Cure Rare Disease, Inc
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be between 18 and 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year
Awards & highlights

Study Summary

This trial is testing a new gene-editing treatment for a rare form of muscular dystrophy.

Eligible Conditions
  • Duchenne Muscular Dystrophy

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 year
This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
To assess the safety of CRD-TMH-001

Trial Design

1Treatment groups
Experimental Treatment
Group I: Single patientExperimental Treatment1 Intervention
Single dose of CRD-TMH-001 administered by IV

Find a Location

Who is running the clinical trial?

Cure Rare Disease, IncLead Sponsor
University of Massachusetts, WorcesterOTHER
342 Previous Clinical Trials
976,609 Total Patients Enrolled
Brenda Wong, MDPrincipal InvestigatorUMass Chan Medical School
1 Previous Clinical Trials
22 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there any remaining openings for participants in this clinical trial?

"At present, clinicaltrials.gov states that this research is not currently enrolling participants; however, the trial began on August 31st 2022 and was most recently updated on the same day. Despite its inactivity, there are still 86 other trials actively searching for volunteers."

Answered by AI

Do participants need to be of a certain age in order to qualify for this research?

"This medical trial is seeking participants aged between 18 and 28."

Answered by AI

What potential hazards are associated with CRD-TMH-001?

"The safety profile of CRD-TMH-001 was assessed to be 1 due to it being a Phase 1 trial, indicating that there is limited evidence for its efficacy and security."

Answered by AI

Who is eligible to partake in this clinical examination?

"This trial is enrolling a single participant aged 18 to 28, diagnosed with Duchenne's muscular dystrophy. To qualify for the study, they must also have signed informed consent documents and possess both a confirmed genetic mutation as well as absence of elevated AAV9 NAbs levels."

Answered by AI

Who else is applying?

How old are they?
18 - 65
What site did they apply to?
UMass Chan Medical School
What portion of applicants met pre-screening criteria?
Met criteria
Recent research and studies
clinicaltrials.govStudy
Treatment of a Single Patient With CRD-TMH-001
2022. "Treatment of a Single Patient With CRD-TMH-001". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05514249.
~0 spots leftby Apr 2025
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