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Treatment of a Single Patient With CRD-TMH-001

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT05514249
Recruitment Status : Active, not recruiting
First Posted : August 24, 2022
Last Update Posted : September 1, 2022
University of Massachusetts, Worcester
Information provided by (Responsible Party):
Cure Rare Disease, Inc

Brief 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.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: CRD-TMH-001 Phase 1

Detailed 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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 1 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Masking Description: Single patient clinical trial
Primary Purpose: Treatment
Official Title: Treatment of a Single Patient With CRD-TMH-001
Actual Study Start Date : August 31, 2022
Estimated Primary Completion Date : September 2023
Estimated Study Completion Date : September 2023

Arm Intervention/treatment
Experimental: Single patient
Single dose of CRD-TMH-001 administered by IV
Drug: CRD-TMH-001
Participant will receive a single dose of CRD-TMH-001 administered via intravenous injection.

Primary Outcome Measures :
  1. To assess the safety of CRD-TMH-001 [ Time Frame: 1 year ]
    To assess the safety and tolerability of the therapeutic by measuring both serious and non-serious adverse events.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years to 28 Years   (Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT05514249

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United States, Massachusetts
UMass Chan Medical School
Worcester, Massachusetts, United States, 01655
Sponsors and Collaborators
Cure Rare Disease, Inc
University of Massachusetts, Worcester
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Principal Investigator: Brenda Wong, MD UMass Chan Medical School
Study Director: Medical Affairs Cure Rare Disease
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Responsible Party: Cure Rare Disease, Inc Identifier: NCT05514249    
Other Study ID Numbers: CS-CRD-TMH-001
First Posted: August 24, 2022    Key Record Dates
Last Update Posted: September 1, 2022
Last Verified: August 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Cure Rare Disease, Inc:
Muscular Dystrophy
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked