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. |
ClinicalTrials.gov Identifier: NCT05514249 |
Recruitment Status :
Active, not recruiting
First Posted : August 24, 2022
Last Update Posted : September 1, 2022
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Duchenne Muscular Dystrophy | Drug: CRD-TMH-001 | Phase 1 |
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
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Drug: CRD-TMH-001
Participant will receive a single dose of CRD-TMH-001 administered via intravenous injection. |
- 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.
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.
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.
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 ClinicalTrials.gov identifier (NCT number): NCT05514249
United States, Massachusetts | |
UMass Chan Medical School | |
Worcester, Massachusetts, United States, 01655 |
Principal Investigator: | Brenda Wong, MD | UMass Chan Medical School | |
Study Director: | Medical Affairs | Cure Rare Disease |
Responsible Party: | Cure Rare Disease, Inc |
ClinicalTrials.gov 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 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Duchenne Muscular Dystrophy CRISPR gene-editing |
Muscular Dystrophies Muscular Dystrophy, Duchenne Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases |
Neuromuscular Diseases Nervous System Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked |