Back to resultsTreatment of a Single Patient With CRD-TMH-001
Primary Purpose
Duchenne Muscular Dystrophy
Status
Active
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
CRD-TMH-001
Sponsored by
About this trial
This is an interventional treatment trial for Duchenne Muscular Dystrophy focused on measuring Duchenne, Muscular Dystrophy, CRISPR, gene-editing
Eligibility Criteria
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.
Sites / Locations
- UMass Chan Medical School
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Single patient
Arm Description
Single dose of CRD-TMH-001 administered by IV
Outcomes
Primary Outcome Measures
To assess the safety of CRD-TMH-001
To assess the safety and tolerability of the therapeutic by measuring both serious and non-serious adverse events.
Secondary Outcome Measures
Full Information
NCT ID
NCT05514249
First Posted
August 11, 2022
Last Updated
August 31, 2022
Sponsor
Cure Rare Disease, Inc
Collaborators
University of Massachusetts, Worcester
1. Study Identification
Unique Protocol Identification Number
NCT05514249
Brief Title
Treatment of a Single Patient With CRD-TMH-001
Official Title
Treatment of a Single Patient With CRD-TMH-001
Study Type
Interventional
2. Study Status
Record Verification Date
August 2022
Overall Recruitment Status
Active, not recruiting
Study Start Date
August 31, 2022 (Actual)
Primary Completion Date
September 2023 (Anticipated)
Study Completion Date
September 2023 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Cure Rare Disease, Inc
Collaborators
University of Massachusetts, Worcester
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Duchenne Muscular Dystrophy
Keywords
Duchenne, Muscular Dystrophy, CRISPR, gene-editing
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Masking Description
Single patient clinical trial
Allocation
N/A
Enrollment
1 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Single patient
Arm Type
Experimental
Arm Description
Single dose of CRD-TMH-001 administered by IV
Intervention Type
Drug
Intervention Name(s)
CRD-TMH-001
Intervention Description
Participant will receive a single dose of CRD-TMH-001 administered via intravenous injection.
Primary Outcome Measure Information:
Title
To assess the safety of CRD-TMH-001
Description
To assess the safety and tolerability of the therapeutic by measuring both serious and non-serious adverse events.
Time Frame
1 year
10. Eligibility
Sex
Male
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
28 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Brenda Wong, MD
Organizational Affiliation
UMass Chan Medical School
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Medical Affairs
Organizational Affiliation
Cure Rare Disease
Official's Role
Study Director
Facility Information:
Facility Name
UMass Chan Medical School
City
Worcester
State/Province
Massachusetts
ZIP/Postal Code
01655
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Treatment of a Single Patient With CRD-TMH-001
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