Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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UNKNOWN
PHASE1
1 participants
INTERVENTIONAL
2022-08-31
2023-09-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Single patient
Single dose of CRD-TMH-001 administered by IV
CRD-TMH-001
Participant will receive a single dose of CRD-TMH-001 administered via intravenous injection.
Interventions
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CRD-TMH-001
Participant will receive a single dose of CRD-TMH-001 administered via intravenous injection.
Eligibility Criteria
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Inclusion Criteria
* Confirmation of genetic mutation
* Confirmation of absence of elevated AAV9 NAbs
Exclusion Criteria
18 Years
28 Years
MALE
No
Sponsors
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University of Massachusetts, Worcester
OTHER
Cure Rare Disease, Inc
OTHER
Responsible Party
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Principal Investigators
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Brenda Wong, MD
Role: PRINCIPAL_INVESTIGATOR
UMass Chan Medical School
Medical Affairs
Role: STUDY_DIRECTOR
Cure Rare Disease
Locations
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UMass Chan Medical School
Worcester, Massachusetts, United States
Countries
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Other Identifiers
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CS-CRD-TMH-001
Identifier Type: -
Identifier Source: org_study_id
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