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Clinical Trial Report
Gene Therapy Trial Report
Summary
Gene Therapy for IGHMBP2-Related Diseases
NCTID
NCT05152823
(View at clinicaltrials.gov)
Description
Open-label, single intrathecal injection study of a AAV9 vector carrying the IGHMBP2 gene for IGHMBP2-related diseases.
(Show More)
Development Status
Active
Indication
Spinal Muscular Atrophy with Respiratory Distress
Disease Ontology Term
DOID:0111064
Compound Name
AAV9-IGHMBP2
Sponsor
Megan Waldrop
Funder Type
Other
Recruitment Status
Enrolling by invitation
Enrollment Count
10
Results Posted
Not Available
Therapy Information
Target Gene/Variant
IGHMBP2
Therapy Type
Gene transfer
Therapy Route
In-vivo
Mechanism of Action
Functional gene replacement
Route of Administration
Intrathecal
Drug Product Type
Viral vector
Target Tissue/Cell
Delivery System
Viral transduction
Vector Type
AAV9
Editor Type
Dose 1
Undisclosed
Dose 2
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2021-11-25
Completion Date
2028-11
Last Update
2024-08-21
Participation Criteria
Eligible Age
2 Months - 14 Years
Standard Ages
Child
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
1
Locations
United States
Regulatory Information
Has US IND
True
FDA Designations
Recent Updates
Resources/Links
Preclinical Publications
In Vitro Modeling as a Tool for Testing Therapeutics for Spinal Muscular Atrophy and IGHMBP2-Related Disorders