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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
GNT0006 Gene Therapy Trial in Patients With LGMDR9
NCTID
NCT05224505
(View at clinicaltrials.gov)
Description
Phase 1-2 study including a dose escalation safety and proof of concept phase (Stage 1, open label), followed by a double-blind, randomized, placebo-controlled confirmatory phase (Stage 2)
(Show More)
Development Status
Active
Indication
Limb-Girdle Muscular Dystrophy, Type 2I/R9
Disease Ontology Term
DOID:0110299
Compound Name
GNT0006
Compound Alias
ATA-100
Sponsor
Atamyo Therapeutics
Funder Type
Industry
Recruitment Status
Recruiting
Enrollment Count
39
Results Posted
Not Available
Therapy Information
Target Gene/Variant
FKRP
Therapy Type
Gene transfer
Therapy Route
In-vivo
Mechanism of Action
Functional gene replacement
Route of Administration
Intravenous
Drug Product Type
Viral vector
Target Tissue/Cell
Delivery System
Viral transduction
Vector Type
AAV2/9
Editor Type
none
Dose 1
9.0E12 vg/kg
Dose 2
2.7E13 vg/kg
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2021-12-15
Completion Date
2030-10
Last Update
2024-07-31
Participation Criteria
Eligible Age
16 Years - 99 Years
Standard Ages
Child, Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
3
Locations
Denmark,United Kingdom,France
Regulatory Information
Has US IND
False
FDA Designations
Fast Track
Recent Updates
Dose-escalation (Phase 1b) recruitment completed September 2024
Resources/Links
Clinical Publications
(Presentation) Preliminary Results from a Phase 1-2 Gene Therapy Study of ATA-100, AAV9 Vector Encoding FKRP, in Patients with Limb Girdle Muscular Dystrophy R9 - World Muscle Society 2024
News and Press Releases
Atamyo Therapeutics Observes LGMD Awareness Day with Updates on Key Milestones in its Clinical Development of Gene Therapies for Patients Suffering from Limb-Girdle Muscular Dystrophies
Preclinical Publications
AAV-mediated transfer of FKRP shows therapeutic efficacy in a murine model but requires control of gene expression