A Phase 1-2, Open-label, Dose Escalation Study to Evaluate the Safety of 2 Doses of Intravenous ATA-200, an Adeno-associated Viral Vector Carrying the Human SGCG Gene, in Patients With Gamma-sarcoglycanopathy (LGMDR5)
Status: Recruiting
Location: See all (3) locations...
Intervention Type: Biological
Study Type: Interventional
Study Phase: Phase 1/Phase 2
SUMMARY
The purpose of ATA-003-GSAR study is to evaluate the safety and tolerability of a single intravenous infusion of ATA-200 in pediatric patients with limb girdle muscular dystrophy type 2c/R5 (LGMD R5). Patients will be treated sequentially in 2 dose-cohorts.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 6
Maximum Age: 13
Healthy Volunteers: f
View:
• Confirmed diagnosis of LGMDR5 before age of 10, based on clinical presentation and genotyping
• Ambulant male or female patients aged 6 to less than 12 years of age at screening
• Able to perform the 10-meter walk test (10MWT) in less than 15 sec and to rise from chair with or without arm support
Locations
United States
Florida
Child Health Research Institute
RECRUITING
Gainesville
Other Locations
France
Hopital Trousseau
NOT_YET_RECRUITING
Paris
Italy
Ospedale Maggiore Policlinico
NOT_YET_RECRUITING
Milan
Contact Information
Primary
Sophie Olivier
s.olivier@atamyo.com
+33638682337
Time Frame
Start Date: 2025-02-15
Estimated Completion Date: 2031-01-31
Participants
Target number of participants: 6
Treatments
Experimental: Cohort 1
ATA-200 Dose level 1: 1.0E+14 vg/Kg, solution for injection, single IV infusion over 2h
Experimental: Cohort 2
ATA-200 Dose level 2: 3.0E+14 vg/Kg, solution for injection, single IV infusion over 2h
Related Therapeutic Areas
Sponsors
Leads: Atamyo Therapeutics