Ignite Creation Date:
2024-05-06 @ 7:20 PM
Last Modification Date:
2024-10-26 @ 3:04 PM
Study NCT ID:
NCT05973630
Status:
NOT_YET_RECRUITING
Last Update Posted:
2024-03-07
First Post:
2023-07-24
Brief Title:
ATA-200 Dose-escalation Gene Therapy Trial in Patients with LGMDR5
Sponsor:
Atamyo Therapeutics
Organization:
Atamyo Therapeutics
Study Overview
Official Title:
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:
NOT_YET_RECRUITING
Status Verified Date:
2024-07
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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 2cR5 LGMD R5 Patients will be treated sequentially in 2 dose-cohorts
Detailed Description:
This is a multicenter Phase 1b assessing the safety and tolerability of 2 doses of ATA-200 for the treatment of LGMDR5
The dose escalation phase will enroll ambulant patients with LGMDR5 Two dose cohorts C1 and C2 will be enrolled sequentially and enrollment will be staggered with at least 4-week interval between 2 patient treatments An initial cohort C1 of three 3 patients will receive a potentially effective dose corresponding to the minimum effective dose MED in preclinical studies
Enrollment of three 3 patients in the 2nd higher dose cohort C2 with a 7-fold safety margin relative to the highest safe dose in the GLP toxicology study will be initiated following review of the one-month safety data post-administration in cohort C1 by an independent Data Safety Monitoring Board DSMB
Time point of primary interest for safety evaluation and dose selection for future studies is at 6 months
All subjects will be followed up for an additional 45 years after completion of the evaluation period
The dose escalation phase will enroll ambulant patients with LGMDR5 Two dose cohorts C1 and C2 will be enrolled sequentially and enrollment will be staggered with at least 4-week interval between 2 patient treatments An initial cohort C1 of three 3 patients will receive a potentially effective dose corresponding to the minimum effective dose MED in preclinical studies
Enrollment of three 3 patients in the 2nd higher dose cohort C2 with a 7-fold safety margin relative to the highest safe dose in the GLP toxicology study will be initiated following review of the one-month safety data post-administration in cohort C1 by an independent Data Safety Monitoring Board DSMB
Time point of primary interest for safety evaluation and dose selection for future studies is at 6 months
All subjects will be followed up for an additional 45 years after completion of the evaluation period
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None