Ignite Creation Date:
2024-05-06 @ 5:11 PM
Last Modification Date:
2024-10-26 @ 2:23 PM
Study NCT ID:
NCT05224505
Status:
RECRUITING
Last Update Posted:
2023-04-06
First Post:
2021-12-15
Brief Title:
GNT0006 Gene Therapy Trial in Patients With LGMDR9
Sponsor:
Atamyo Therapeutics
Organization:
Atamyo Therapeutics
Study Overview
Official Title:
A Phase 1-2 Multicenter Study 2-stages to Evaluate the Safety and Efficacy of Intravenous GNT0006 Adeno-associated Viral Vector Carrying the FKRP Gene in Patients With FKRP-related Limb-girdle Muscular Dystrophy LGMDR9 Formerly LGMD2I
Status:
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:
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
Detailed Description:
Multicenter Phase 1-2 study evaluating safety pharmacodynamic efficacy and immunogenicity of GNT0006 an Adeno-Associated Virus AAV vector carrying the human FKRP transgene
This study will consist of 2 phases an open-label dose escalation phase Stage 1 and a double-blind placebo controlled randomized phase Stage 2 both with long-term follow-up LTFU period
Stage 1 Two dose cohorts will be enrolled sequentially and enrollment An initial cohort of three 3 patients will receive a potentially effective dose followed by a 2nd higher dose cohort of 3 patients
Stage 2 After selection of the effective dose in Stage 1 thirty-three 33 ambulant patients will be randomized at the optimal selected dose and followed up to the primary efficacy timepoint ie one year after investigational medicinal product IMP or placebo administration
At one-year post-IMP administration timepoint of primary interest for efficacy patients enrolled in placebo group will receive active IMP while patients randomized in the active IMP group will receive a placebo infusion
All subjects will be followed for up to 5 years after active IMP GNT0006 administration
This study will consist of 2 phases an open-label dose escalation phase Stage 1 and a double-blind placebo controlled randomized phase Stage 2 both with long-term follow-up LTFU period
Stage 1 Two dose cohorts will be enrolled sequentially and enrollment An initial cohort of three 3 patients will receive a potentially effective dose followed by a 2nd higher dose cohort of 3 patients
Stage 2 After selection of the effective dose in Stage 1 thirty-three 33 ambulant patients will be randomized at the optimal selected dose and followed up to the primary efficacy timepoint ie one year after investigational medicinal product IMP or placebo administration
At one-year post-IMP administration timepoint of primary interest for efficacy patients enrolled in placebo group will receive active IMP while patients randomized in the active IMP group will receive a placebo infusion
All subjects will be followed for up to 5 years after active IMP GNT0006 administration
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
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 2021-004276-33 | EUDRACT_NUMBER | None | None |