Ignite Creation Date:
2024-05-06 @ 12:45 PM
Last Modification Date:
2024-10-26 @ 1:03 PM
Study NCT ID:
NCT03837184
Status:
COMPLETED
Last Update Posted:
2022-11-22
First Post:
2019-02-08
Brief Title:
Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies
Sponsor:
Novartis Gene Therapies
Organization:
Novartis
Study Overview
Official Title:
Phase 3 Open-Label Single-Arm Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion
Status:
COMPLETED
Status Verified Date:
2022-11
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:
This is a Phase 3 open-label single-arm single-dose trial of onasemnogene abeparvovec-xioi gene replacement therapy in participants with spinal muscular atrophy SMA Type 1 and who are genetically defined by a biallelic pathogenic mutation of the survival motor neuron 1 gene SMN1 with one or two copies of survival motor neuron 2 gene SMN2 The primary objective of the study is to evaluate the efficacy of onasemnogene abeparvovec-xioi by assessing the proportion of symptomatic SMA Type 1 participants who achieve the ability to sit unaided for at least 10 seconds up to and including the 18 months of age trial visit At least 6 participants aged 6 months 180 days at the time of gene replacement therapy Day 1 will be enrolled
Detailed Description:
This is a Phase 3 open-label single-arm single-dose trial of onasemnogene abeparvovec-xioi gene replacement therapy in participants with SMA Type 1 with one or 2 copies of SMN2 At least 6 participants 6 months 180 days of age at the time of gene replacement therapy Day 1 will be enrolled
The trial includes 3 trial periods screening gene replacement therapy and follow-up During the screening period Days -30 to -2 participants whose parentslegal guardians provide informed consent will undergo screening procedures to determine eligibility for trial enrollment participants who meet the entry criteria will enter the in-patient gene replacement therapy period Day -1 to Day 3 On Day -1 participants will be admitted to the hospital for pre-treatment baseline procedures On Day 1 participants will receive a one-time intravenous IV infusion of the equivalent of onasemnogene abeparvovec-xioi cohort 2 dose received in the AVXS-101-CL-101 trial over approximately 60 minutes and will undergo in-patient safety monitoring over the next 48 hours Participants may be discharged 48 hours after gene replacement therapy based on Investigator judgment During the outpatient follow-up period Days 4 to End of Trial at 18 months of age participants will return at regularly scheduled intervals for efficacy and safety assessments until the participant reaches 18 months of age
The trial includes 3 trial periods screening gene replacement therapy and follow-up During the screening period Days -30 to -2 participants whose parentslegal guardians provide informed consent will undergo screening procedures to determine eligibility for trial enrollment participants who meet the entry criteria will enter the in-patient gene replacement therapy period Day -1 to Day 3 On Day -1 participants will be admitted to the hospital for pre-treatment baseline procedures On Day 1 participants will receive a one-time intravenous IV infusion of the equivalent of onasemnogene abeparvovec-xioi cohort 2 dose received in the AVXS-101-CL-101 trial over approximately 60 minutes and will undergo in-patient safety monitoring over the next 48 hours Participants may be discharged 48 hours after gene replacement therapy based on Investigator judgment During the outpatient follow-up period Days 4 to End of Trial at 18 months of age participants will return at regularly scheduled intervals for efficacy and safety assessments until the participant reaches 18 months of age
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
True
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 194664 | OTHER | None | None |
| 201900208 | OTHER | None | None |
| COAV101A12304 | OTHER | Novartis Pharmaceuticals | None |