Ignite Creation Date:
2024-05-06 @ 11:13 AM
Last Modification Date:
2024-10-26 @ 12:42 PM
Study NCT ID:
NCT03461289
Status:
COMPLETED
Last Update Posted:
2022-08-16
First Post:
2018-03-04
Brief Title:
Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
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:
2022-08
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:
STRIVE-EU
Brief Summary:
Phase 3 open-label single-arm single-dose trial of onasemnogene abeparvovec-xioi gene replacement therapy in patients with spinal muscular atrophy SMA Type 1 who meet enrollment criteria and 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 Up to 30 patients 6 months 180 days of age at the time of gene replacement therapy Day 1 will be enrolled
Detailed Description:
Phase 3 open-label single-arm single-dose trial of onasemnogene abeparvovec-xioi gene replacement therapy in patients with spinal muscular atrophy SMA Type 1 who meet enrollment criteria and 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 30 patients 6 months 180 days of age at the time of gene replacement therapy Day 1 will be enrolled
The trial includes a screening period a gene replacement therapy period and a follow-up period During the screening period Days -30 to -2 patients whose parentslegal guardians provide informed consent will complete screening procedures to determine eligibility for trial enrollment Patients who meet the entry criteria will enter the in-patient gene replacement therapy period Day -1 to Day 3 On Day -1 patients will be admitted to the hospital for pre-treatment baseline procedures On Day 1 patients will receive a one-time intravenous IV infusion of onasemnogene abeparvovec-xioi and will undergo in-patient safety monitoring over the next 48 hours Patients may be discharged 48 hours after the infusion based on Investigator judgment During the outpatient follow-up period Days 4 to End of Trial at 18 months of age patients will return at regularly scheduled intervals for efficacy and safety assessments until the End of Trial when the patient reaches 18 months of age After the End of Trial visit eligible patients will be asked to participate into the long-term follow up trial
All post-treatment visits will be relative to the date on which gene replacement therapy is administered until the patient is 14 months of age after which they will be relevant to the patients date of birth
The trial includes a screening period a gene replacement therapy period and a follow-up period During the screening period Days -30 to -2 patients whose parentslegal guardians provide informed consent will complete screening procedures to determine eligibility for trial enrollment Patients who meet the entry criteria will enter the in-patient gene replacement therapy period Day -1 to Day 3 On Day -1 patients will be admitted to the hospital for pre-treatment baseline procedures On Day 1 patients will receive a one-time intravenous IV infusion of onasemnogene abeparvovec-xioi and will undergo in-patient safety monitoring over the next 48 hours Patients may be discharged 48 hours after the infusion based on Investigator judgment During the outpatient follow-up period Days 4 to End of Trial at 18 months of age patients will return at regularly scheduled intervals for efficacy and safety assessments until the End of Trial when the patient reaches 18 months of age After the End of Trial visit eligible patients will be asked to participate into the long-term follow up trial
All post-treatment visits will be relative to the date on which gene replacement therapy is administered until the patient is 14 months of age after which they will be relevant to the patients date of birth
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 |
|---|---|---|---|
| COAV101A12301 | OTHER | Novartis Pharmaceuticals | None |
| 2017-000266-29 | EUDRACT_NUMBER | None | None |