Ignite Creation Date:
2024-05-06 @ 2:47 AM
Last Modification Date:
2024-10-26 @ 11:23 AM
Study NCT ID:
NCT02122952
Status:
COMPLETED
Last Update Posted:
2022-09-15
First Post:
2014-04-23
Brief Title:
Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1
Sponsor:
Novartis Gene Therapies
Organization:
Novartis
Study Overview
Official Title:
Phase I Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS-101
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:
None
Brief Summary:
The purpose of this trial is to evaluate safety and efficacy of intravenous delivery of AVXS-101 as a treatment of spinal muscular atrophy Type 1 SMN1
Detailed Description:
The study will evaluate safety and efficacy of gene therapy in spinal muscular atrophy Type 1 SMA1 patients SMA is caused by low levels of the survival motor neuron SMN protein and affects all muscles in the body There is no effective treatment for SMA and current drug therapy has been unsuccessful in stabilizing or reversing this disease Only supportive care is currently possible
Open-label dose-escalation clinical trial of AVXS-101 injected intravenously through a peripheral limb vein Short-term safety will be evaluated over a two year period Patients will be tested at baseline and return for follow up visits on days 7 14 21 30 followed by once every month through 12 months post dose and then every three months through two 2 years post infusion Unscheduled visits may occur if the PI determines that they are necessary
The primary analysis for efficacy will be assessed when all patients reach 136 months of age a database lock will be performed at the time point at which all patients reach 136 months of age A follow-up safety analysis will be completed at the time point at which the last patient reaches 24 months post-dose
Upon completion of the 2-year study period patients will be monitored annually as per standard of care for up to 15 years
Open-label dose-escalation clinical trial of AVXS-101 injected intravenously through a peripheral limb vein Short-term safety will be evaluated over a two year period Patients will be tested at baseline and return for follow up visits on days 7 14 21 30 followed by once every month through 12 months post dose and then every three months through two 2 years post infusion Unscheduled visits may occur if the PI determines that they are necessary
The primary analysis for efficacy will be assessed when all patients reach 136 months of age a database lock will be performed at the time point at which all patients reach 136 months of age A follow-up safety analysis will be completed at the time point at which the last patient reaches 24 months post-dose
Upon completion of the 2-year study period patients will be monitored annually as per standard of care for up to 15 years
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
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 |
|---|---|---|---|
| COAV101A12101 | OTHER | Novartis Pharmaceuticals | None |