Ignite Creation Date:
2024-05-06 @ 4:44 PM
Last Modification Date:
2024-10-26 @ 2:15 PM
Study NCT ID:
NCT05073133
Status:
COMPLETED
Last Update Posted:
2024-05-31
First Post:
2021-09-20
Brief Title:
Safety and Efficacy of Intravenous OAV101 AVXS-101 in Pediatric Patients With Spinal Muscular Atrophy SMA OFELIA
Sponsor:
Novartis Pharmaceuticals
Organization:
Novartis
Study Overview
Official Title:
A Phase IV Open-label Single-arm Single-dose Multicenter Study to Evaluate the saFEty toLerability and effIcacy of Gene Replacement Therapy With intravenousOAV101AVXS101 in Pediatric Patients From Latin America With Spinal Muscular Atrophy SMA - OFELIA
Status:
COMPLETED
Status Verified Date:
2024-10
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:
OFELIA
Brief Summary:
This was a phase IV Open-label single-arm single-dose multicenter study to evaluate the safety tolerability and efficacy of intravenous administration of OAV101 AVXS-101 in patients with SMA with bi-allelic mutations in the survival motor neuron 1 SMN1 gene 24 months and weighing 17 kg over a 18-month period post infusion
Detailed Description:
This is an open-label single arm multi-center study to evaluate the safety tolerability and efficacy of IV OAV101 in symptomatic SMA pediatric participants The study enrolled participants 24 months old that weigh 17 kg Participants who met eligibility criteria at Screening and Baseline visits received a single dose of IV OAV101 t the approved dose of 11e14 vgkg and were followed for 18 months The study included a 20-day screening period in which there were 2 Screening visits during which eligibility was assessed Screening 1 weight was collected for dose calculation Screening 2 and baseline assessments were performed prior to treatment
On Day -1 participants were admitted to the hospital for pre-treatment baseline procedures including prednisolone treatment per study protocol On Day 1 participants received a single IV infusion of OAV101 Participants were discharged 12-48 hours after the infusion based on Investigator judgment Safety monitoring was performed on an ongoing basis per protocol requirement and was evaluated by the clinical safety team as well as DMC Data monitoring committee
On Day -1 participants were admitted to the hospital for pre-treatment baseline procedures including prednisolone treatment per study protocol On Day 1 participants received a single IV infusion of OAV101 Participants were discharged 12-48 hours after the infusion based on Investigator judgment Safety monitoring was performed on an ongoing basis per protocol requirement and was evaluated by the clinical safety team as well as DMC Data monitoring committee
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 |
|---|---|---|---|
| 2023-000864-67 | EUDRACT_NUMBER | None | None |