Ignite Creation Date:
2024-05-06 @ 4:03 PM
Last Modification Date:
2024-10-26 @ 2:02 PM
Study NCT ID:
NCT04851873
Status:
COMPLETED
Last Update Posted:
2024-01-05
First Post:
2021-04-06
Brief Title:
Safety and Efficacy of Intravenous OAV101 AVXS-101 in Pediatric Patients With Spinal Muscular Atrophy SMA
Sponsor:
Novartis Pharmaceuticals
Organization:
Novartis
Study Overview
Official Title:
A Phase IIIb Open-label Single-arm Single-dose Multicenter Study to Evaluate the Safety Tolerability and Efficacy of Gene Replacement Therapy With Intravenous OAV101 AVXS-101 in Pediatric Patients With Spinal Muscular Atrophy SMA
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:
SMART
Brief Summary:
To evaluate the safety tolerability and efficacy of intravenous administration of OAV101 AVXS-101 in patients with spinal muscular atrophy SMA with bi-allelic mutations in the survival motor neuron 1 SMN1 gene weighing 85 kg and 21 kg over a 12 month period
Detailed Description:
This was an open-label single arm multi-center study designed to evaluate the safety tolerability and efficacy of OAV101 in participants with SMA who weigh 85 kg and 21 kg The study aimed to enroll approximately 24 to 30 participants with approximately 6 to 10 participants across each of 3 weight brackets 85 to 13 kg 13 to 17 kg 17 to 21 kg
Eligible participants received a single administration of OAV101 at the approved dose of 11e14 vgkg on Day 1 Treatment period and were followed for a period of 12 months
Participants were admitted to the hospital on Day -1 for pre-treatment baseline procedures After receiving OAV101 on Day 1 participants underwent in-patient safety monitoring over the next 48 hours after which the participant could be discharged based on Investigator judgment
After study completion eligible participants could enroll into a Long Term follow-up study to collect additional safety and efficacy data COAV101A12308 NCT05335876 httpsclassicclinicaltrialsgovct2showNCT05335876termCOAV101A12308draw2rank1
Eligible participants received a single administration of OAV101 at the approved dose of 11e14 vgkg on Day 1 Treatment period and were followed for a period of 12 months
Participants were admitted to the hospital on Day -1 for pre-treatment baseline procedures After receiving OAV101 on Day 1 participants underwent in-patient safety monitoring over the next 48 hours after which the participant could be discharged based on Investigator judgment
After study completion eligible participants could enroll into a Long Term follow-up study to collect additional safety and efficacy data COAV101A12308 NCT05335876 httpsclassicclinicaltrialsgovct2showNCT05335876termCOAV101A12308draw2rank1
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?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 2020-005995-37 | EUDRACT_NUMBER | None | None |