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Clinical trial

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)

NCT04851873 is registered in ClinicalTrials.gov with Novartis Pharmaceuticals as lead sponsor.

NCT NCT04851873
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Registry record

As filed in ClinicalTrials.gov.

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)
Lead sponsor
Novartis Pharmaceuticals
Phase
Phase 3
Overall status
Completed
Start date
September 8, 2021
Completion date
June 13, 2023
Enrollment
24 (actual)
Sites
13

Conditions

As the registry lists them; a condition with its own page is linked.

spinal muscular atrophy