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Clinical trial

A Phase IV Open-label, Single-arm, Single-dose, Multicenter Study to Evaluate the saFEty, toLerability and effIcacy of Gene Replacement Therapy With intravenousOAV101(AVXS101) in Pediatric Patients From Latin America With Spinal Muscular Atrophy (SMA) - OFELIA

NCT05073133 is registered in ClinicalTrials.gov with Novartis Pharmaceuticals as lead sponsor.

NCT NCT05073133
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Registry record

As filed in ClinicalTrials.gov.

Title
A Phase IV Open-label, Single-arm, Single-dose, Multicenter Study to Evaluate the saFEty, toLerability and effIcacy of Gene Replacement Therapy With intravenousOAV101(AVXS101) in Pediatric Patients From Latin America With Spinal Muscular Atrophy (SMA) - OFELIA
Lead sponsor
Novartis Pharmaceuticals
Phase
Phase 4
Overall status
Completed
Start date
November 4, 2021
Completion date
August 8, 2023
Enrollment
16 (actual)
Sites
5

Conditions

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

muscular atrophy, spinal