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Clinical trial

Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion

NCT03306277 is registered in ClinicalTrials.gov with Novartis Gene Therapies as lead sponsor.

NCT NCT03306277
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Registry record

As filed in ClinicalTrials.gov.

Title
Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion
Lead sponsor
Novartis Gene Therapies
Phase
Phase 3
Overall status
Completed
Start date
October 24, 2017
Completion date
November 12, 2019
Enrollment
22 (actual)
Sites
16

Conditions

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

gene therapy, sma - spinal muscular atrophy