These trials completed more than 12 months ago and should have reported results.

These trials are not yet due to report results: some are ongoing, some completed within the past 12 months.

These trials have problematic data on the registry. Details why »

Status Trial ID Title Completion date Category
Ongoing 2017-000266-29 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 Inf... not-yet-due
Ongoing 2017-004087-35 A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2 not-yet-due
Other 2019-002611-26 A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101 not-yet-due
Trial is outside EEC, and reported results 2020-000095-38 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 Inf... bad-data
Trial is outside EEC 2020-001235-27 Phase I Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS-101 bad-data