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NCT05335876Phase 3RECRUITING

Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials

Study Summary

This is a global, prospective, multi-center study that is designed to assess the long-term safety and efficacy of OAV101 in patients who participated in an OAV101 clinical trial. The assessments of safety and efficacy in Study COAV101A12308 will continue for 5 years after enrollment in this study.

Conditions
Spinal Muscular Atrophy (SMA)
Interventions / Drugs
onasemnogene abeparvovec
View on ClinicalTrials.gov ↗
Sponsor
NVS
Novartis AG
Diversified Pharma
D36/100
Study Details
Enrollment175 participants
Primary Completion—
Lead SponsorNovartis Pharmaceuticals
Data Retrieved2026-04-11 02:55:49.152075+00:00
What This Trial Means for Biotech Investors
Trial Phase Context

Phase 3 trials are pivotal studies with large patient populations (hundreds to thousands) designed to prove efficacy and safety for FDA submission. A successful Phase 3 readout is typically the last major hurdle before a New Drug Application (NDA) or BLA filing.

Status & Expected Data

Currently recruiting: the trial is enrolling patients. A data readout is not expected until after enrollment closes and the follow-up period is complete.

Enrolled: 175 participants
Sponsor Catalyst Profile

Novartis AG (NVS) is the sponsoring company for this trial. BiotechSign currently grades this company D (36/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.

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