Home›Trial›NCT03421977
NCT03421977ACTIVE NOT RECRUITING

Long-Term Follow-up Study for Patients From AVXS-101-CL-101

Study Summary

This is a long term, safety follow up study of patients in the AVXS-101-CL-101 gene replacement therapy clinical trial for SMA Type 1 delivering onasemnogene abeparvovec-xioi. Patients will roll over from the parent study into this long-term study for continuous safety monitoring for up to 15 years.

Conditions
Spinal Muscular Atrophy 1
Interventions / Drugs
Onasemnogene Abeparvovec-xioi
View on ClinicalTrials.gov ↗
Sponsor
NVS
Novartis AG
Diversified Pharma
D36/100
Study Details
Enrollment13 participants
Primary Completion—
Lead SponsorNovartis Gene Therapies
Data Retrieved2026-04-11 02:55:49.152297+00:00
What This Trial Means for Biotech Investors
Trial Phase Context
Status & Expected Data

Enrollment is complete. The trial is in its follow-up or data collection phase — a readout may occur on or near the primary completion date.

Enrolled: 13 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.

NVS Full Pipeline →All Companies →
Not financial advice. BiotechSign data is sourced from ClinicalTrials.gov, SEC EDGAR, and other public databases for research and informational purposes only. It does not constitute investment recommendations. Always do your own due diligence before making investment decisions.