Active Not Recruiting PHASE3 INTERVENTIONAL 1-arm NCT04042025

Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi

A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101

Sponsor: Novartis Gene Therapies

Conditions SMA Spinal Muscular Atrophy Type I Spinal Muscular Atrophy Type II Spinal Muscular Atrophy Type III

Interventions Onasemnogene Abeparvovec-xioi

Updated 26 times since 2019 Last updated: Apr 23, 2025 Started: Feb 10, 2020 Primary completion: Dec 31, 2035 Completion: Dec 31, 2035

This information is for research purposes only and is not medical advice. Consult a healthcare provider before making any medical decision.

Listed as NCT04042025, this PHASE3 trial focuses on SMA and Spinal Muscular Atrophy Type I and remains ongoing. Sponsored by Novartis Gene Therapies, it has been updated 26 times since 2020, reflecting substantial change activity. This study adds to the evidence base for this therapeutic area through structured, versioned documentation.

Status Flow

Change History

26 versions recorded

Sep 2025 — Present [monthly]

Active Not Recruiting PHASE3
May 2025 — Sep 2025 [monthly]

Active Not Recruiting PHASE3
Sep 2024 — May 2025 [monthly]

Active Not Recruiting PHASE3
Jul 2024 — Sep 2024 [monthly]

Active Not Recruiting PHASE3
Mar 2024 — Jul 2024 [monthly]

Active Not Recruiting PHASE3

▶ Show 21 earlier versions

Aug 2023 — Mar 2024 [monthly]

Active Not Recruiting PHASE3
Jan 2023 — Aug 2023 [monthly]

Active Not Recruiting PHASE3

Status: Enrolling By Invitation → Active Not Recruiting
Dec 2022 — Jan 2023 [monthly]

Enrolling By Invitation PHASE3
Sep 2022 — Dec 2022 [monthly]

Enrolling By Invitation PHASE3

Phase: PHASE4 → PHASE3
Sep 2021 — Sep 2022 [monthly]

Enrolling By Invitation PHASE4
Apr 2021 — Sep 2021 [monthly]

Enrolling By Invitation PHASE4
Mar 2021 — Apr 2021 [monthly]

Enrolling By Invitation PHASE4
Jan 2021 — Mar 2021 [monthly]

Enrolling By Invitation PHASE4
Dec 2020 — Jan 2021 [monthly]

Enrolling By Invitation PHASE4
Nov 2020 — Dec 2020 [monthly]

Enrolling By Invitation PHASE4
Oct 2020 — Nov 2020 [monthly]

Enrolling By Invitation PHASE4
Sep 2020 — Oct 2020 [monthly]

Enrolling By Invitation PHASE4
Aug 2020 — Sep 2020 [monthly]

Enrolling By Invitation PHASE4
Jul 2020 — Aug 2020 [monthly]

Enrolling By Invitation PHASE4
May 2020 — Jul 2020 [monthly]

Enrolling By Invitation PHASE4

Status: Recruiting → Enrolling By Invitation
Apr 2020 — May 2020 [monthly]

Recruiting PHASE4
Feb 2020 — Apr 2020 [monthly]

Recruiting PHASE4
Jan 2020 — Feb 2020 [monthly]

Recruiting PHASE4

Status: Not Yet Recruiting → Recruiting
Nov 2019 — Jan 2020 [monthly]

Not Yet Recruiting PHASE4
Oct 2019 — Nov 2019 [monthly]

Not Yet Recruiting PHASE4
Sep 2019 — Oct 2019 [monthly]

Not Yet Recruiting PHASE4

First recorded

Eligibility Summary

No eligibility information available.

Contact Information

Sponsor contact:

Novartis Gene Therapies

Data source: Novartis

For direct contact, visit the study record on ClinicalTrials.gov .

Study Locations

Aurora, United States , Baltimore, United States , Boston, United States , Chicago, United States , Columbus, United States , Dallas, United States , Durham, United States , Genova, Italy , Ghent, Belgium , Grand Rapids, United States and 19 more locations