deltatrials
Completed PHASE3 INTERVENTIONAL 1-arm NCT03837184

Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies

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

Sponsor: Novartis Gene Therapies

Conditions Spinal Muscular Atrophy Type I
Interventions Onasemnogene Abeparvovec-xioi
Updated 21 times since 2019 Last updated: Jan 7, 2026 Started: May 31, 2019 Primary completion: Jun 29, 2021 Completion: Jun 29, 2021
This information is for research purposes only and is not medical advice. Consult a healthcare provider before making any medical decision.

A PHASE3 clinical study on Spinal Muscular Atrophy Type I, this trial is completed. The trial is conducted by Novartis Gene Therapies and has accumulated 21 data snapshots since 2019. Longitudinal tracking of this trial contributes to a broader understanding of treatment development timelines.

Status Flow

~Mar 2019 – ~May 2019 · 2 months · monthly snapshot~May 2019 – ~Jul 2019 · 2 months · monthly snapshot~Jul 2019 – ~Aug 2019 · 31 days · monthly snapshot~Aug 2019 – ~Sep 2019 · 31 days · monthly snapshot~Sep 2019 – ~Jan 2020 · 4 months · monthly snapshot~Jan 2020 – ~Feb 2020 · 31 days · monthly snapshot~Feb 2020 – ~Apr 2020 · 2 months · monthly snapshot~Apr 2020 – ~May 2020 · 30 days · monthly snapshot~May 2020 – ~Oct 2020 · 5 months · monthly snapshot~Oct 2020 – ~Dec 2020 · 2 months · monthly snapshot~Dec 2020 – ~Jan 2021 · 31 days · monthly snapshot~Jan 2021 – ~Apr 2021 · 3 months · monthly snapshot~Apr 2021 – ~Sep 2021 · 5 months · monthly snapshot~Sep 2021 – ~Feb 2022 · 5 months · monthly snapshot~Feb 2022 – ~Sep 2022 · 7 months · monthly snapshot~Sep 2022 – ~Dec 2022 · 3 months · monthly snapshot~Dec 2022 – ~Jul 2024 · 19 months · monthly snapshot~Jul 2024 – ~Sep 2024 · 2 months · monthly snapshot~Sep 2024 – ~Sep 2025 · 12 months · monthly snapshot~Sep 2025 – ~Feb 2026 · 5 months · monthly snapshot~Feb 2026 – present · 5 months · monthly snapshot

Change History

21 versions recorded

  1. Feb 2026 — Present [monthly]

    Completed PHASE3

  2. Sep 2025 — Feb 2026 [monthly]

    Completed PHASE3

  3. Sep 2024 — Sep 2025 [monthly]

    Completed PHASE3

  4. Jul 2024 — Sep 2024 [monthly]

    Completed PHASE3

  5. Dec 2022 — Jul 2024 [monthly]

    Completed PHASE3

▶ Show 16 earlier versions

  1. Sep 2022 — Dec 2022 [monthly]

    Completed PHASE3

  2. Feb 2022 — Sep 2022 [monthly]

    Completed PHASE3

  3. Sep 2021 — Feb 2022 [monthly]

    Completed PHASE3

    Status: Active Not Recruiting → Completed

  4. Apr 2021 — Sep 2021 [monthly]

    Active Not Recruiting PHASE3

  5. Jan 2021 — Apr 2021 [monthly]

    Active Not Recruiting PHASE3

  6. Dec 2020 — Jan 2021 [monthly]

    Active Not Recruiting PHASE3

  7. Oct 2020 — Dec 2020 [monthly]

    Active Not Recruiting PHASE3

    Status: Recruiting → Active Not Recruiting

  8. May 2020 — Oct 2020 [monthly]

    Recruiting PHASE3

  9. Apr 2020 — May 2020 [monthly]

    Recruiting PHASE3

  10. Feb 2020 — Apr 2020 [monthly]

    Recruiting PHASE3

  11. Jan 2020 — Feb 2020 [monthly]

    Recruiting PHASE3

  12. Sep 2019 — Jan 2020 [monthly]

    Recruiting PHASE3

  13. Aug 2019 — Sep 2019 [monthly]

    Recruiting PHASE3

  14. Jul 2019 — Aug 2019 [monthly]

    Recruiting PHASE3

    Status: Not Yet Recruiting → Recruiting

  15. May 2019 — Jul 2019 [monthly]

    Not Yet Recruiting PHASE3

  16. Mar 2019 — May 2019 [monthly]

    Not Yet Recruiting PHASE3

    First recorded

Eligibility Summary

No eligibility information available.

Contact Information

Sponsor contact:
  • Novartis Gene Therapies
  • PRA Health Sciences
Data source: Novartis

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

Study Locations