deltatrials
Completed PHASE3 INTERVENTIONAL 1-arm NCT03370913

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301) (BMN 270-301)

A Phase 3 Open-Label, Single-Arm Study To Evaluate The Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients With Residual FVIII Levels ≤ 1 IU/dL Receiving Prophylactic FVIII Infusions

Sponsor: BioMarin Pharmaceutical

Conditions Hemophilia A
Interventions valoctocogene roxaparvovec
Updated 34 times since 2017 Last updated: Mar 17, 2025 Started: Dec 19, 2017 Primary completion: Nov 16, 2020 Completion: Nov 20, 2024
This information is for research purposes only and is not medical advice. Consult a healthcare provider before making any medical decision.

Listed as NCT03370913, this PHASE3 trial focuses on Hemophilia A and remains completed. Sponsored by BioMarin Pharmaceutical, it has been updated 34 times since 2017, reflecting substantial change activity. This study adds to the evidence base for this therapeutic area through structured, versioned documentation.

Status Flow

~Dec 2017 – ~May 2018 · 5 months · monthly snapshot~May 2018 – ~Jun 2018 · 31 days · monthly snapshot~Jun 2018 – ~Jul 2018 · 30 days · monthly snapshot~Jul 2018 – ~Aug 2018 · 31 days · monthly snapshot~Aug 2018 – ~Sep 2018 · 31 days · monthly snapshot~Sep 2018 – ~Nov 2018 · 2 months · monthly snapshot~Nov 2018 – ~Dec 2018 · 30 days · monthly snapshot~Dec 2018 – ~Jan 2019 · 31 days · monthly snapshot~Jan 2019 – ~Feb 2019 · 31 days · monthly snapshot~Feb 2019 – ~Mar 2019 · 28 days · monthly snapshot~Mar 2019 – ~Jun 2019 · 3 months · monthly snapshot~Jun 2019 – ~Jul 2019 · 30 days · monthly snapshot~Jul 2019 – ~Aug 2019 · 31 days · monthly snapshot~Aug 2019 – ~Sep 2019 · 31 days · monthly snapshot~Sep 2019 – ~Oct 2019 · 30 days · monthly snapshot~Oct 2019 – ~Nov 2019 · 31 days · monthly snapshot~Nov 2019 – ~Dec 2019 · 30 days · monthly snapshot~Dec 2019 – ~Jan 2020 · 31 days · monthly snapshot~Jan 2020 – ~Aug 2020 · 7 months · monthly snapshot~Aug 2020 – ~Dec 2020 · 4 months · monthly snapshot~Dec 2020 – ~Jan 2021 · 31 days · monthly snapshot~Jan 2021 – ~Feb 2021 · 31 days · monthly snapshot~Feb 2021 – ~Nov 2021 · 9 months · monthly snapshot~Nov 2021 – ~Dec 2021 · 30 days · monthly snapshot~Dec 2021 – ~Apr 2022 · 4 months · monthly snapshot~Apr 2022 – ~Sep 2022 · 5 months · monthly snapshot~Sep 2022 – ~Jan 2023 · 4 months · monthly snapshot~Jan 2023 – ~Dec 2023 · 11 months · monthly snapshot~Dec 2023 – ~Jan 2024 · 31 days · monthly snapshot~Jan 2024 – ~Jul 2024 · 6 months · monthly snapshot~Jul 2024 – ~Sep 2024 · 2 months · monthly snapshot~Sep 2024 – ~Apr 2025 · 7 months · monthly snapshot~Apr 2025 – ~Sep 2025 · 5 months · monthly snapshot~Sep 2025 – present · 12 months · monthly snapshot

Change History

34 versions recorded

  1. Sep 2025 — Present [monthly]

    Completed PHASE3

  2. Apr 2025 — Sep 2025 [monthly]

    Completed PHASE3

    Status: Active Not Recruiting → Completed

  3. Sep 2024 — Apr 2025 [monthly]

    Active Not Recruiting PHASE3

  4. Jul 2024 — Sep 2024 [monthly]

    Active Not Recruiting PHASE3

  5. Jan 2024 — Jul 2024 [monthly]

    Active Not Recruiting PHASE3

▶ Show 29 earlier versions

  1. Dec 2023 — Jan 2024 [monthly]

    Active Not Recruiting PHASE3

  2. Jan 2023 — Dec 2023 [monthly]

    Active Not Recruiting PHASE3

  3. Sep 2022 — Jan 2023 [monthly]

    Active Not Recruiting PHASE3

  4. Apr 2022 — Sep 2022 [monthly]

    Active Not Recruiting PHASE3

  5. Dec 2021 — Apr 2022 [monthly]

    Active Not Recruiting PHASE3

  6. Nov 2021 — Dec 2021 [monthly]

    Active Not Recruiting PHASE3

  7. Feb 2021 — Nov 2021 [monthly]

    Active Not Recruiting PHASE3

  8. Jan 2021 — Feb 2021 [monthly]

    Active Not Recruiting PHASE3

  9. Dec 2020 — Jan 2021 [monthly]

    Active Not Recruiting PHASE3

  10. Aug 2020 — Dec 2020 [monthly]

    Active Not Recruiting PHASE3

  11. Jan 2020 — Aug 2020 [monthly]

    Active Not Recruiting PHASE3

    Status: Recruiting → Active Not Recruiting

  12. Dec 2019 — Jan 2020 [monthly]

    Recruiting PHASE3

  13. Nov 2019 — Dec 2019 [monthly]

    Recruiting PHASE3

  14. Oct 2019 — Nov 2019 [monthly]

    Recruiting PHASE3

  15. Sep 2019 — Oct 2019 [monthly]

    Recruiting PHASE3

  16. Aug 2019 — Sep 2019 [monthly]

    Recruiting PHASE3

  17. Jul 2019 — Aug 2019 [monthly]

    Recruiting PHASE3

  18. Jun 2019 — Jul 2019 [monthly]

    Recruiting PHASE3

  19. Mar 2019 — Jun 2019 [monthly]

    Recruiting PHASE3

  20. Feb 2019 — Mar 2019 [monthly]

    Recruiting PHASE3

  21. Jan 2019 — Feb 2019 [monthly]

    Recruiting PHASE3

  22. Dec 2018 — Jan 2019 [monthly]

    Recruiting PHASE3

  23. Nov 2018 — Dec 2018 [monthly]

    Recruiting PHASE3

  24. Sep 2018 — Nov 2018 [monthly]

    Recruiting PHASE3

  25. Aug 2018 — Sep 2018 [monthly]

    Recruiting PHASE3

  26. Jul 2018 — Aug 2018 [monthly]

    Recruiting PHASE3

  27. Jun 2018 — Jul 2018 [monthly]

    Recruiting PHASE3

  28. May 2018 — Jun 2018 [monthly]

    Recruiting PHASE3

  29. Dec 2017 — May 2018 [monthly]

    Recruiting PHASE3

    First recorded

Eligibility Summary

No eligibility information available.

Contact Information

Sponsor contact:
  • BioMarin Pharmaceutical
Data source: BioMarin Pharmaceutical

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

Study Locations

A Coruña, Spain , Adelaide, Australia , Ann Arbor, United States , Aurora, United States , Berlin, Germany , Birmingham, United Kingdom , Bonn, Germany , Brisbane, Australia , Cambridge, United Kingdom , Campinas, Brazil and 36 more locations