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18Cases

2021-12-01 - 2025-12-31

Phase III

Active
  • Condition/Disease

  • Test Drug

Participate Sites
5Sites

Not yet recruiting3Sites

Recruiting2Sites

2023-10-01 - 2028-10-07

Phase IV

Active
  • Condition/Disease

  • Test Drug

Participate Sites
1Sites

Recruiting1Sites

2023-03-01 - 2026-12-31

Phase III

Active
  • Condition/Disease

  • Test Drug

Participate Sites
3Sites

Recruiting3Sites

2022-11-15 - 2028-11-26

Phase III

Active
  • Condition/Disease

  • Test Drug

Participate Sites
3Sites

Recruiting3Sites

2019-09-01 - 2027-12-31

Phase III

Active
Phase 3, open label, single arm study to evaluate efficacy and safety of FIX gene transfer with PF-06838435 (rAAV-Spark100-hFIX-Padua) in adult male participants with moderately severe to severe hemophilia B (FIX:C≤2%) (BeneGene-2)
  • Condition/Disease

    The study compared the differences between the trial drug before and after infusion, and evaluated the non-inferiority of the annual average bleeding rate (ABR) of total bleeding events (treatment-free and treatment-free) from week 12 to month 15 compared with standard of care (SOC) FIX prophylactic alternative therapy.

  • Test Drug

    injection

Participate Sites
5Sites

Recruiting5Sites

2019-10-30 - 2026-11-30

Phase III

Completed
  • Condition/Disease

  • Test Drug

Participate Sites
7Sites

Recruiting7Sites

2024-05-01 - 2032-12-31

Phase III

Active
  • Condition/Disease

  • Test Drug

Participate Sites
5Sites

Recruiting5Sites

2023-05-09 - 2026-07-31

Phase II

Completed
xxxxxx
  • Condition/Disease

    xxxxxx

  • Test Drug

    243 270

Participate Sites
4Sites

Recruiting4Sites

2021-08-01 - 2027-10-27

Phase I/II

Active
  • Condition/Disease

  • Test Drug

Participate Sites
3Sites

Recruiting3Sites

2024-05-01 - 2039-02-01

Phase IV

Not yet recruiting
A Long-Term Follow-Up Study in Subjects With Severe Hemophilia A Who Received BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in a Prior BioMarin Clinical Trial
  • Condition/Disease

    Hemophilia A

  • Test Drug

    BMN 270

Participate Sites
5Sites

Recruiting5Sites

1 2