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5Cases

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

2020-04-01 - 2026-09-01

Phase I/II

Active
A Phase I/II Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Healthy Volunteers and Patients with Hemophilia A
  • Condition/Disease

    Hemophilia A

  • Test Drug

    NXT007

Participate Sites
5Sites

Recruiting5Sites

2019-06-14 - 2021-11-30

Phase III

ATLAS-PPX: an open-label, multinational, switching study to describe the efficacy and safety of fitusiran prophylaxis in patients with hemophilia A and B previously receiving factor or bypassing agent prophylaxis.
  • Condition/Disease

    Acquired hemophilia

  • Test Drug

    Fitusiran

Participate Sites
6Sites

Recruiting4Sites

Terminated2Sites

2018-03-01 - 2020-05-31

Phase III

ATLAS-A/B: A Phase 3 Study to Evaluate the Efficacy and Safety of Fitusiran in Patients With Hemophilia A or B, Without Inhibitory Antibodies to Factor VIII or IX
  • Condition/Disease

    Hemophilia A or B

  • Test Drug

    Fitusiran (ALN-AT3SC)

Participate Sites
7Sites

Not yet recruiting1Sites

Recruiting6Sites