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Hemophilia Clinical Trials

A listing of Hemophilia medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

RESULTS

Found (22) clinical trials

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg

This clinical trial is being conducted to learn more about a potential treatment (valoctocogene roxaparvovec) for people with severe hemophilia A. This research study will test and confirm the safety and effectiveness of the 4E13 vg/kg dose of the study drug (valoctocogene roxaparvovec) that contains the correct gene to make ...

Phase

BAX 855 Previously Untreated Patient (PUP)

The purpose of this study is to investigate safety, immunogenicity and hemostatic efficacy of PEGylated recombinant FVIII (BAX 855) in previously untreated patients (PUPs) < 6 years of age with severe hemophilia A (baseline FVIII level < 1%) and < 3 EDs to ADVATE, BAX 855 or plasma transfusion.

Phase

Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Previously Untreated Patients With Haemophilia B

This trial is conducted globally. The aim of the trial is to investigate the safety and efficacy of nonacog beta pegol (N9-GP) in previously untreated patients with Haemophilia B.

Phase

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients

This clinical trial is being conducted to learn more about a potential treatment (valoctocogene roxaparvovec) for people with severe hemophilia A. This research study will test and confirm the safety and effectiveness of the 6E13 vg/kg dose of the study drug (valoctocogene roxaparvovec) that contains the correct gene to make ...

Phase

A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors

The purpose of this study is to determine the frequency of bleeding episodes in patients receiving fitusiran as prophylactic treatment of hemophilia compared with patients who are assigned to continue with their regular medication. In addition, the study will assess safety, quality of life, pharmacodynamics (PD), and pharmacokinetics (PK).

Phase

rVWF IN PROPHYLAXIS

The purpose of this phase 3 study is to investigate the efficacy and safety, including immunogenicity and thrombogenicity of prophylactic treatment with recombinant von Willebrand factor (rVWF) in subjects with severe von Willebrand disease (VWD).

Phase

Trial of Acquired Haemophilia With Steroid Combined With Cyclophosphamide Versus Steroid Combined With Rituximab

This is a prospective randomized multi-center controlled pilot trial comparing the regimen of steroid with rituximab and steroid with cyclophosphamide to eradicate anti-factor VIII antibodies in Chinese patients with acquired hemophilia A. Patients will be randomized to two regimens: methylprednisolone 0.8mg/kg/day (or equivalent corticosteroid doses) for 3 weeks (then tapering ...

Phase

BAX 111 rVWF in Pediatrics

The purpose of this study in pediatric participants (<18 years of age) with severe hereditary von Willebrand disease (VWD) is: To assess the efficacy, safety, and tolerability of recombinant von Willebrand Factor (rVWF), with or without ADVATE, in the treatment and control of nonsurgical bleeding events To assess the efficacy ...

Phase

Study to Determine the Safety and Efficacy of rFIXFc in Untreated Males With Severe Hemophilia B

The primary objective of the study is to evaluate the safety of rFIXFc (recombinant coagulation factor IX Fc fusion protein, BIIB029) in previously untreated participants with severe hemophilia B. Secondary objectives are to evaluate the efficacy of rFIXFc in the prevention and treatment of bleeding episodes in previously untreated participants ...

Phase

Prospective Biomarkers of Bone Metabolism in Hemophilia A

This is a pilot study to determine the impact of factor replacement on bone biomarkers in up to 20 hemophilia A subjects. Subjects will be recruited over 1 year for the 5-day protocol. Following a 72-hour washout period, factor levels and bone biomarkers will be followed before and after 50 ...

Phase