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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.


Found (99) 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 ...


The Effects of Core Stabilization Exercises in Addition to Balance Exercises in Adult Hemophilia Patients on Balance Quality of Life Lower Limb Muscle Strength and Functional Level

The aim of this study is to investigate the effects of core stabilization exercises in addition to balance exercises on balance, quality of life, lower extremity muscle strength and functional level in adult hemophilic patients.

Phase N/A

Patient Reported Outcomes Burdens and Experiences - Phase 3

BACKGROUND The PROBE questionnaire is a tool for the assessment of patient-reported outcome, burdens and experiences. Until now, it has been used in persons living with hemophilia (PWH) and healthy controls. In phase 1 of the study, the investigators developed the questionnaire. In phase 2 of the study, the investigators ...

Phase N/A

A Study of Fitusiran in Severe Hemophilia A and B Patients Previously Receiving Factor or Bypassing Agent Prophylaxis

Study duration per participant is approximately 13 months including 6-Month factor or BPA prophylaxis period and 7-Month fitusiran treatment period. Study duration for patients in the subgroup of Cohort A is approximately 7 months corresponding to a 7-Month fitusiran treatment period. Participants completing the treatment period will be proposed to ...


PF-06741086 Long-term Treatment in Severe Hemophilia

This study is designed to evaluate the safety, tolerability and efficacy of long-term treatment with PF-06741086 in subjects with severe hemophilia who participated in the 3-month Phase 1b/2 B7841002 study. Additionally, de novo subjects will be recruited into this study.


HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients

This is an open-label, single-dose, multi-center, multinational trial to demonstrate the efficacy of AMT-061 and to further describe its safety profile. The study drug is identified as AAV5-hFIXco-Padua (AMT- 061). AMT-061 is a recombinant adeno-associated viral vector of serotype 5 (AAV5) containing the Padua variant of a codon-optimized human FIX ...


A Survey on the Success of Inhibitor Elimination Using Individualized Concentrate Selection and Controlled ITI

As a result of many technological advances in the last two decades, current factor VIII (FVIII) concentrates (both plasma-derived and recombinant products) are considered very safe in terms of pathogen safety. The development of inhibitors against FVIII or factor IX (FIX) is considered as a major complication during replacement therapy ...

Phase N/A

Weight-based Dosing in Hemophilia A

The investigators propose a single center, randomized, controlled, open-label, crossover trial to determine if recombinant factor VIII (rFVIII) dosed according to lean body mass (LBM) and ideal body weight (IBW) achieves a targeted FVIII recovery with better precision than based on total body weight (TBW). The investigators hypothesize the use ...


Global Haemostatic Methods Following Administration of Bypassing Agents to Patients With Haemophilia With Inhibitors

Background The treatment of haemophilia A and B has been revolutionized by the use of factor concentrate, both as prophylaxis and to treat bleeding episodes (on-demand treatment). However, despite its advantages, repeated treatment with factor concentrate can lead to development of inhibitors (antibodies) towards the coagulation factor in the concentrate. ...

Phase N/A

UK - EHL Outcomes Registry

Severe haemophilia A and B (SHA, SHB) are inherited bleeding disorders affecting male patients and are characterised by low levels of circulating clotting factors VIII and IX respectively. Clinically low levels present with multiple recurrent bleeds into joints and muscle from the first couple of years of life. In addition ...

Phase N/A