Clinical trials: Hemophilia A, Severe
Hemophilia A
A Post Approval Commitment Study to Gain More Information on How Safe and Effective KOVALTRY is in Chinese Children, Adolescents /Adults With Severe Hemophilia A
The goal of this study is to gather more information on safety and efficacy of Kovaltry for the prevention and treatment of bleeds in Chinese children, adolescents/adults with severe hemophilia A.
- Trial name
- NCT04565236
- Testing
- Recombinant Factor VIII (Kovaltry, BAY81-8973) Treatment Group 1, Recombinant Factor VIII (Kovaltry, BAY81-8973) Treatment Group 2, Recombinant Factor VIII (Kovaltry, BAY81-8973) Treatment Group 3
- Where
- 10 sites in China
- Volunteers
- 45 people
- Sponsor
- Bayer
Hemophilia A
Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 (DTX 201), a Drug Therapy That Delivers a Healthy Version of the Defective Factor VIII Gene Into the Nucleus of Liver Cells Using an Altered, Non-infectious Virus (AAV) as a "Shuttle"
In this study researchers want to gather more information about safety and effectiveness of BAY 2599023 (DTX201), a drug therapy that delivers the human factor VIII gene into the human body by use of a viral vector to treat the…
- Trial name
- NCT03588299
- Testing
- BAY2599023 (DTX201)
- Where
- 13 sites in United States, Bulgaria and 4 more
- Volunteers
- 11 people
- Sponsor
- Bayer
Hemophilia B
Study of AAV5-hFIX in Severe or Moderately Severe Haemophilia B
This is an open-label, extension study enrolling patients who have successfully completed all assessments in Study CT-AMT-060-01 (Years 1-5).
- Trial name
- NCT05360706
- Testing
- AAV5-hFIX
- Where
- 6 sites in Germany, Netherlands
- Volunteers
- 9 people
- Sponsor
- CSL Behring
Hemophilia A
BAX 855 Dose-Escalation Safety Study
The objectives of this study are to assess the tolerability and safety after single dose treatments of BAX 855 in previously treated patients (PTPs) with severe hemophilia A, to determine the pharmacokinetic (PK) parameters of…
- Trial name
- NCT01599819
- Testing
- Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method, PEGylated Recombinant Factor VIII
- Where
- 8 sites in Bulgaria, Germany and 2 more
- Volunteers
- 19 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A, Severe Hemophilia A
An Open-label Safety and Efficacy Study of Recombinant FVIII in Patients With Severe Hemophilia A
This multicenter, open-label, phase 3 extension study will investigate the safety and efficacy of rVIII-SingleChain for prophylaxis and on-demand treatment of bleeding episodes in at least 200 previously treated patients (PTPs)…
- Trial name
- NCT02172950
- Testing
- rVIII-SingleChain
- Where
- 64 sites in United States, Australia and 23 more
- Volunteers
- 246 people
- Sponsor
- CSL Behring
Hemophilia B
Study of Recombinant Factor IX Product, IB1001, in Previously Treated Subjects With Hemophilia B
To evaluate the safety (acute adverse effects associated with infusions, and inhibitor development), pharmacokinetics (PK), and efficacy with respect to breakthrough bleeding and control of hemorrhaging during prophylaxis of…
- Trial name
- NCT02048111
- Testing
- IB1001
- Where
- 2 sites in United States, United Kingdom
- Sponsor
- Cangene Corporation
Hemophilia A
BAY94-9027 PK Study Comparing to Another Long Acting Product
The purpose of this study is to compare the pharmacokinetics of BAY94-9027 and Elocta after intravenous administration.
- Trial name
- NCT03364998
- Testing
- Damoctocog (Jivi, BAY94-9027), Elocta
- Where
- Bulgaria
- Volunteers
- 18 people
- Sponsor
- Bayer
Severe Hemophilia A With an Inhibitor
The FEIBA NovoSeven Comparative Study
FENOC is a prospective, open-label, randomized, cross-over, multi-center study to investigate and compare the hemostatic effect and cost-efficacy of two different by-passing agents in the treatment of joint hemorrhages in…
- Trial name
- NCT00166309
- Testing
- FEIBA and NovoSeven
- Where
- Sweden
- Volunteers
- 60 people
- Sponsor
- Skane University Hospital
Hemophilia B
Hemophilia B Gene Therapy With AAV8 Vector
Hemophilia B is a bleeding disease in males due to very low levels of coagulation factor IX (FIX) in the blood. The current treatment is intravenous injection of FIX clotting factor concentrates, in response to bleeding.
- Trial name
- NCT01620801
- Testing
- AAV8-hFIX19
- Where
- 3 sites in United States, Australia
- Volunteers
- 4 people
- Sponsor
- Spark Therapeutics, Inc.
Hemophilia A, Hemophilia B
A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B
The purpose of this clinical trial is to learn about the safety and effects of the study medicine (called marstacimab) for the potential treatment of hemophilia in pediatric patients.
- Trial name
- BASIS KIDS (NCT05611801)
- Testing
- marstacimab
- Where
- 63 sites in United States, Argentina and 21 more
- Volunteers
- 100 people
- Sponsor
- Pfizer
Von Willebrand Disease
A Study to Compare the Pharmacokinetics and Safety of Current Factor VIII Concentrate and Optivate® in Haemophilia A.
The main objective of the study is to compare the pharmacokinetics of Optivate® with the subject's current FVIII concentrate when given as a bolus dose of 50IU/kg.
- Trial name
- NCT02246881
- Testing
- Optivate® (Human Coagulation Factor VIII)
- Where
- 8 sites in Poland, United Kingdom
- Sponsor
- Bio Products Laboratory
Cystic Fibrosis, Hemophilia A
Medication Adherence and Non-adherence in Adults With Rare Disease
The purpose of this study is to use the Medication Adherence Reasons Scale (MAR-Scale) to determine the extent of non-adherence to specific medications indicated to treat cystic fibrosis, hemophilia (A or B), idiopathic pulmonary…
- Conditions
- Cystic Fibrosis, Hemophilia A, Hemophilia B, Idiopathic Pulmonary Fibrosis
- Trial name
- NCT04541875
- Testing
- MAR-Scale
- Volunteers
- healthy volunteers welcome
- Sponsor
- Xperiome
Severe Forms of Hemophilia, Other Constitutional Bleeding Disorders
Input of a Multidisciplinary Device to Accompany the Announce of Diagnosis for Young Children With Serious Constitutional Hemorrhagic Disease and Their Families
Severe forms of hemophilia and other constitutional bleeding disorders represent a group of rare diseases. In last decades, news therapies have dramatically increased life expectancy and joint's protection.
- Trial name
- ACADHEM (NCT02834130)
- Testing
- Evaluation of psychological and psychopathological
- Where
- France
- Volunteers
- 30 people
- Sponsor
- Assistance Publique Hopitaux De Marseille
Hemophilia A
Biomarkers Serum Collection Methodology Pilot Study
The purpose of this study is to compare the standard collection of whole blood in tiger top tubes for serum collection versus a collection method of added FVIII to whole blood samples in the hemophilia population for routine…
- Trial name
- NCT01318707
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Georgetown University
Hemophilia A
Single Dose Study in Subjects With Severe Hemophilia A Comparing Pharmacokinetic Parameters for BAY81-8973 and Advate
The purpose of this study is to compare the pharmacokinetics of BAY81-8973 and Advate after intravenous administration.
- Trial name
- NCT02483208
- Testing
- BAY81-8973, Advate
- Where
- Bulgaria
- Volunteers
- 18 people
- Sponsor
- Bayer
Severe Hemophilia A
A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Participants From Birth to 12 Months of Age With Hemophilia A Without Inhibitors
This is a Phase IIIb, multicenter, open-label, single-arm study of prophylactic emicizumab in previously untreated and minimally treated patients at study enrollment from birth to ≤12 months of age with severe hemophilia A…
- Trial name
- HAVEN 7 (NCT04431726)
- Testing
- Emicizumab
- Where
- 32 sites in United States, Australia and 12 more
- Volunteers
- 55 people
- Sponsor
- Hoffmann-La Roche
Hemophilia A, Hemophilia B
Females With Severe or Moderate Hemophilia A or B: A Multi-Center Study
To collect and analyze data on females with hemophilia so as to better define the difference between the study population and the male population with hemophilia.
- Trial name
- NCT00936845
- Where
- 19 sites in United States, Puerto Rico
- Volunteers
- 22 people
- Sponsor
- Weill Medical College of Cornell University
Hemophilia A, Hemophilia B
Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B
Treatment with PF-06741086 is anticipated to demonstrate a clinically relevant advantage and/or a major contribution to patient care in comparison to current methods of treatment for hemophilia A or B because it works differently…
- Trial name
- NCT03938792
- Testing
- PF-06741086
- Where
- 62 sites in United States, Bulgaria and 18 more
- Volunteers
- 188 people
- Sponsor
- Pfizer
Severe Hemophilia A
Pharmacokinetics, Efficacy, Safety, and Immunogenicity of Wilate in Previously Treated Paediatric Patients With Severe Haemophilia A
A prospective, non-controlled, international, multi-centre phase 3 study to investigate the pharmacokinetics, efficacy, safety, and immunogenicity of Wilate in previously treated children with severe haemophilia A
- Trial name
- NCT03376516
- Testing
- Wilate
- Where
- 3 sites in Russia, Ukraine
- Volunteers
- 11 people
- Sponsor
- Octapharma
Haemophilia A, Haemophilia B
Impact of Conservative Treatment by Custom-made Orthoses in Patients With Haemophilic Ankle Arthropathy
The purpose of this study intend to evaluate the effectiveness of functional orthoses (including custom-made insoles and shoes) for preventing and controlling repetitive haemarthrosis in patients suffering of haemophilic ankle…
- Trial name
- NCT00638001
- Testing
- custom-molded insoles and custom-molded shoes
- Where
- Belgium
- Volunteers
- 70 people
- Sponsor
- Cliniques universitaires Saint-Luc- Université Catholique de Louvain