Clinical trials: Hemophilia A, Congenital
Hemophilia A
Evaluation of Patient and Physician Reported Reasons for Switching Factor VIII Replacement Therapies
This US study will assess hemophilia A patient characteristics, health history and reasons for switching or not switching from both patient/caregiver and physician perspectives.
- Trial name
- PARkER (NCT03405337)
- Testing
- FVIII products, Conventional FVIII replacement therapies
- Where
- United States
- Volunteers
- 160 people
- Sponsor
- Bayer
Hemophilia A
Evaluation of SCT800 in Prophylaxis Therapy on Previous Treated Patients With Severe Hemophilia A
This study is a multi-center, open-label, single-arm trial to evaluate the efficacy,safety and pharmacokinetics of SCT800 in regular prophylaxis and perioperative treatment in patients (≥12 years old) with severe hemophilia A who…
- Trial name
- NCT03815318
- Testing
- Recombinant Human Coagulation FVIII
- Where
- China
- Volunteers
- 73 people
- Sponsor
- Sinocelltech Ltd.
Mild Haemophilia A Without Inhibitor
DDAVP vs. Exercise in Patients With Mild Hemophilia A
Individuals with mild hemophilia A (MHA) bleed infrequently but can in the setting of trauma which often is when participating in sports/exercise.
- Trial name
- NCT03136003
- Testing
- DDAVP, Exercise
- Where
- 2 sites in Canada
- Volunteers
- 30 people
- Sponsor
- The Hospital for Sick Children
Severe Hemophilia A
Study to Investigate the Long-term Efficacy and Safety of Human-cl rhFVIII in Previously Treated Patients (PTPs)
The purpose of the study is to study the long-term efficacy, safety and tolerability of Human-cl rhFVIII in previously treated patients with severe hemophilia A.
- Trial name
- NCT01341912
- Testing
- Human-cl rhFVIII
- Where
- 2 sites in United States, Bulgaria
- Volunteers
- 3 people
- Sponsor
- Octapharma
Hemophilia
A Feasibility Study to Collect Data in Patients With Haemophilia
The purpose of this study is to collect reference data in patients with haemophilia. The study will also collect and store blood samples for potential future exploratory research in the disease area.
- Trial name
- NCT00798499
- Testing
- Laboratory variables
- Where
- 2 sites in Sweden
- Volunteers
- 50 people
- Sponsor
- AstraZeneca
Hemophilia, Hemophilia A
ATHN 8: Previously Untreated Patients (PUPs) Matter Study
This is a multi-center cohort study of approximately 250 previously untreated patients (PUPs) with congenital moderate to severe hemophilia A or B in a network of up to 50 US Hemophilia Treatment Centers (HTCs).
- Conditions
- Hemophilia, Hemophilia A, Hemophilia B, Hemophilia A With Inhibitor
- Trial name
- NCT03818529
- Where
- 27 sites in United States
- Volunteers
- 237 people
- Sponsor
- American Thrombosis and Hemostasis Network
Hemophilia, Hemophilia A
Improved Factor VIII Inhibitor Evaluation
Commercial one and two-stage factor VIII assays may not detect some clinically significant inhibitor antibodies. The purpose of the proposed study is to standardize and validate a platelet-based factor VIII activity assay with…
- Trial name
- IFIE (NCT02726139)
- Where
- United States
- Sponsor
- Boston VA Research Institute, Inc.
Sonography, Haemophilia
Sonography and Haemophilia
The aim of this study is to evaluate the possibility of establishing a patient-based ultrasound monitoring of the knee (representing a target joint accessible with any hand) with a handheld device: The following questions will be…
- Trial name
- NCT04550988
- Testing
- feasibility study
- Where
- Germany
- Volunteers
- 5 people
- Sponsor
- University Hospital, Bonn
Von Willebrand Diseases, Hemophilia A
BT200 in Hereditary Bleeding Disorders
BT200 is a PEGylated aptamer that binds to the A1 domain of human von Willebrand factor (VWF). At low doses, BT200 blocks the clearance of VWF antigen (VWF Ag) from the circulation and causes an increase in concentrations of both…
- Trial name
- NCT04677803
- Testing
- BT200
- Where
- Austria
- Volunteers
- 26 people
- Sponsor
- Medical University of Vienna
Hemophilia
Bilateral Deficit Phenomenon in Patients With Haemophilic Arthropathy
Trial to assess the bilateral deficit phenomenon during dynamic plantar flexion task in patients with haemophilic arthropathy Describe the differences in terms of the physical variables (muscular strength, range of motion and…
- Trial name
- PROPRIO-HE (NCT02165462)
- Testing
- Patients with haemophilia
- Where
- Spain
- Volunteers
- 17 people
- Sponsor
- Universidad Católica San Antonio de Murcia
Hemophilia A
Study of a Gene Therapy Treatment for Hemophilia A
The purpose of this study is to evaluate the efficacy of SPK-8011 in preventing bleed episodes compared with FVIII prophylaxis in participants with hemophilia A without FVIII inhibitors on routine FVIII prophylaxis.
- Trial name
- KEYSTONE 1 (NCT06297486)
- Testing
- SPK-8011
- Where
- 27 sites in United States
- Sponsor
- Spark Therapeutics, Inc.
Hemophilia A
A Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously Received Preventive Treatment With Emicizumab
This is an exploratory, single group, Phase 4, study to assess treatment with fitusiran prophylaxis after switching from emicizumab prophylaxis.
- Trial name
- NCT06145373
- Testing
- Fitusiran (SAR439774), Clotting factor concentrates (CFC) or bypassing agents (BPA), Antithrombin concentrate (ATIIIC)
- Where
- 3 sites in United States, Taiwan
- Volunteers
- 20 people
- Sponsor
- Sanofi
Congenital Bleeding Disorder, Haemophilia A
Safety and Efficacy of Turoctocog Alfa in Haemophilia A Subjects
This trial is conducted in Asia, Europe, and North and South America. The trial consists of a main trial and a sub-trial. The main trial investigates safety and efficacy of turoctocog alfa (recombinant factor VIII, rFVIII (N8))…
- Trial name
- guardian™1 (NCT00840086)
- Testing
- turoctocog alfa
- Where
- 63 sites in United States, Brazil and 16 more
- Volunteers
- 150 people
- Sponsor
- Novo Nordisk A/S
Hemophilia B with Inhibitor, Hemophilia B
A Study of SerpinPC in Participants with Hemophilia B (HemB) with Inhibitors
The purpose of the study is to evaluate the efficacy, safety, tolerability and pharmacokinetic (PK) profile of prophylactic SerpinPC in participants with Hemophilia B with inhibitors, as part of the SerpinPC registrational…
- Trial name
- PRESent-3 (NCT05789537)
- Testing
- SerpinPC
- Where
- 20 sites in United States, Armenia and 9 more
- Volunteers
- 3 people
- Sponsor
- ApcinteX Ltd
Hemophilia A
Satisfaction Regarding the Dispensing of Emicizumab (HEMLIBRA®) for Patients With Haemophilia A in France
Haemophilia A (HA) is a rare constitutional haemorrhagic disease whose drug management is based on the use of chronic lifelong replacement therapy.
- Trial name
- PASODOBLEDEMI2 (NCT05450640)
- Testing
- satisfaction survey
- Where
- France
- Volunteers
- 176 people
- Sponsor
- Hospices Civils de Lyon
Hemophilia A
Efficacy and Safety of Recombinant Factor VIII (GreenGene) in Patients With Hemophilia A
The purpose of this study is to study the evaluation of efficacy and safety of GreenGene (Recombinant Factor VIII).
- Trial name
- NCT01568580
- Testing
- GreenGene
- Volunteers
- 71 people
- Sponsor
- Green Cross Corporation
Hemophilia B
Evaluation of a Recombinant Factor IX Product, APVO101, in Previously-Treated Pediatric Patients With Hemophilia B
Phase 3/4, single arm, open-label study to evaluate PK, safety, and efficacy of APVO101 prophylaxis in severe or moderately severe hemophilia B subjects < 12 years of age.
- Trial name
- NCT03855280
- Testing
- APVO101
- Where
- 11 sites in Brazil, Georgia and 4 more
- Volunteers
- 21 people
- Sponsor
- Medexus Pharma, Inc.
Blood Coagulation Disorders, Hematologic Diseases
Cooperative Study of Factor VIII Inhibitors
To test the efficacy of prothrombin complex concentrates (Factor IX) in the treatment of hemophiliac patients who had inhibitors to Factor VIII.
- Conditions
- Blood Coagulation Disorders, Hematologic Diseases, Hemophilia A, Hemorrhagic Disorders
- Trial name
- NCT00000582
- Testing
- factor ix
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Haemophilia A
Efficacy and Safety of Turoctocog Alfa Pegol (N8-GP) for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Chinese Patients With Haemophilia A (pathfinder10)
The study investigates how well the medicine called turoctocog alfa pegol (N8-GP) works in previously treated Chinese patients with severe haemophilia A. Participants will be treated with N8-GP.
- Trial name
- Pathfinder10 (NCT05082116)
- Testing
- turoctocog alfa pegol (N8-GP)
- Where
- 11 sites in China
- Volunteers
- 36 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A
Comparison of Different Prophylaxis Regimens for Moderate to Severe Hemophilia A Pediatric Patients
Title: Comparison of different prophylaxis regimens for hemophilia A pediatric patients Principal Investigator: Assistant Professor Darintr Sosothikul, MD Research Question: Does different factor VIII dosage effect outcome of…
- Trial name
- NCT02727647
- Testing
- FVIII
- Where
- Thailand
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Chulalongkorn University