Clinical trials: Haemophilia A
Congenital Bleeding Disorder, Haemophilia B
A Trial Comparing Nonacog Beta Pegol (N9-GP) and ALPROLIX® in Patients With Haemophilia B
This trial is conducted in Europe and the United States of America. The aim of this trial is to compare the pharmacokinetics (the exposure of the trial drug in the body) of nonacog beta pegol (N9-GP) and ALPROLIX® in patients…
- Trial name
- paradigm™7 (NCT03075670)
- Testing
- N9-GP, ALPROLIX®
- Where
- 12 sites in United States, Germany and 1 more
- Volunteers
- 15 people
- Sponsor
- Novo Nordisk A/S
Von Willebrand Disease (VWD), Inherited Bleeding Disorders in Children
Clinical Spectrum and Management of Von Willebrand Disease Among Children in Assiut Governorate
Von Willebrand disease (VWD) is the most common inherited bleeding disorder in children. It occurs due to a deficiency or dysfunction of von Willebrand factor, a protein that plays an essential role in blood clotting.
- Conditions
- Von Willebrand Disease (VWD), Inherited Bleeding Disorders in Children, Pediatric Hemostatic Disorders
- Trial name
- VWD-ASSIUT (NCT07410130)
- Testing
- Tranexamic Acid, Von Willebrand Factor-Containing Concentrates
- Where
- Egypt
- Volunteers
- 25 people
- Sponsor
- Assiut University
Hemophilia
A Study to Learn About the Study Medicine (Called Marstacimab) in People With Hemophilia A or B
The purpose of this non-interventional, observational, real-world study is to find out what are the real-world experiences of people living with hemophilia A or B[OK1.1] who start the study medicine (called marstacimab) in…
- Trial name
- NCT07859527
- Testing
- Marstacimab
- Volunteers
- 100 people
- Sponsor
- Pfizer
Hemophilia A, Gene Therapy
Gene Therapy for Chinese Hemophilia A
IHBDH-GTHA-2020 is an open- label, non- randomized study to evaluate the safety, tolerability and kinetics of a single intravenous infusion of GS001 in hemophilia A subjects with <1 IU/dl residual FVIII levels.
- Trial name
- HA (NCT04728841)
- Testing
- Injection of GS001
- Where
- China
- Volunteers
- 12 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Hemophilia
Does the Thrombin Generation Test Performed During the Pharmacokinetic Profile of the Substitutive Factor VIII Bring Benefits to the Personalized Treatment of Pediatric Patients and Adult Hemophilia A Patients Under Prophylaxis ?
In the context of hemophilia, it is well know that the level of factor VIII alone does not reflect the clinical phenotype of the patients in an accurate way.
- Trial name
- NCT02803502
- Testing
- Chronometric method, Chromogenic method, Thrombin generation test (TGT)
- Where
- 5 sites in Belgium
- Volunteers
- 50 people
- Sponsor
- Brugmann University Hospital
Hemophilia A
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg
This Phase III clinical study will assess the efficacy of BMN 270 defined as FVIII activity, during weeks 49-52 following intravenous infusion of BMN 270 and assess the impact of BMN 270 on usage of exogenous FVIII replacement…
- Trial name
- GENEr8-2 (NCT03392974)
- Testing
- Valoctocogene Roxaparvovec
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- BioMarin Pharmaceutical
Hemophilia
Restricting Blood Flow in Improving Muscle Strength in Patients With Hemophilic Arthropathy
Background. The main physical sequela of patients with hemophilia is the development of a progressive, degenerative intra-articular lesion, known as hemophilic arthropathy).
- Trial name
- NCT05039008
- Testing
- Blood flow restriction
- Where
- Spain
- Volunteers
- 23 people, healthy volunteers welcome
- Sponsor
- Investigación en Hemofilia y Fisioterapia
Congenital Bleeding Disorder, Haemophilia A With Inhibitors
Trial of NovoSeven® in Haemophilia - Joint Bleeds
This trial is conducted in the United States of America (USA). This study compares the effectiveness and safety of NovoSeven® to FEIBA (FEIBA VH) in haemophilia patients with inhibitors being treated for joint bleeds.
- Conditions
- Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
- Trial name
- NCT00108797
- Testing
- eptacog alfa (activated), Feiba VH
- Where
- 20 sites in United States
- Volunteers
- 42 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A
A Study to Evaluate the Safety and Pharmacokinetic of Recombinant Human Coagulation Factor VIII ，Fc Fusion Protein for Injection
Primary objective: To assess the pharmacokinetics of Recombinant Human Coagulation Factor VIII, Fc Fusion Protein for Injection (FRSW107) Secondary objectives: To assess Safety and Tolerability by monitoring FVIII recovery and…
- Trial name
- NCT05251090
- Testing
- ADVATE, FRSW107
- Where
- 6 sites in China
- Volunteers
- 13 people
- Sponsor
- Hangzhou Gensciences Biopharmaceutical Co., Ltd.
Hemophilia
MOTHIF II : THERAPEUTIC MANAGEMENT AND USE OF CLOTTING FACTORS IN HEMOPHILIA A & B IN FRANCE II
MOTHIF II is a non-interventional, multicenter, retrospective, observational data collection in seven French Haemophilia Treatment Centers of the BERHLINGO network.
- Trial name
- MOTHIF II (NCT03596814)
- Testing
- No Interventional study
- Where
- 7 sites in France
- Volunteers
- 2,072 people
- Sponsor
- Nantes University Hospital
Haemophilia A
Efficacy and Safety Evaluation Study of SCT800 in Previously Untreated Hemophilia A Patients.
This is an Open Label, Uncontrolled Study to Evaluate the the Safety and Efficacy of SCT800 for Prophylaxis Treatment in Severe Previously Untreated Hemophilia Patients.
- Trial name
- NCT07437404
- Testing
- SCT800 prophylaxis and treatment for bleeding events
- Where
- China
- Volunteers
- 36 people
- Sponsor
- Sinocelltech Ltd.
Haemophilia
National Survey of People With Haemophilia in Portugal
The purpose of this study is to implement a national survey to collect information regarding Portuguese people with haemophilia (PWH) on socio-demographic, clinical and psychosocial factors.
- Trial name
- NCT02870114
- Where
- Portugal
- Volunteers
- 146 people
- Sponsor
- University of Minho
Hemophilia A
Clot Formation and Clot Stability in Patients With Severe Haemophilia A
In the present study we are examining the clot formation and clot stability in patients with severe haemophilia A after they receive recombinat factor VIII and after addition of tranexamic acid.
- Trial name
- NCT00279578
- Testing
- ReFacto (Recombinant factor VIII) and Tranexamic acid
- Where
- Denmark
- Volunteers
- 8 people
- Sponsor
- University of Aarhus
Hemophilia A, Factor VIII
POCUS: Hemostatic Potential and Joint Health in Patients With Severe Hemophilia A on Novel Replacement Therapies
This is a prospective, randomized control trial in which each patient will be randomly assigned to receive either extended half-life factor VIII based replacement therapy or non-FVIII based replacement therapy, which are both…
- Trial name
- NCT04690322
- Testing
- Eloctate, Adynovate, Emicizumab
- Where
- United States
- Sponsor
- University of Texas Southwestern Medical Center
Hemophilia A, Hemophilia B
A Phase 1 Study of an Investigational Drug, ALN-AT3SC, in Healthy Volunteers and Hemophilia A or B Patients
The purpose of this study is to determine the safety, tolerability, and pharmacokinetics of ALN-AT3SC in healthy volunteers and Hemophilia A or B patients.
- Trial name
- NCT02035605
- Testing
- ALN-AT3SC, Sterile Normal Saline (0.9% NaCl)
- Where
- 14 sites in United States, Bulgaria and 3 more
- Volunteers
- 51 people, healthy volunteers welcome
- Sponsor
- Sanofi
Hemophilia A
A Study in Children, Teenagers and Adults With Severe Hemophilia A Who Switched From Other Factor VIII Treatments to Adynovate
The main aims of the study are to assess the safety profile of Adynovate as well as how well people respond to the preventive treatment with Adynovate.
- Trial name
- NCT04876365
- Testing
- Non-Interventional
- Where
- Canada
- Volunteers
- 153 people
- Sponsor
- Takeda
Hemophilia A
Study Evaluating Pharmacovigilance Of Refacto AF
The purpose of this observational study is to describe the incidence of adverse events among patients treated with Refacto AF in usual health care settings in Germany and Austria.
- Trial name
- NCT00895037
- Testing
- ReFacto AF (Moroctocog alfa)
- Where
- 24 sites in Austria, Germany
- Volunteers
- 101 people
- Sponsor
- Pfizer
Hemophilia A
Moroctocog Alfa (AF-CC) for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Hemophilia A Patients
Moroctocog-alfa (AF-CC) is indicated for the control and prevention of hemorrhagic episodes and for routine and surgical prophylaxis in patients with hemophilia A (congenital factor VIII deficiency or classic hemophilia).
- Trial name
- NCT04396639
- Testing
- Moroctocog-alfa (AF-CC)
- Where
- 5 sites in India
- Volunteers
- 50 people
- Sponsor
- Pfizer
Haemophilia A
A Survey on the Success of Inhibitor Elimination Using Individualized Concentrate Selection and Controlled ITI
This research program is initiated to evaluate and document data on the success of ITI in 300 haemophilia A patients with newly developed or already existing FVIII-inhibitors (also patients who might potentially have failed in…
- Trial name
- NCT02207894
- Where
- Germany
- Volunteers
- 300 people
- Sponsor
- Haemophilia Centre Rhine Main
Hemophilia
Safety and Efficacy of STSP-0601 in Adult Patients with Hemophilia a or B with Inhibitor
This study will assess the safety and efficacy of multiple-dose of STSP-0601 for the treatment of bleeding episodes in hemophilia A or B patients with inhibitor.
- Trial name
- NCT06289166
- Testing
- STSP-0601 for Injection
- Where
- 20 sites in China
- Volunteers
- 25 people
- Sponsor
- Staidson (Beijing) Biopharmaceuticals Co., Ltd