Clinical trials: Haemophilia A
Hemophilia B
A Trial That Evaluates Disease Characteristics in Hemophilia B Adult Male Participants Receiving Prophylaxis With Standard of Care Factor IX Protein (FIX) Replacement Therapy
This study is focused on males who have Hemophilia B and who need regular preventive treatment with factor IX protein (FIX) replacement therapy to prevent and also to control their bleeding events.
- Trial name
- Honey-B (NCT05568459)
- Testing
- Non-Interventional
- Where
- 17 sites in United States, Canada and 2 more
- Volunteers
- 11 people
- Sponsor
- Regeneron Pharmaceuticals
Hemophilia A, Bleeding Disorder
Characterizing the Impact and Treatment of Reproductive Tract Bleeding on Women and Post-menarchal Girls With Bleeding Disorders
Characterizing the impact and treatment of reproductive tract bleeding on women and post-menarchal girls with bleeding disorders.
- Trial name
- NCT03834727
- Where
- United States
- Volunteers
- 256 people
- Sponsor
- Oregon Health and Science University
Haemophilia A (Moderate or Severe), Hemophilia, Classic
A Study to Investigate the Course of Synovial Hypertrophy in Patients With Haemophilia A on Efanesoctocog Alfa Prophylaxis
The rationale for conducting this open-label phase 4 study is to assess whether once-weekly prophylaxis with efanesoctocog alfa (50 IU/kg) improves the disease course of existing synovial hypertrophy and prevents the risk of…
- Conditions
- Haemophilia A (Moderate or Severe), Hemophilia, Classic, Haemophilia, Hemophilia
- Trial name
- NCT06752850
- Testing
- Efanesoctocog alfa
- Where
- 10 sites in Italy, Norway and 2 more
- Volunteers
- 37 people
- Sponsor
- Swedish Orphan Biovitrum
Hemophilia A, Prophylaxis of Bleeding
A Study to Learn More About the Safety of Damoctocog-alfa-pegol When Used in Routine Medical Care in Korean Participants With Hemophilia A
In this study, researchers will observe and study the data from participants with hemophilia A who receive damoctocog alfa pegol as prescribed by their doctors.
- Conditions
- Hemophilia A, Prophylaxis of Bleeding, Treatment of Bleeding
- Trial name
- NCT06222697
- Testing
- Damoctocog-alfa-pegol (Jivi, BAY94-9027)
- Where
- South Korea
- Volunteers
- 20 people
- Sponsor
- Bayer
Hemophilia A
BAX 855 PK-guided Dosing
1. To compare the efficacy and safety of pharmacokinetic (PK)-guided treatment with BAX 855 targeting FVIII trough levels of 1-3% and approximately 10% (8-12%) 2.
- Trial name
- PROPEL (NCT02585960)
- Testing
- PEGylated Recombinant Factor VIII
- Where
- 83 sites in United States, Australia and 20 more
- Volunteers
- 135 people
- Sponsor
- Baxalta now part of Shire
Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia
Palliative Care Needs of Children With Rare Diseases and Their Families
The palliative care needs of family caregivers of children with rare diseases and their children are largely unmet, including the need for support to prepare for future medical decision making.
- Conditions
- Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia, Asparagine Synthetase Deficiency, CHARGE Syndrome
- Trial name
- FACE-Rare (NCT06938542)
- Testing
- Family Centered pediatric palliative care for family caregivers of children with rare diseases.
- Where
- United States
- Volunteers
- 480 people, healthy volunteers welcome
- Sponsor
- Children's National Research Institute
Hemophilia A
Study of Pharmacokinetics, Efficacy and Safety of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Hemophilia A Patients - A Continuation of Clinical Study 069901
The purpose of this study is to evaluate whether rAHF-PFM is safe and effective in the treatment of hemophilia A subjects. The study consists of 2 parts: Part 1 is a pharmacokinetic and safety study, and Part 2 is an evaluation…
- Trial name
- NCT00157053
- Testing
- Antihemophilic factor, recombinant, manufactured protein-free
- Where
- 24 sites in United States, Austria and 7 more
- Volunteers
- 82 people
- Sponsor
- Baxalta now part of Shire
Hemophilia a, Hemophilia B
The Long-term Safety and Efficacy of SerpinPC in Subjects with Hemophilia Who Completed a Sponsored SerpinPC Clinical Trial
The purpose of the study is to evaluate the efficacy, safety, tolerability and pharmacokinetic (PK) profile of prophylactic SerpinPC in participants with hemophilia
- Conditions
- Hemophilia a, Hemophilia B, Hemophilia a with Inhibitor, Hemophilia B with Inhibitor
- Trial name
- PRESent-6 (NCT06568302)
- Testing
- SerpinPC
- Where
- 2 sites in Georgia, Moldova
- Volunteers
- 15 people
- Sponsor
- ApcinteX Ltd
Hemophilia
Impact of Moderate Intensity Physical Activities on PK-guided EHL FVIII Concentrates Prophylaxis Severe HA Patients
To compare clinical outcomes before and after using combination between moderate to vigorous-intensity physical activities and pharmacokinetic-guided extended half-life FVIII concentrates prophylaxis in moderate to severe…
- Trial name
- NCT05728528
- Testing
- combination between moderate to vigorous-intensity physical activities and pharmacokinetic-guided extended half-life FVIII concentrates prophylaxis, pharmacokinetic-guided extended half-life FVIII concentrates prophylaxis alone
- Where
- Thailand
- Volunteers
- 13 people
- Sponsor
- Chulalongkorn University
Hemophilia A
Prophylaxis Study of Recombinant Factor VIII Manufactured Protein-Free (rAHF-PFM) in Patients With Hemophilia A
The primary purpose of this randomized, two-arm parallel clinical study in 66 previously treated patients with severe or moderately severe hemophilia A is to compare the rate of bleeding episodes for standard prophylaxis (20-40…
- Trial name
- NCT00243386
- Testing
- Antihemophilic factor, recombinant, manufactured protein-free
- Where
- 29 sites in United States, Austria and 8 more
- Volunteers
- 82 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A
Study of rFVIIIFc for Immune Tolerance Induction (ITI) in Haemophilia A Patients With Inhibitors Who Have Failed Previous ITI Therapies
The primary purpose of this study is to describe the outcome of Immune Tolerance Induction (ITI) treatment performed with rFVIIIFc within a timeframe of 60 weeks in patients with haemophilia A who have failed previous attempts at…
- Trial name
- ReITIrate (NCT03103542)
- Testing
- Recombinant coagulation factor (rFVIIIFc)
- Where
- 12 sites in United States, Canada and 5 more
- Volunteers
- 16 people
- Sponsor
- Swedish Orphan Biovitrum
Immune Thrombocytopenia, Thrombotic Thrombocytopenic Purpura
Longitudinal Cohort of Thrombosis and Hemostasis Diseases
This is a multicenter, prospective, longitudinal, observational cohort study to investigate thrombosis and hemostasis diseases in Chinese patients.
- Conditions
- Immune Thrombocytopenia, Thrombotic Thrombocytopenic Purpura, Hemophilia A, Acquired, Disseminated Intravascular Coagulation
- Trial name
- NCT06727669
- Where
- 5 sites in China
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- Peking University People's Hospital
Hemophilia
Combination Regimen With Sodium Valproate for Severe Hemophilia: a Single-arm, Phase 1, Pilot Trial.
The goal of this clinical trial is to determine the clinical efficacy and toxic effects of sodium valproate, sirolimus and calcitriol in the treatment of severe haemophilia in participants with severe haemophilia .
- Trial name
- NCT05920512
- Testing
- Sodium valproate extended-release tablets
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Xue-chun Lu
Congenital Bleeding Disorder, Haemophilia A
Pharmacokinetics of Single Bolus Dose of NovoSeven® in Paediatric and Adult Patients With Haemophilia A or B in a Non- Bleeding State
This trial is conducted in Europe. The aim of this trial is to determine the pharmacokinetics of activated recombinant human factor VII (NovoSeven®) in haemophiliac patients in a non-bleeding state.
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia B
- Trial name
- NCT01562587
- Testing
- activated recombinant human factor VII
- Where
- 8 sites in Germany, Greece and 3 more
- Volunteers
- 18 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A
Lentiviral FVIII Gene Therapy
This study is a Phase I trial using an advanced lentiviral vector to deliver a functional gene for human clotting factor VIII into patients with hemophilia A, to evaluate the safety and efficacy of infusion of lentiviral gene…
- Trial name
- NCT03217032
- Testing
- YUVA-GT-F801
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Shenzhen Geno-Immune Medical Institute
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
Assessment of Proprioceptive and Functional Characteristics in Patients With Hemophilia
Analyze and evaluate the skeletal muscle of patients affected with hemophilia in the Region of Murcia. Describe the periarticular muscular deficit of the lower limbs, depending on the degree of injury: acute and chronic, or…
- Trial name
- HE-BALANCE (NCT02165592)
- Where
- Spain
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Universidad Católica San Antonio de Murcia
Hemophilia A
Assessment of the Risk of Inhibitor Formation in Previously Treated Patients With Severe Hemophilia A
Most transient inhibitor formation, if any, will develop within the first 4 weeks. The study is to further monitor whether participants with severe Hemophilia A will develop inhibitors or antibodies at the later stage when…
- Trial name
- NCT00621673
- Testing
- Kogenate (BAY 14-2222)
- Where
- 2 sites in United States
- Volunteers
- 1 people
- Sponsor
- Bayer
Hemophilia A
Post Marketing Surveillance To Observe Safety And Efficacy Of Xyntha® In Subjects With Hemophilia A
This study is to describe the safety and efficacy of Xyntha® during the usual care setting.
- Trial name
- NCT01790828
- Testing
- Xyntha : coagulation factor IIIV (recombinant)
- Where
- 4 sites in South Korea
- Volunteers
- 42 people
- Sponsor
- Pfizer
Bleeding Disorder
Screening for Bleeding Disorders in Children
The investigators try to improve the screening of bleeding disorders in children by identifying symptoms, laboratory abnormalities and clinical scores discriminating patients congenital bleeding disorders in order to create a…
- Trial name
- NCT05214300
- Where
- France
- Volunteers
- 96 people
- Sponsor
- University Hospital, Montpellier