Clinical trials: Blood Coagulation Disorder
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
Abnormalities, Blood Coagulation Disorder
Study of Abnormal Blood Clotting in Children With Stroke
Effective treatment and prevention strategies for childhood stroke and porencephaly can only be developed once the causes are understood.
- Conditions
- Abnormalities, Blood Coagulation Disorder, Brain Disease, Cerebrovascular Accident
- Trial name
- NCT00001927
- Where
- United States
- Volunteers
- 130 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Thrombotic Thrombocytopenic Purpura (TTP)
A Study of TAK-755 in Participants With Congenital Thrombotic Thrombocytopenic Purpura
Thrombotic thrombocytopenic purpura (or TTP for short) is a condition where blood clots form in small blood vessels throughout the body.
- Trial name
- NCT04683003
- Testing
- TAK-755
- Where
- 27 sites in United States, Austria and 9 more
- Volunteers
- 77 people
- Sponsor
- Takeda
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
Wiskott-Aldrich Syndrome
Gene Therapy for WAS
This is a phase I/II study to evaluate the safety and efficacy of Hematopoietic Stem Cell genetherapy for the Wiskott-Aldrich Syndrome.
- Trial name
- NCT01347346
- Testing
- Autologous CD34 positive cells transduced with a lentiviral vector containing human WAS gene
- Where
- France
- Volunteers
- 5 people
- Sponsor
- Genethon
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 B
Study to Determine the Safety and Efficacy of rFIXFc in Previously Untreated Males With Severe Hemophilia B
The primary objective of the study was to evaluate the safety of recombinant coagulation factor IX Fc fusion protein (rFIXFc, BIIB029) in previously untreated patients (PUPs) with severe hemophilia B.
- Trial name
- PUPs B-LONG (NCT02234310)
- Testing
- rFIXFc
- Where
- 27 sites in United States, Australia and 9 more
- Volunteers
- 33 people
- Sponsor
- Bioverativ, a Sanofi company
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
Von Willebrand Disease, Type 1
Evaluation of Bleeding Score in Egyptian Patients With vWD Type I and Correlate it With Laboratory Parameters
Applying the ISTH-BAT questionnaire on Egyptian patients with type I VWD aiming to correlate the BS with the laboratory findings
- Trial name
- NCT03915873
- Testing
- The von Willebrand Antigen level
- Volunteers
- 20 people
- Sponsor
- Assiut University
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
Blood Coagulation Disorders, Inherited, Thrombotic Disorder
Evaluation of Hemostasis in Bleeding and Thrombotic Disorders
The objective of this study will be to assess the coagulation system in-vitro in a variety of bleeding and clotting disorders using the ROTEG analyzer and the thrombin generation assay.
- Trial name
- NCT00178594
- Where
- 2 sites in United States
- Volunteers
- 1,000 people
- Sponsor
- The University of Texas Health Science Center, Houston
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