Clinical trials: Clotting Factor Deficiency
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Liver Cirrhosis, Hemophilia
Alterations in Coagulation Factor Levels in Patients With End Stage Liver Disease
The goal of this observational study is to learn about the changes in coagulation factor VIII and IX levels in patients undergoing liver transplantation to help guide future management of coagulation factor replacement in…
- Conditions
- Liver Cirrhosis, Hemophilia, Hemophilia A, Hemophilia B
- Trial name
- NCT06014320
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Stanford University
Deep Venous Thromboembolism
The Association of Warfarin Dosage and Plasma Enantiomer Concentration With the Gene Polymorphisms of CYP and VKOR
Oral warfarin anticogulation for the prevention and treatment of patients with venous thromboembolism is one of the most used therapies in clinical practice.
- Trial name
- NCT00247702
- Testing
- warfarin
- Where
- Taiwan
- Volunteers
- 120 people
- Sponsor
- National Taiwan University Hospital
Embolism and Thrombosis, Embolism
The Clinical and Economic Impact of Pharmacogenomic Testing of Warfarin Therapy in Typical Community Practice Settings
The purpose of this quasi-experiment study, which could also be classified as a prospective observational intervention study, is to assess the impact of cytochrome P450 2C9 (CYP 2C9) and vitamin K epoxide reductase complex…
- Conditions
- Embolism and Thrombosis, Embolism, Vascular Diseases, Warfarin
- Trial name
- MHSMayoWarf1 (NCT00830570)
- Testing
- CYP 2C9 and VKORC1 Testing for Warfarin
- Where
- United States
- Volunteers
- 1,635 people
- Sponsor
- Medco Health Solutions, Inc.
Hemophilia B, Factor IX Deficiency
Phase I/II Study of Monoclonal Factor IX Concentrate for Factor IX Deficiency
OBJECTIVES: Assess the safety and long-term efficacy of monoclonal factor IX concentrate in patients with factor IX deficiency.
- Trial name
- NCT00004801
- Testing
- monoclonal factor IX replacement therapy
- Volunteers
- 3 people
- Sponsor
- National Center for Research Resources (NCRR)
Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited
Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants
The aim of this prospective, observational study is to establish a dataset on the frequency of bleeding events, as well as other characteristics of bleeding events and FVIII infusions, in patients with clinically severe…
- Conditions
- Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, Hemophilia A
- Trial name
- NCT03876301
- Testing
- Standard of Care FVIII Replacement therapy
- Where
- 11 sites in United States, Australia and 2 more
- Volunteers
- 25 people
- Sponsor
- Spark Therapeutics, Inc.
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
Severe Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor
An Observational Study to Evaluate Physical Activity, Bleeding Incidence and Health Related Quality of Life, in Participants With Haemophilia A Without Inhibitors Receiving Standard of Care Treatment
This multicenter, non-interventional, prospective study will collect information about activity status, bleeds, health-related quality of life (HRQoL), health status, and safety in participants with moderate or severe haemophilia…
- Conditions
- Severe Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor, Haemophilia A
- Trial name
- POWER (NCT04165135)
- Testing
- Standard of Care for Haemophilia A
- Where
- 17 sites in Italy
- Volunteers
- 103 people
- Sponsor
- Hoffmann-La Roche
Atrial Fibrillation, Cardiac Thrombus
VKORC1 and CYP2C9 Gene Polymorphisms and Warfarin Management
The investigators aimed to use pharmacogenetic information in clinical practise which may lead to rapid, efficient, and safe warfarin dosing in this observational prospective study.
- Conditions
- Atrial Fibrillation, Cardiac Thrombus, Deep Vein Thrombosis, Pulmonary Embolism
- Trial name
- NCT00970892
- Testing
- Warfarin dose titration
- Where
- Turkey (Türkiye)
- Volunteers
- 500 people
- Sponsor
- Ankara University
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
Factor VIII Deficiency
Efanesoctocog Alfa Prophylaxis in Patients With Hemophilia A With Synovial Hypertrophy
The objective of the study is to assess the improvement of synovial hypertrophy during the 12 months of efanesoctocog alfa prophylaxis once per week (QW) in joints with existing evidence of synovial hypertrophy in participants…
- Trial name
- SYNOVIIIUS (NCT06941870)
- Testing
- Efanesoctocog alfa
- Where
- 14 sites in United States, Canada and 2 more
- Volunteers
- 35 people
- Sponsor
- Sanofi
HIV Infections, Hemophilia A
Effect of Indinavir Plus Two Other Anti-HIV Drugs on Blood Clotting in HIV-Positive Males With Hemophilia
The purpose of this study is to see if indinavir plus two other anti-HIV drugs affect blood clotting in HIV-positive patients with hemophilia.
- Trial name
- NCT00002386
- Testing
- Indinavir sulfate, Lamivudine, Stavudine
- Where
- 8 sites in United States, Canada
- Volunteers
- 55 people
- Sponsor
- Merck Sharp & Dohme LLC
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
Congenital Afibrinogenemia, Hypofibrinogenemia
FIB Grifols Congenital Deficiency for On-demand Treatment and Surgical Prophylaxis
This is a phase 3, multi-center, prospective, open-label, single-arm, clinical trial to be carried out in subjects with congenital fibrinogen deficiency manifested as afibrinogenemia or severe hypofibrinogenemia.
- Trial name
- NCT04636268
- Testing
- FIB Grifols
- Sponsor
- Instituto Grifols, S.A.
Hemophilia C
A Single and Low Dose of Recombinant Factor VIIa in Patients With Severe Factor XI Deficiency Undergoing Surgery
Lately the investigators found that patients with severe factor XI deficiency and inhibitors could undergo major surgery with a single low dose of recombinant factor VIIa and tranexamic agent.
- Trial name
- rFVIIa (NCT01538160)
- Testing
- recombinant factor VIIa
- Where
- 3 sites in Israel
- Volunteers
- 15 people
- Sponsor
- Sheba Medical Center
Prothrombin Complex Factor Deficiency
Efficacy and Safety of Prothromplex Total (Prothrombin Complex Concentrate) in Oral Anticoagulant Reversal
The purpose of the study is to assess the efficacy and safety of Prothromplex Total as a treatment for the immediate reversal of oral anticoagulant therapy with vitamin K antagonists in patients with acquired deficiency of…
- Trial name
- NCT01159210
- Testing
- Prothrombin complex concentrate (coagulation factors IX, II, VII and X in combination)
- Where
- 6 sites in Austria, Hungary
- Volunteers
- 61 people
- Sponsor
- Baxalta now part of Shire
Factor VII Deficiency
Treatment of Inherited Factor VII Deficiency
FVII deficiency is a rare coagulation disorder. A limited number of patients are found in most treatment centres and countries. Treatment demands vary considerably amongst FVII deficient patients.
- Trial name
- STER (NCT01269138)
- Testing
- plasma derived Factor VII, recombinant FVIIa, Fresh Frozen Plasma
- Where
- Italy
- Volunteers
- 223 people
- Sponsor
- University of L'Aquila
Factor XI Deficiency, Factor XII Deficiency
The Proteins of the Contact Activation System
Cardiovascular diseases are important causes of morbidity and mortality in the industrialized world. Abnormalities in the coagulation system, causing a hypercoagulable state, are a known risk factor for arterial and venous…
- Trial name
- CONTACT (NCT02785718)
- Testing
- Global Haemostasis assays
- Where
- France
- Volunteers
- 18 people
- Sponsor
- Hospices Civils de Lyon
Hemophilia A
Trial of Rituximab Versus Oral Cyclophosphamide to Eradicate or Suppress Autoimmune Anti-Factor VIII Antibodies in Acquired Hemophilia A
The purpose of this study is to evaluate the rate of response when administering rituximab to suppress or eliminate the anti-body in a patient's blood that inhibits the effectiveness of their factor replacement product compared…
- Trial name
- NCT00306670
- Testing
- Rituxan, prednisone
- Volunteers
- 2 people, healthy volunteers welcome
- Sponsor
- Georgetown University
Acquired Coagulation Factor Deficiency
Prothrombin Complex Concentrate for Anticoagulant Reversal
Patients on oral anticoagulants need rapid reversal of the anticoagulant effect in case of acute bleeding or emergency surgery. Prothrombin Complex Concentrates are known to provide a fast reversal of the anticoagulant effect…
- Trial name
- NCT00168077
- Testing
- Prothrombin Complex Concentrate
- Where
- 15 sites in Austria, Germany and 6 more
- Volunteers
- 40 people
- Sponsor
- CSL Behring