Clinical trials: FVIII Deficiency
Hemophilia A, Acquired
Emicizumab in Acquired Hemophilia A
This study is an international, multicenter, open-label, single arm, prospective clinical trial and will evaluate the efficacy of prophylactic emicizumab administered on a scheduled basis to prevent bleeds in patients with…
- Trial name
- NCT04188639
- Testing
- Emicizumab Injection
- Where
- 16 sites in Austria, Germany
- Volunteers
- 47 people
- Sponsor
- GWT-TUD GmbH
Hemophilia
Joint Outcome Study Continuation for Children With Severe Factor VIII Deficiency
The original Joint Outcome Study (JOS) enrolled 65 boys with hemophilia from 16 sites nationally. The subjects were randomized to one of two arms (prophylaxis or an enhanced episode-based treatment)and were followed prospectively…
- Trial name
- JOSC (NCT01000844)
- Volunteers
- 11 people
- Sponsor
- University of Colorado, Denver
Severe Haemophilia A
Subclinical Joint Bleeding in Irish Adults With Severe Haemophilia A on Personalised Prophylaxis Regimens
This trial is designed to assess if there is evidence of subclinical joint bleeding on MRI/X-Ray in adults with severe Haemophilia A while on standard and/or pharmacokinetically tailored prophylaxis regimens.
- Trial name
- PERSONAL (NCT02314325)
- Testing
- ADVATE [Antihemophilic Factor (Recombinant)]
- Where
- Ireland
- Volunteers
- 20 people
- Sponsor
- St. James's Hospital, Ireland
Adeno-Associated Virus (AAV), Blood Coagulation Disorder
Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors
SPK-8016 is in development for the treatment of patients with inhibitors to FVIII. This Phase 1/2, open-label, non-randomized, dose-finding study to evaluate the safety, efficacy, and tolerability of SPK-8016 in adult males with…
- Conditions
- Adeno-Associated Virus (AAV), Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders
- Trial name
- NCT03734588
- Testing
- SPK-8016
- Where
- 11 sites in United States
- Volunteers
- 4 people
- Sponsor
- Spark Therapeutics, Inc.
Acquired Hemophilia A
Trial of Acquired Haemophilia With Steroid Combined With Cyclophosphamide Versus Steroid Combined With Rituximab
Purpose: To evaluate the efficacy when administering steroid combined with single dose rituximab to eliminate the antibody in acquired hemophilia A patients compared to treatment using steroid with cyclophosphamide.
- Trial name
- NCT03384277
- Testing
- Steroid, Rituximab, Cyclophosphamide
- Where
- China
- Volunteers
- 66 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Hemophilia A, Hemophilia B
Correlation Between Vitamin D Status and Bone Mineral Density in Patients With Hemophilia
Study will look at baseline Vitamin D levels, calcium, albumin, liver enzymes, testosterone, osteocalcin, urine N telopeptides, bone mineral density, nutritional assessment and physical activity assessment of boys with hemophilia…
- Conditions
- Hemophilia A, Hemophilia B, Vitamin D Deficiency
- Trial name
- NCT01090206
- Testing
- Vitamin D and calcium
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Virginia Commonwealth University
Acquired Hemophilia A
Study of TAK-672 in Participants With Acquired Hemophilia A
The main aims of the study are to learn if TAK-672 can control bleeds in participants with acquired hemophilia A and if the participants have side effects from TAK-672.
- Trial name
- NCT04580407
- Testing
- TAK-672
- Where
- 9 sites in Japan
- Volunteers
- 5 people
- Sponsor
- Takeda
Hemophilia A, Hemophilia B
Study of Prophylaxis, ACtivity and Effectiveness (SPACE) in Hemophilia Patients Currently Treated With ADVATE or RIXUBIS
This 6-month prospective outcomes study addresses the association between timing of infusion, patient activity levels, and bleeding episodes through patient-reported measurements.
- Trial name
- SPACE (NCT02190149)
- Testing
- ADVATE (Antihemophilic Factor [Recombinant]), RIXUBIS (Coagulation Factor IX [Recombinant])
- Where
- 17 sites in United States
- Volunteers
- 64 people
- Sponsor
- Baxalta now part of Shire
Inherited Bleeding Disorders
Study to Evaluate the Use of Ultrasound in the Diagnosis of Hemophilic Joint Disease
The purpose of this study is to see whether power Doppler sonography (PDS) can be used to diagnose synovitis in patients with inherited bleeding disorders.
- Trial name
- NCT00212485
- Testing
- Power Doppler Sonography, MRI
- Where
- United States
- Volunteers
- 43 people
- Sponsor
- New York Presbyterian Hospital
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
HIV Infection, Hemophilia A
Efficacy and Safety of MK-0518 in Treatment-Experienced HIV-1 Infected Adult Patients With Hemophilia
This is an randomized, double-blind, placebo-controlled study to assess the safety, tolerability and efficacy of MK-0518 compared to placebo when combined with other antiretroviral drugs, in treatment-experienced HIV-infected…
- Trial name
- NCT00797381
- Where
- China
- Volunteers
- 100 people
- Sponsor
- Shanghai Public Health Clinical Center
Hemophilia, Factor XIII Deficiency
Clinical Trial of Factor XIII (FXIII) Concentrate
Congenital deficiency of Factor XIII is a rare but potentially life threatening disorder. It is inherited in an autosomal recessive fashion.
- Trial name
- NCT00640289
- Testing
- Fibrogammin P
- Where
- United States
- Volunteers
- 72 people
- Sponsor
- Children's Hospital of Orange County
Hemophilia A, Hemophilia B
An Institutional Pilot Study to Investigate Physical Activity Patterns in Boys With Hemophilia
This pilot project was developed to investigate subjective and objective data related to the patterns of physical activity participation among hemophilia patients (FVIII or FIX) between the ages of 5 and 18 years.
- Conditions
- Hemophilia A, Hemophilia B, Factor VIII Deficiency, Factor IX Deficiency
- Trial name
- HEPS (NCT02199717)
- Testing
- Accelerometer
- Where
- Canada
- Volunteers
- 66 people
- Sponsor
- The Hospital for Sick Children
Acquired Hemophilia A
Expanded Access to B-Domain Deleted Recombinant Porcine Factor VIII (OBI-1) in the Treatment of Acquired Hemophilia A Due to Factor VIII Inhibitory Auto-antibodies
To provide emergency access to OBI-1 for patients with Acquired Hemophilia and a serious bleeding episode who do not have access to an active clinical study site associated with the OBI-1-301 study.
- Trial name
- NCT01968655
- Testing
- OBI-1
- Where
- 4 sites in United States
- Sponsor
- Baxalta now part of Shire
Hemophilia A
Long-term Study Evaluating Joint Health in People With Haemophilia A Receiving Real-world Prophylactic Treatment With Efanesoctocog Alfa
The rationale for this study is to further understand and describe the long-term prophylactic effectiveness of efanesoctocog alfa in preventing joint bleeds in a real-life setting.
- Trial name
- NCT06940830
- Testing
- Efanesoctocog alfa
- Where
- 31 sites in Croatia, Czechia and 6 more
- Volunteers
- 252 people
- Sponsor
- Swedish Orphan Biovitrum
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 Hemophilia A
Prospective and Retrospective, Non-interventional Study to Evaluate the Safety and Effectiveness of Obizur in Real-life Practice
The study addresses the safety, utilisation and effectiveness of Obizur in the treatment of bleeding episodes in real-life clinical practice in Europe and the United States.
- Trial name
- NCT03199794
- Testing
- OBIZUR
- Where
- 26 sites in United States, Austria and 5 more
- Volunteers
- 50 people
- Sponsor
- Baxalta now part of Shire
Factor VIII Deficiency
A Phase 3 Open-label Interventional Study of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein, Efanesoctocog Alfa (BIVV001), in Patients With Severe Hemophilia A
Primary Objective: To evaluate the efficacy of BIVV001 as a prophylaxis treatment in prophylaxis treatment arm. Secondary Objectives: To evaluate the efficacy of BIVV001 as a prophylaxis treatment.
- Trial name
- XTEND-1 (NCT04161495)
- Testing
- efanesoctocog alfa (BIVV001)
- Where
- 47 sites in United States, Argentina and 17 more
- Volunteers
- 159 people
- Sponsor
- Bioverativ, a Sanofi company
Factor VIII Deficiency, Factor IX Deficiency
The European Paediatric Network for Haemophilia Management ( PedNet Registry)
Rationale: Haemophilia is a rare disease; to improve knowledge international collaboration is needed. Well-defined clinical data will be collected from complete cohorts in order to prevent selection bias.
- Trial name
- PedNet (NCT02979119)
- Where
- 39 sites in Austria, Belgium and 17 more
- Volunteers
- 4,000 people
- Sponsor
- PedNet Haemophilia Research Foundation