Clinical trials: Factor VIII Deficiency, Congenital, open to volunteers
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
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 A Patient
Efanesoctocog Prophylaxis Or The Optimization Of Treatment: Real-World Experience Of Taylorizing Clotting Therapy In Hemophilia A
Prophylactic management of severe Hemophilia A (HA) without inhibitors has historically relied on frequent intravenous infusions of standard or EHL FVIII concentrates or on subcutaneous emicizumab administered weekly to monthly…
- Trial name
- E-PROTECT (NCT07830693)
- Testing
- Study questionnaires, Clinical/pharmacological data collection
- Where
- 8 sites in France
- Volunteers
- 553 people
- Sponsor
- Nantes University Hospital
Haemophilia A, Synovitis
Prevalence of Synovitis in Patients With Haemophilia A
The aim of this study is to evaluate the prevalence of synovitis in adult patients with haemophilia A.
- Conditions
- Haemophilia A, Synovitis, Hemophilia Arthropathy, Sonography
- Trial name
- SynoPrev (NCT06352216)
- Testing
- Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US); Haemophilia joint health score (HJHS)
- Where
- Germany
- Volunteers
- 300 people
- Sponsor
- University Hospital, Bonn
Hemophilia A, Hemophilia A, Severe
Hemophilia A Research Program
This study longitudinally observes the intergenerational (mother-child) continuum in hemophilia A from pregnancy through early childhood.
- Conditions
- Hemophilia A, Hemophilia A, Severe, Factor VIII (FVIII), FVIII Deficiency
- Trial name
- HARP (NCT07414511)
- Where
- United States
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- University of Washington
Hemophilia A
Treatment of Hemophilia A Patients With FVIII Inhibitors
This is a non-interventional, multicenter, observational, international study in male persons with haemophilia A who have developed inhibitors to any replacement coagulation factor VIII (FVIII) product.
- Trial name
- MOTIVATE (NCT04023019)
- Testing
- Nuwiq, Octanate, Wilate
- Where
- 2 sites in United States, Germany
- Volunteers
- 120 people
- Sponsor
- Emory University
Factor VIII Deficiency, Congenital
Hemlibra in Mild Hemophilia A
This is a single arm, phase 4, prospective, open-label, United States single-center study to determine the hemostatic characteristics of Hemlibra (emicizumab) as measured by coagulation laboratory parameters in the mild…
- Trial name
- NCT04567511
- Testing
- Emicizumab
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Indiana Hemophilia &Thrombosis Center, Inc.
Healthy Participants, Hemophilia A
A Study of Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SR604 in Two Participants Groups (Part A: Healthy Participants, and Part B: Participants With Hemophilia A or Hemophilia B or Factor VII Deficiency)
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamic (PD) of SR604 in healthy participants (Part A) and to evaluate the safety, tolerability, PK, PD, and efficacy of SR604…
- Conditions
- Healthy Participants, Hemophilia A, Hemophilia B, Factor VII Deficiency
- Trial name
- NCT06349473
- Testing
- SR604
- Where
- 10 sites in United States, Canada
- Volunteers
- 31 people, healthy volunteers welcome
- Sponsor
- Equilibra Bioscience LLC
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 A, Haemophilia B
Post-marketing Surveillance (Special Use-results Surveillance) on Treatment With Alhemo
The purpose of the study is to investigate the safety and effectiveness of Alhemo in participants under real-world clinical practice in Japan. Total duration of this study is about 6 years.
- Trial name
- NCT06831734
- Testing
- Concizumab
- Where
- 23 sites in Japan
- Volunteers
- 30 people
- Sponsor
- Novo Nordisk A/S
Haemophilia A, Haemophilia B
Post-Marketing Surveillance (All Case Surveillance) on Treatment With Alhemo® in Patients With Haemophilia A or Haemophilia B With Inhibitors
The purpose of the study is to investigate the safety and effectiveness of Alhemo® in all participants under real-world clinical practice in Japan. Participants will get Alhemo® as prescribed by the study doctor.
- Trial name
- NCT06285071
- Testing
- Concizumab
- Where
- 11 sites in Japan
- Volunteers
- 23 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A
Nuwiq Dosing and Outcomes In the ManagEment of Women/Girls With Haemophilia A Needing FVIII Treatment for Surgery
Recombinant factor VIII for the prevention of bleeding in women/girls with haemophilia A undergoing major surgery
- Trial name
- NCT05936580
- Testing
- Nuwiq
- Where
- 16 sites in United States, Finland and 8 more
- Volunteers
- 28 people
- Sponsor
- Octapharma
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
Hemophilia A
Long-term Anticoagulation in a Patient With Severe Hemophilia A
To date, and to our knowledge, no case of severe hemophilia A patients receiving long-term anticoagulation has been published. Severe hemophilia A is a hereditary bleeding disorder characterized by a factor VIII (FVIII)…
- Trial name
- HAAC (NCT07314983)
- Where
- France
- Volunteers
- 1 people
- Sponsor
- University Hospital, Strasbourg, France
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
Haemophilia A
Efficacy, Safety & Utilisation of Nuwiq, Octanate and Wilate in Previously Untreated & Minimally Treated Haemophilia A Patients
International, post-authorisation non-interventional study to evaluate real-life effectiveness, safety and utilisation patterns of Octapharma's FVIII concentrates Nuwiq, Octanate, and Wilate in previously untreated and minimally…
- Trial name
- Protect-NOW (NCT03695978)
- Where
- 59 sites in United States, Argentina and 16 more
- Volunteers
- 200 people
- Sponsor
- Octapharma
Hemophilia, Embolization
Embolization in Hereditary Coagulopathies
This is a longitudinal, prospective study, which will include 30 subjects with hereditary coagulopathies, with arthropathy, chronic synovitis resulting from hemarthrosis of the elbows, knees and/or ankles followed up at the…
- Conditions
- Hemophilia, Embolization, Hemarthrosis, Clotting Factor Deficiency
- Trial name
- EHCO (NCT05629130)
- Testing
- embolization with spherical microparticles embosphere
- Where
- Brazil
- Volunteers
- 30 people
- Sponsor
- University of Sao Paulo General Hospital
Hemophilia A, Severe
An Open-label Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Immunogenicity of SKP-0141 for the Treatment and Prophylaxis in Severe Hemophilia a Patients
This is a prospective, multicenter, open-label study to assess efficacy, safety, pharmacokinetics (PK), and immunogenicity of human plasma-derived Factor VIII (FVIII) in previously treated patients (PTPs) with severe hemophilia…
- Trial name
- NCT06738901
- Testing
- SKP-0141
- Volunteers
- 55 people
- Sponsor
- SK Plasma Co., Ltd.
Haemophilia A
Patient Preferences in Treatment of Haemophilia A With and Without Inhibitors: a Discrete Choice Experiment in Germany
This study aims to generate structured patient preference evidence on prophylactic treatment options for haemophilia A for adults with and without inhibitors to support health technology assessment and benefit evaluation…
- Trial name
- NCT07653139
- Testing
- No treatment given
- Where
- Germany
- Volunteers
- 150 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A Without Inhibitor
Multidimensional Assessment of Chronic Pain in Severe Haemophilia A
Introduction: Haemophilia is a congenital coagulopathy characterised by haemarthrosis, mainly in the knees, ankles and elbows. Prophylactic treatment is the most effective therapeutic option for preventing or minimising these…
- Trial name
- NCT07096349
- Where
- Spain
- Volunteers
- 109 people
- Sponsor
- Investigación en Hemofilia y Fisioterapia