Clinical trials: Coagulation Abnormalities, open to volunteers
Von Willebrand Disease, Type 3
A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebrand Disease
This is a Phase III, multicenter, open-label clinical study designed to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of emicizumab prophylaxis in participants aged 1 month and above, who have been…
- Trial name
- WILL-EMI (NCT06998524)
- Testing
- Emicizumab, von Willebrand Factor (VWF) Concentrates, Factor VIII (FVIII) Concentrates
- Where
- 29 sites in United States, Belgium and 12 more
- Volunteers
- 75 people
- Sponsor
- Hoffmann-La Roche
Von Willebrand Diseases, Glanzmann Thrombasthenia
Videomicroscopy for the Prediction of Bleeding in Constitutional Haemorrhagic Diseases
In Willebrand disease, there is currently no test available to identify non-invasively patients with a high risk of bleeding from angiodysplasias The study propose to use a sublingual capillary bed analysis by video-microscopy, a…
- Trial name
- VIDEO-BLEED (NCT04119908)
- Testing
- Sublingual videomicroscopy, blood sample
- Where
- France
- Volunteers
- 400 people
- Sponsor
- University Hospital, Lille
Hemophilia A or B
Safety of KN057 Prophylaxis in Patients With Haemophilia A or B
The purposes of this open-label, multicenter III clinical trial are to evaluate the safety and efficacy of long-term preventive treatment with KN057 in Haemophilia A or B patients with or without inhibitors, and to assess the…
- Trial name
- NCT07545395
- Testing
- KN057
- Where
- China
- Volunteers
- 70 people
- Sponsor
- Suzhou Alphamab Co., Ltd.
Hemophilia B
The Efficacy and Safety of ZS801 in Chinese Hemophilia B Patients.
A non-randomized, open-label study to evaluate the safety, kinetics and efficacy of a single intravenous infusion of ZS801 in hemophilia B subjects with endogenous FIX ≤2%.
- Trial name
- NCT05630651
- Testing
- ZS801
- Where
- China
- Volunteers
- 6 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Hemophilia B
Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222)
The primary purpose of this study is to assess the long-term safety and efficacy in male adults with hemophilia B who were treated with CSL222 (CSL222) in parent studies CSL222_2001 (NCT03489291) or CSL222_3001 (NCT03569891).
- Trial name
- IX-TEND 3003 (NCT05962398)
- Testing
- AAV5-hFIXco-Padua
- Where
- 28 sites in United States, Belgium and 6 more
- Volunteers
- 56 people
- Sponsor
- CSL Behring
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 A, Hemophilia B
German Pediatric Hemophilia Research Database
The German Pediatric Hemophilia Research Database will collect data on the prophylactic and therapeutic use of factor concentrates, complications, outcome measures (joint scores, QoL) and living circumstances in newly diagnosed…
- Conditions
- Hemophilia A, Hemophilia B, Children, Drug Specific Antibodies
- Trial name
- GEPHARD (NCT02912143)
- Testing
- documentation only
- Where
- Germany
- Volunteers
- 800 people
- Sponsor
- Goethe University
Chronic Myeloproliferative Disorders, Leukemia
Tissue, Blood, and Body Fluid Sample Collection From Patients With Hematologic Cancer
RATIONALE: Collecting and storing samples of tissue, blood, and body fluid from patients with cancer to study in the laboratory may help the study of cancer in the future.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Lymphoproliferative Disorder
- Trial name
- NCT01137643
- Testing
- biologic sample preservation procedure, cytology specimen collection procedure
- Where
- United States
- Volunteers
- 15,000 people
- Sponsor
- UNC Lineberger Comprehensive Cancer Center
Haemophilia
Determinants and Consequences of the Transition to Adulthood for Adolescents With Severe Haemophilia: TRANSHEMO 2, an Ancillary Study to the TRANSHEMO Project
Haemophilia is a rare genetic disorder which, in its severe form and in the absence of treatment, can be life-threatening. Since the 1960s and the introduction of coagulation factor concentrates, the life expectancy of people…
- Trial name
- TRANSHEMO2 (NCT07418827)
- Testing
- questionnaires
- Where
- 25 sites in France
- Volunteers
- 75 people
- Sponsor
- Assistance Publique Hopitaux De Marseille
Hemophilia A
Low Dose Emicizumab vs Low Dose Factor VIII in Prophylaxis in Hemophilia A Patients
Low dose factor VIII prophylaxis is practiced around the world. Role of standard dose Emicizumab prophylaxis is well established. Emicizumab is an expensive drug.
- Trial name
- AFEEL (NCT06938659)
- Testing
- Emicizumab Injection, Factor VIII (FVIII)
- Where
- Bangladesh
- Volunteers
- 20 people
- Sponsor
- Dhaka Medical College
Haemophilia A
A Survey on the Success of Inhibitor Elimination Using Individualized Concentrate Selection and Controlled ITI
This research program is initiated to evaluate and document data on the success of ITI in 300 haemophilia A patients with newly developed or already existing FVIII-inhibitors (also patients who might potentially have failed in…
- Trial name
- NCT02207894
- Where
- Germany
- Volunteers
- 300 people
- Sponsor
- Haemophilia Centre Rhine Main
Transthyretin (TTR) Amyloid Cardiomyopathy, Cardiac Amyloidosis
Liver and Coagulation Disorders in Cardiac Transthyretin Amyloidosis
Transthyretin cardiac amyloidosis (ATTR-CA) is a progressive infiltrative cardiomyopathy caused by the deposition of misfolded transthyretin protein within the myocardium.
- Conditions
- Transthyretin (TTR) Amyloid Cardiomyopathy, Cardiac Amyloidosis, Cardiomyopathies, Wild-Type Transthyretin Cardiac Amyloidosis
- Trial name
- LICA2025 (NCT07766135)
- Where
- Italy
- Volunteers
- 70 people
- Sponsor
- University of Messina
Hemophilia B
Gene Therapy for Hemophilia B Patients Aged 12-18 Years Old
This is a Phase 1, open- label, non- randomized, uncontrolled, single dose pilot study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of BBM-H901 in hemophilia B subjects with ≤2IU/dl residual…
- Trial name
- NCT05709288
- Testing
- BBM-H901
- Where
- China
- Volunteers
- 9 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Hemophilia A
Evaluation of the Safety and Efficacy of Hemophilia A Gene Therapy Drugs
This is a multi-center, Phase 1/2/3, single-arm, open-label, single-dose treatment clinical study to evaluate the safety, tolerability and efficacy of BBM-H803 injection in severe Hemophilia A subjects.
- Trial name
- NCT06111638
- Testing
- Single dose intravenous injection of BBM-H803
- Where
- 10 sites in China
- Volunteers
- 55 people
- Sponsor
- Shanghai Xinzhi BioMed Co., Ltd.
Hemophilia B
A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B
Participants in this study have a genetic mutation, specifically in the coagulation (blood clotting) Factor 9 gene that causes severe or moderately severe hemophilia B.
- Trial name
- BEYOND-9 (NCT06379789)
- Testing
- REGV131, LNP1265
- Where
- 52 sites in United States, Australia and 7 more
- Volunteers
- 130 people
- Sponsor
- Regeneron Pharmaceuticals
Vasculitis, Behcet's Disease
Vasculitis Pregnancy Registry
The purpose of this study is to learn about the experience of women with vasculitis who become pregnant. In particular, the study will consist of several online surveys to assess 1.
- Conditions
- Vasculitis, Behcet's Disease, CNS Vasculitis, Cryoglobulinemic Vasculitis
- Trial name
- V-PREG (NCT02593565)
- Testing
- Online questionnaires
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- University of Pennsylvania
Hemophilia A
Cartilage Biomarkers and HEAD-US Score in Severe Hemophilia A Patients Receiving FVIII Prophylaxis
Patients with severe hemophilia A often develop joint complication requiring careful monitoring. This study aimed to compare the effectiveness of standard low dose weight-based FVIII concentrates (CFCs) prophylaxis with…
- Trial name
- HEADUS_SHA8 (NCT06816953)
- Testing
- HEAD-US, Urinary carboxy-terminal cross linked telopeptide of type II collagen, Inflammatory markers
- Where
- Thailand
- Volunteers
- 19 people
- Sponsor
- Chulalongkorn University
Hemophilia A, Hemophilia B
An Open-label, Multicenter Phase I/II Clinical Trial to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetic/Pharmacodynamic (PK/PD) Characteristics of SR604 Injection in Patients With Hemophilia A/B and Congenital Factor VII Deficiency
The purpose of this study is to evaluate the safety, tolerability, immunogenicity , PK, and PD of a single dose of SR604 in participants with Hemophilia A or Hemophilia B, with or without inhibitors （Part A）and to evaluate the…
- Conditions
- Hemophilia A, Hemophilia B, Factor VII Deficiency
- Trial name
- NCT07644832
- Testing
- SR604
- Where
- 9 sites in China
- Volunteers
- 76 people
- Sponsor
- Shanghai RAAS Blood Products Co., Ltd.
Hemophilia
Effectiveness of an Educational Program for Pain Management in Patients With Hemophilic Arthropathy
Introduction: Hemophilic arthropathy is a common complication of hemophilia, characterized by chronic pain, functional limitation, and impaired quality of life.
- Trial name
- NCT07420387
- Testing
- Educational group
- Where
- Spain
- Volunteers
- 70 people
- Sponsor
- Investigación en Hemofilia y Fisioterapia
Hemophilia A
A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A
This study will assess the safety and tolerability of SPK-8011QQ in adult males with moderately severe to severe hemophilia A.
- Trial name
- NCT07226206
- Testing
- SPK-8011QQ
- Where
- 6 sites in United States
- Volunteers
- 5 people
- Sponsor
- Hoffmann-La Roche