Clinical trials: Blood Coagulation Disorders, open to volunteers
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
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
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
ITP - Immune Thrombocytopenia
"Cytotoxic T Lymphocyte-Associated Antigen-4 (CTLA-4) Gene Single-nucleotide Polymorphism in Primary Immune Thrombocytopenic Purpura in Children"
the investigators aim in this study to investigate the potential association of single gene polymorphisms of CTLA-4 (SNPs; rs: 3087243) using real-time PCR in children with primary ITP.
- Trial name
- ITP (NCT07014904)
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Sohag University
Hemophilia A
Lentiviral FVIII Gene Therapy
This study is a Phase I trial using an advanced lentiviral vector to deliver a functional gene for human clotting factor VIII into patients with hemophilia A, to evaluate the safety and efficacy of infusion of lentiviral gene…
- Trial name
- NCT03217032
- Testing
- YUVA-GT-F801
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Shenzhen Geno-Immune Medical Institute
Immune Thrombocytopenia (ITP), Warm Autoimmune Hemolytic Anemia (WAIHA)
A Phase 1b/2a Study of Velinotamig in Adults With Relapsed/Refractory ITP and wAIHA
A Phase 1b/2a, open-label dose escalation and dose expansion study of subcutaneously (SC) administered velinotamig for the treatment of adults with relapsing and refractory immune thrombocytopenia (ITP) and warm autoimmune…
- Trial name
- NCT07738822
- Testing
- Velinotamig specified dose on specified days.
- Where
- United States
- Volunteers
- 72 people
- Sponsor
- Cullinan Therapeutics Inc.
Liver Transplantation
Assessment of a Quantra-guided Transfusion Algorithm in Liver Transplantation
Liver transplantation carries a substantial risk of bleeding, making precise haemostasis control essential, although assessing coagulation remains challenging.
- Trial name
- QUALIVERT (NCT06749405)
- Testing
- Transfusion algorithm based on Quantra®, Conventional transfusion algorithm
- Where
- France
- Volunteers
- 56 people
- Sponsor
- University Hospital, Toulouse
Acute Ischemic Stroke, Cerebral Infarction
Safely Quenching Complement in Stroke Survivors
This study will include adults (ages 18-80) who have had a stroke caused by a large blood clot blocking blood flow in the brain.
- Trial name
- SUCCESS (NCT07495722)
- Testing
- Pegcetacoplan Injection [Empaveli]
- Volunteers
- 20 people
- Sponsor
- Columbia University
Immune Thrombocytopenia (ITP)
Level of Serum Lactate Dehydrogenase in Immune Thrombocytopenia
Assessment of relation between serum lactate dehydrogenase and immune thrombocytopenia
- Trial name
- NCT07046013
- Testing
- Lactate dehydrogenase
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Sohag University
Hemophilia
Feasibility and Safety of Blood-Flow-Restriction Training in Patients With Hemophilia
In the course of a cross-over study design, 12 patients with hemophilic arthropathy will perform a training load on the knee extensor muscles.
- Trial name
- NCT07259356
- Testing
- Control, BFR Exercise (50% of the LOP) while Leg Press Exercise (4 Sets, All out)
- Where
- Germany
- Volunteers
- 12 people
- Sponsor
- University Hospital, Bonn
Hemophilia A
Safety and Efficacy Study of NGGT003 in Hemophilia A Patients
This is an early phase 1, open-label, single-center, dose-escalation pilot trial to evaluate the safety and efficacy of an intravenous infusion of NGGT003 in hemophilia A patients.
- Trial name
- NCT06238908
- Testing
- NGGT003
- Where
- China
- Volunteers
- 6 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Immune Thrombocytopenia (ITP)
An Exploratory Study of Golidocitinib in Adult Patients With ITP
This is a multicenter clinical study to evaluate the safety and efficacy of golidocitinib in patients with primary immune thrombocytopenia (ITP). The study consists of two parts: Part A dose escalation and Part B dose expansion.
- Trial name
- NCT07196163
- Testing
- Golidocitinib
- Where
- China
- Volunteers
- 138 people
- Sponsor
- Peking University People's Hospital
Henoch-Schönlein Purpura, Gastrointestinal Injury
Mycophenolate Mofetil in Gastrointestinal Henoch-Schonlein Purpura
Henoch Schönlein purpura (HSP) is the most common type of vasculitis in children, with an incidence of ~10/100,000, whereas >90% of the patients develop symptoms at <10 years of age.
- Conditions
- Henoch-Schönlein Purpura, Gastrointestinal Injury, Mycophenolate Mofetil
- Trial name
- NCT05951517
- Testing
- MMF
- Where
- China
- Volunteers
- 24 people
- Sponsor
- Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
Crohn Disease, Ulcerative Colitis
Safety and Efficacy of Fecal Microbiota Transplantation
The gut microbiota is critical to health and functions with a level of complexity comparable to that of an organ system. Dysbiosis, or alterations of this gut microbiota ecology, have been implicated in a number of disease…
- Conditions
- Crohn Disease, Ulcerative Colitis, Celiac Disease, Irritable Bowel Syndrome
- Trial name
- NCT04014413
- Testing
- Fecal Microbiota Transplantation
- Where
- Hong Kong
- Volunteers
- 450 people
- Sponsor
- Chinese University of Hong Kong
MRI, Hemophilia
MRI Role in Knee Hemophilic Arthopathy
The aim of this study is to assess role of MRI in detecting synovial, cartilaginous , osseous abnormalities ، bleeding inside knee joint and to use a system for assessing HA as support for therapeutic regimes and for monitoring…
- Conditions
- MRI, Hemophilia, Arthropathy Hemophilic, Arthropathy of Knee
- Trial name
- NCT07233122
- Volunteers
- 95 people
- Sponsor
- Assiut University
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
Hemophilia A
A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)
The BMN 270 clinical development program consists of multiple interventional studies designed to assess the safety and efficacy of a single infusion of BMN 270 for at least 5 years post-infusion.
- Trial name
- GENEr8-LTE (NCT05768386)
- Where
- 41 sites in United States, Australia and 10 more
- Volunteers
- 172 people
- Sponsor
- BioMarin Pharmaceutical
Hemophilia
The World Federation of Hemophilia Gene Therapy Registry
The aim of the WFH GTR is to provide a database in which long-term data on PWH who receive gene therapy from around the world, will be collected and housed.
- Trial name
- WFH GTR (NCT04883710)
- Testing
- Gene therapy
- Volunteers
- 5,000 people
- Sponsor
- World Federation of Hemophilia
Thrombotic Thrombocytopenic Purpura (TTP)
A Study in Children and, Adults With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated With Adzynma
Congenital thrombotic thrombocytopenic purpura (cTTP) is a rare blood disorder that some people are born with. It is caused by a problem in a gene passed down from parents to children, which affects the body's ability to produce…
- Trial name
- NCT07353099
- Testing
- No Intervention
- Where
- 7 sites in Austria, Germany
- Volunteers
- 50 people
- Sponsor
- Takeda