Clinical trials: Hemophilia, open to volunteers
Hemophilia, Upper Extremity
Comparison of Upper Extremity Functional Performance in Hemophilic and Healthy Children
The goal of this observational study is to evaluate the functional performance of the upper extremity joints (scapula, shoulder, elbow, and wrist) in haemophilic children and compare muscle strength and ADL limitations with…
- Conditions
- Hemophilia, Upper Extremity, Strength, Performance
- Trial name
- NCT06826209
- Where
- Turkey (Türkiye)
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Hacettepe University
Hemophilia A, Hemophilia B
Liver Biopsy Following Gene Therapy For Hemophilia
This observational study will obtain liver biopsy samples and evaluate the long-term effect of adeno-associated virus (AAV)-mediated gene therapy on the liver tissue in adult patients with hemophilia A or hemophilia B who have…
- Trial name
- NCT05932914
- Testing
- Liver Biopsy
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- St. Jude Children's Research Hospital
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.
Post-acute Infectious Syndrome (PAIS), Post-COVID 19 Condition (PCC or Long COVID)
CD19-B Cell Depletion in PAIS-ME/CFS Patients
Post-acute infection syndromes (PAIS) are long-lasting health problems that can develop after an infection. They include post-COVID-19 syndrome and similar illnesses following other infections.
- Conditions
- Post-acute Infectious Syndrome (PAIS), Post-COVID 19 Condition (PCC or Long COVID), Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS)
- Trial name
- PIONEER_PAIS (NCT07724834)
- Testing
- Inebilizumab
- Where
- Germany
- Volunteers
- 38 people
- Sponsor
- Charite University, Berlin, Germany
Hemophilia A
A Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously Received Preventive Treatment With Emicizumab
This is an exploratory, single group, Phase 4, study to assess treatment with fitusiran prophylaxis after switching from emicizumab prophylaxis.
- Trial name
- NCT06145373
- Testing
- Fitusiran (SAR439774), Clotting factor concentrates (CFC) or bypassing agents (BPA), Antithrombin concentrate (ATIIIC)
- Where
- 3 sites in United States, Taiwan
- Volunteers
- 20 people
- Sponsor
- Sanofi
Hemophilia, Chronic Pain
Multimodal Telerehabilitation Versus Face-to-Face Care for Adults With Hemophilia and Chronic Pain
Hemophilia is an inherited bleeding disorder that is commonly associated with chronic joint pain, reduced physical function, and decreased quality of life.
- Trial name
- HEMO-TELE (NCT07682519)
- Testing
- other, Exercise
- Where
- Chile
- Volunteers
- 24 people
- Sponsor
- University of Chile
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, Rebalancing Agents
Monitoring of Anti-TFPI in Hemophilia
During the development of anti-TFPI antibodies, thrombin generation assay (TGA) was employed using both in vitro measurements (antibodies added to blood samples) and ex vivo approaches (blood samples from patients in phase II and…
- Conditions
- Hemophilia, Rebalancing Agents, Prophylaxis
- Trial name
- EUREKA (NCT07101926)
- Testing
- blood sampling
- Where
- 2 sites in France
- Volunteers
- 11 people
- Sponsor
- Hospices Civils de Lyon
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
Hemophilia B
Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B
This is a phase 3, prospective, open-label, single-arm, single-dose, multicenter study investigating the efficacy, safety, and tolerability of CSL222 (AAV5-hFIXco-Padua) in adolescent male participants with severe or moderately…
- Trial name
- IX-TEND 3004 (NCT07080905)
- Testing
- CSL222 (Adeno-associated viral vector serotype 5 [AAV5]-hFIXco-Padua)
- Where
- 17 sites in United States, Australia and 6 more
- Volunteers
- 20 people
- Sponsor
- CSL Behring
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.
Cardiac Valve Disease
Prothrombin Complex Concentrate vs Fresh Frozen Plasma in Goal-directed Bleeding Management in Cardiac Surgery
The primary endpoint of this study is the completion time of hemostasis treatment when administered Fresh frozen plasma (FFP) and frozen powder coagulation factor concentrate (PCC) in goal-directed bleeding management for cardiac…
- Trial name
- NCT06144125
- Testing
- fresh frozen plasma, prothrombin complex concentrate
- Where
- South Korea
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Konkuk University Medical Center
Septic Shock, Sepsis
Effects of Anisodamine on Sublingual Microcirculation and Vascular Waterfall Phenomenon in Patients With Septic Shock
This prospective, multicenter, single-arm, open-label interventional pilot study aims to evaluate the short-term physiological effects of intravenous anisodamine on sublingual microcirculation and vascular-waterfall parameters in…
- Conditions
- Septic Shock, Sepsis, Critical Illness
- Trial name
- ANISO-MICRO (NCT07657702)
- Testing
- Anisodamine intravenous infusion
- Where
- China
- Volunteers
- 20 people
- Sponsor
- First Affiliated Hospital of Wannan Medical College
Hemophilia A or B
A Study to Describe Patient REal-world FitusiRan ExpeRience
This is a real-world, mixed-methods observational study conducted among adolescent (ages 12-17 years) and adult participants diagnosed with hemophilia A or B, with or without Factor VIII (FVIII) or Factor IX (FIX) inhibitors, who…
- Trial name
- PREFER (NCT07895381)
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Sanofi
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
Severe Hemophilia A
Study of Efficacy and Safety of FRSW107 in Pediatric Patients With Severe Hemophilia A
This study was divided into four stages: screening period, main trial period, extension period and follow-up period. In the main trial, both groups received FRSW107 prophylactic therapy.
- Trial name
- NCT06136507
- Testing
- FRSW107
- Where
- 13 sites in China
- Volunteers
- 76 people
- Sponsor
- Hangzhou Gensciences Biopharmaceutical Co., Ltd.
Hemophilia B
Study of the Safety, Pharmacodynamics and Efficacy of ANB-002 in Patients With Hemophilia B (SAFRAN)
The goal of this multicenter, two-stage, open-label study is to investigate the safety, immunogenicity, and efficacy of ANB-002 in subjects with hemophilia В.
- Trial name
- NCT06120582
- Testing
- ANB-002, dose 1, ANB-002, dose 2, ANB-002, dose 3
- Where
- 17 sites in Belarus, Russia
- Volunteers
- 28 people
- Sponsor
- Biocad
Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
Cell-free fetal DNA (cffDNA) is present in the maternal blood from the early first trimester of gestation and makes up 5%-20% of the total circulating cell-free DNA (cfDNA) in maternal plasma.
- Conditions
- Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease, Cystic Fibrosis, Fragile X Syndrome
- Trial name
- DANNIgene (NCT06147414)
- Testing
- Blood sample
- Where
- France
- Volunteers
- 550 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
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
Hemophilia A, Hemophilia B
A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B
The purpose of this clinical trial is to learn about the safety and effects of the study medicine (called marstacimab) for the potential treatment of hemophilia in pediatric patients.
- Trial name
- BASIS KIDS (NCT05611801)
- Testing
- marstacimab
- Where
- 63 sites in United States, Argentina and 21 more
- Volunteers
- 100 people
- Sponsor
- Pfizer