Clinical trials: Hemophilia B, active
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
Hemophilia A or B
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
A study to evaluate the safety of Hympavzi under the actual use in patients with congenital hemophilia who do not have inhibitors.
- Trial name
- HIZ (NCT07161687)
- Testing
- Marstacimab
- Where
- Japan
- Volunteers
- 50 people
- Sponsor
- Pfizer
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
Haemophilia B
Observatory of Patients With Haemophilia B Treated by IdElvion®
OrPHEe is a non-interventional, prospective and national study which aim is to record real life data in haemophilia B French patients treated with Idelvion® to confirm the efficacy and safety of this product established in…
- Trial name
- OrPHEe (NCT05086575)
- Where
- 28 sites in France
- Volunteers
- 222 people
- Sponsor
- CSL Behring
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
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
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
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
Hematologic Disorder, Bleeding Disorder
ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders
In parallel with the growth of ATHN's clinical studies, the number of new therapies for all blood disorders is increasing significantly.
- Conditions
- Hematologic Disorder, Bleeding Disorder, Connective Tissue Disorder, Hemophilia
- Trial name
- NCT04398628
- Where
- 76 sites in United States
- Volunteers
- 3,000 people
- Sponsor
- American Thrombosis and Hemostasis Network
Haemophilia B
A Study Following Males With Haemophilia B on Prophylaxis With Refixia/REBINYN
This study will collect information on side effects and how well Refixia/REBINYN works during long-term treatment (prophylaxis) in males with haemophilia B.
- Trial name
- NCT03745924
- Testing
- Nonacog beta pegol
- Where
- 30 sites in Austria, Belgium and 11 more
- Volunteers
- 60 people
- Sponsor
- Novo Nordisk A/S
Post-acute Infectious Syndrome (PAIS), Post-COVID-19 Condition (PCC or Long COVID)
Post-acute Infectious Syndrome - Aripiprazole Symptom Evaluation (PAIS-AriSE)
Post-acute infectious syndrome (PAIS), including post-COVID-19 condition (PCC or Long COVID), can develop after an infection and may cause persistent symptoms such as fatigue, problems with memory and concentration ("brain fog")…
- Trial name
- PAISE-AriSE (NCT07714213)
- Testing
- Aripiprazole (low-dose, 1mg)
- Where
- Germany
- Volunteers
- 138 people
- Sponsor
- Christiana Franke
Hemophilia A, Hemophilia B
World Bleeding Disorders Registry
The WBDR is an international observational disease registry of patients with hemophilia. It will provide a platform for a network of hemophilia treatment centres (HTCs) around the world to collect uniform and standardized patient…
- Conditions
- Hemophilia A, Hemophilia B, Von Willebrand Diseases
- Trial name
- WBDR (NCT03327779)
- Where
- Canada
- Volunteers
- 20,000 people
- Sponsor
- World Federation of Hemophilia
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
Hemophilia
A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B
This is a parallel, Phase 3, two-arm, open-label study to evaluate the efficacy and safety of treatment with fitusiran prophylaxis administered to male pediatric participants (aged 1 to <12 years) who have severe hemophilia A or…
- Trial name
- ATLAS-KIDS (NCT07285460)
- Testing
- Fitusiran, Clotting factor concentrates (CFC) or bypassing agents (BPA), Antithrombin concentrate (ATIIIC)
- Where
- 41 sites in United States, Belgium and 11 more
- Volunteers
- 85 people
- Sponsor
- Sanofi
Hemophilia B
Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B
The purpose of this study is to evaluate the safety of single ascending IV doses of a Factor IX (FIX) Gene Therapy in up to 16 Adults with Hemophilia B.
- Trial name
- NCT01687608
- Testing
- AskBio009
- Where
- 15 sites in United States
- Volunteers
- 30 people
- Sponsor
- Baxalta now part of Shire