Clinical trials: Hemophilia B
Hemophilia B
Study To Compare On-Demand Treatment To A Prophylaxis Regimen Of BeneFIX In Subjects With Moderately Severe to Severe Hemophilia B
The purpose of this study will be to determine if a once-weekly prophylaxis regimen of BeneFIX in subjects with moderately severe to severe Hemophilia B is safe and effective.
- Trial name
- NCT01335061
- Testing
- Nonacog alfa
- Where
- 17 sites in Bulgaria, Canada and 7 more
- Volunteers
- 25 people
- Sponsor
- Pfizer
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
Hemophilia B, Severe, Hemophilia A, Severe
Liver Biopsy In Haemophilia Gene Therapy
To perform a liver biopsy in haemophilia A and B patients with endogenous FVIII:C/FIX:C expression at >1% any time after gene transfer following AAV mediated gene transfer.
- Trial name
- NCT04817462
- Testing
- Liver biopsy
- Where
- United Kingdom
- Volunteers
- 10 people
- Sponsor
- University College, London
Hemophilia B Without Inhibitor
Safety and Efficacy Clinical Study of Severe Hemophilia B Treatment With KL001
This is an open- label, non- randomized, uncontrolled, dose-escalation pilot study to evaluate the safety and efficacy of KL001 injection solution in hemophilia B subjects with ≤2 IU/dl residual FIX levels.
- Trial name
- NCT06125756
- Testing
- Low dose KL001, Middle dose KL001, High dose KL001
- Volunteers
- 9 people
- Sponsor
- Affiliated Hospital of Guangdong Medical University
Congenital Bleeding Disorder, Haemophilia B
Safety and Efficacy of NNC-0156-0000-0009 in Haemophilia B Patients
This trial is conducted in Africa, Asia, Europe, Japan and North America. The aim of this trial is to evaluate the safety and efficacy, including pharmacokinetics (the rate at which the body eliminates the trial drug), of…
- Trial name
- paradigm™ 2 (NCT01333111)
- Testing
- nonacog beta pegol
- Where
- 43 sites in United States, Canada and 13 more
- Volunteers
- 74 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A, Hemophilia B
Vitamin K Antagonist Versus Direct Oral Anticoagulant Treatments in Hemophilia
Hemophilia is a rare X-linked bleeding disorder responsible for deficiency of coagulation factor VIII (FVIII) or IX (FIX). The main clinical manifestation is increased bleeding throughout the life which is directly correlated to…
- Conditions
- Hemophilia A, Hemophilia B, Anticoagulant-induced Bleeding
- Trial name
- KADOAH (NCT05804734)
- Testing
- Vitamin K Antagonist - Drug
- Where
- 6 sites in France
- Volunteers
- 54 people
- Sponsor
- Groupe Maladies hémorragiques de Bretagne
Hemophilia A or B
PF-06741086 Multiple Dose Study in Severe Hemophilia
This study is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of multiple subcutaneous and/or intravenous doses of PF-06741086 in subjects with severe hemophilia.
- Trial name
- NCT02974855
- Testing
- PF-06741086
- Where
- 10 sites in United States, Chile and 4 more
- Volunteers
- 27 people
- Sponsor
- Pfizer
Congenital Bleeding Disorder, Haemophilia A
Investigating Safety and Pharmacokinetics of 2 Different Single Doses of NNC128-0000-2011 in Haemophilia A or B Patients
This trial is conducted in Europe and Japan. The aim of this trial is to assess the safety and pharmacokinetics (the rate at which the body eliminates the trial drug) of single doses of NNC128-0000-2011, when administered i.v.
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia B
- Trial name
- NCT01288391
- Testing
- NNC 0128-0000-2011
- Where
- 3 sites in Germany, Japan and 1 more
- Volunteers
- 15 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A
Non Neutralizing Antibodies: Prevalence and Characterization
Antibodies (Abs) directed against factorVIII (FVIII) remain the main iatrogenic complication in haemophilia A (HA) patients. Anti-FVIII Abs inhibiting pro-coagulant properties of the molecule are named inhibitors whereas Abs…
- Trial name
- NCT01541527
- Testing
- blood test
- Where
- France
- Sponsor
- University Hospital, Montpellier
Hemophilia A, Hemophilia B
Socialization of Adult Men With Congenital Hemophilia A or B
The goal of this pilot study is to understand the socialization of adult men with hemophilia living in the United States and their quality of life in order to improve comprehensive care for persons with congenital bleeding…
- Trial name
- PWBCD (NCT01510418)
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- Dana-Farber Cancer Institute
Drug Action Reversal
Reversal of Dabigatran With Prothrombin Complex Concentrate
The purpose of this study is to investigate whether Prothrombin Complex Concentrate (PCC) is effective in reversing the anticoagulant effect of Dabigatran, as assessed by two modified skin-bleeding tests.
- Trial name
- NCT02463591
- Testing
- Beriplex
- Where
- Netherlands
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- University Medical Center Groningen
Hemophilia B
Mutation p.Ile112Thr : Discrepancy Between Factor IX Level and Bleeding Phenotype
It appears that the mutation p.Ile112Thr in the factor IX gene confers a discrepancy between mild factor IX level and severe bleeding phenotype. Databases and litterature analysis are poor on this matter.
- Trial name
- Hémophilie B (NCT03946384)
- Testing
- data collection
- Where
- France
- Volunteers
- 12 people
- Sponsor
- Centre Hospitalier Universitaire Dijon
Mild Hemophilia
Ultrasound Assessment of Joint Health in Patients With Mild Hemophilia (Factor Levels 5-40%)
Currently, few recommendations exist for patients with mild hemophilia, who represent approximately 60% of the hemophilia population.
- Trial name
- echoSAHM (NCT07719647)
- Testing
- Joint Ultrasound (HEAD-US), Blood Sample for Coagulation Factor Measurement
- Where
- France
- Volunteers
- 150 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Hemophilia B, Hemophilia A
A Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%)
To establish baseline prospective efficacy data of current FIX prophylaxis replacement therapy in the usual care setting of hemophilia B subjects, who are negative for nAb to AAV-Spark100, prior to the Phase 3 gene therapy study.
- Trial name
- NCT03587116
- Testing
- Standard of Care FIX Replacement therapy, Standard of Care FVIII Replacement therapy
- Where
- 75 sites in United States, Australia and 16 more
- Volunteers
- 212 people
- Sponsor
- Pfizer
Hemophilia A, Hemophilia B
A Study of a Long-Acting r-Factor 7a (Factor VIIa) in Adult Men With Hemophilia A or B
The purpose of the current Phase 1/2a single dose, dose-escalating study is to evaluate the acute safety, pharmacokinetics (PK) and pharmacodynamics (PD) properties of MOD-5014 in adult subjects with moderate/severe congenital…
- Trial name
- NCT02418793
- Testing
- MOD-5014
- Where
- 11 sites in United States
- Volunteers
- 27 people
- Sponsor
- OPKO Health, Inc.
Hemophilia
Tissue Factor Pathway Inhibitor (TFPI) and Haemorrhagic Manifestations in Haemophilia A and B Patients
Haemophilia is a rare and serious congenital defect of blood coagulation due to a genetic mutation on a sexual chromosome. It affects quasi-essentially the men and it is responsible for bleeding.
- Trial name
- NCT02540187
- Testing
- blood specimen
- Where
- 7 sites in France
- Volunteers
- 164 people
- Sponsor
- Centre Hospitalier Universitaire de Saint Etienne
Hemophilia B
Study Evaluating rFIX; BeneFIX® in Hemophilia B
The primary objective of this clinical research study is to assess the safety and efficacy of rFIX for a minimum of 6 months in previously treated patients (PTPs) with hemophilia B (FIX:C ≤2%) during standard-of-care treatment…
- Trial name
- NCT00093171
- Testing
- rFIX
- Where
- 7 sites in United States
- Volunteers
- 23 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Hemophilia A
Study Comparing Blood Levels of ReFacto and Advante in Hemophilia A
The purpose of the study is to compare the pharmacokinetic parameters of ReFacto and Advate, using the chromogenetic substrate assay to measure plasma Factor VIII activity in plasma.
- Trial name
- NCT00168051
- Testing
- ReFacto, Advante
- Where
- 13 sites in United States, Belgium and 6 more
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Hemophilia B
Trial of AAV5-hFIX in Severe or Moderately Severe Hemophilia B
This study evaluates how safe gene therapy treatment with AAV5-hFIX is in adult patients with severe or moderately severe hemophilia B and severe bleeding type.
- Trial name
- NCT02396342
- Testing
- AAV5-hFIX
- Where
- 7 sites in Denmark, Germany and 1 more
- Volunteers
- 10 people
- Sponsor
- CSL Behring
Hemophilia A, Hemophilia B
Comparing the Burden of Illness of Hemophilia in the Developing and the Developed World
Because of high cost, persons with hemophilia in many developing countries cannot afford adequate treatment. For example, many persons with hemophilia in India and China are only rarely treated with factor replacement in response…
- Trial name
- NCT01217255
- Where
- 4 sites in Brazil, Canada
- Volunteers
- 100 people
- Sponsor
- The Hospital for Sick Children