Clinical trials: Moderate Hemophilia A
Hemophilia A
Safety/Efficacy Study to Assess Whether FVIII/VWF Concentrate Can Induce Immune Tolerance in Haemophilia A Patients
The purpose of this study is to assess the role of a FVIII/VWF complex concentrate (Emoclot) in successfully inducing immune tolerance (I.T.I.) in patients with Haemophilia A with inhibitors, including patients at high risk of…
- Trial name
- ITI (NCT02479087)
- Testing
- Plasma-derived FVIII/VWF concentrate
- Where
- 4 sites in Egypt, India
- Volunteers
- 20 people
- Sponsor
- Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Hemophilia B
Pivotal Study (Pharmacokinetics, Efficacy, Safety) of BAX 326 (rFIX) in Hemophilia B Patients
The purpose of this pivotal Phase 1/3 study is to determine the pharmacokinetic (PK) parameters, the hemostatic efficacy, and the safety of BAX 326, a recombinant factor IX, in previously treated patients (PTPs) with severe and…
- Trial name
- NCT01174446
- Testing
- BAX 326, BeneFIX
- Where
- 25 sites in Argentina, Brazil and 12 more
- Volunteers
- 86 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A
Relative Bioavailability Of Two Formulations Of Moroctocog Alfa (AF-CC)
This study is being conducted to compare how moroctocog alfa (AF-CC) acts in the body when administered as 2 different dose presentations.
- Trial name
- NCT01579903
- Testing
- moroctocog alfa (AF-CC)
- Where
- 3 sites in Bulgaria, Hungary and 1 more
- Volunteers
- 16 people
- Sponsor
- Pfizer
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 A
Evaluate Efficacy and Safety of Recombinant Factor VIII （rFVIII）Treatment of Severe or Moderately Severe Hemophilia A
Efficacy, Safety and Pharmacokinetics Study of a rFVIII in Chinese subjects with Hemophilia A.To assess efficacy and safety of rFVIII administered as treatment and as on-demand therapy in adult and adolescent (12-65 years)…
- Trial name
- NCT02930317
- Testing
- Recombinant Factor VIII (50 IU/kg), Recombinant Factor VIII (On-demand treatment)
- Where
- 4 sites in China
- Volunteers
- 60 people
- Sponsor
- Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
Hemophilia
KN057 Multiple Dose Study in Moderately Severe to Severe Hemophilia
This study is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of multiple subcutaneous doses of KN057 in subjects with hemophilia A or B, with or without inhibitors to Factor VIII…
- Trial name
- NCT05421429
- Testing
- KN057 doseⅠ, KN057 dose Ⅱ, KN057 dose Ⅲ
- Where
- 2 sites in China
- Volunteers
- 24 people
- Sponsor
- Suzhou Alphamab Co., Ltd.
Hemophilia B
Study of AAV5-hFIX in Severe or Moderately Severe Haemophilia B
This is an open-label, extension study enrolling patients who have successfully completed all assessments in Study CT-AMT-060-01 (Years 1-5).
- Trial name
- NCT05360706
- Testing
- AAV5-hFIX
- Where
- 6 sites in Germany, Netherlands
- Volunteers
- 9 people
- Sponsor
- CSL Behring
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
Hemophilia A, Hemophilia B
Females With Severe or Moderate Hemophilia A or B: A Multi-Center Study
To collect and analyze data on females with hemophilia so as to better define the difference between the study population and the male population with hemophilia.
- Trial name
- NCT00936845
- Where
- 19 sites in United States, Puerto Rico
- Volunteers
- 22 people
- Sponsor
- Weill Medical College of Cornell University
Hemophilia A, Hemophilia B
Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B
Treatment with PF-06741086 is anticipated to demonstrate a clinically relevant advantage and/or a major contribution to patient care in comparison to current methods of treatment for hemophilia A or B because it works differently…
- Trial name
- NCT03938792
- Testing
- PF-06741086
- Where
- 62 sites in United States, Bulgaria and 18 more
- Volunteers
- 188 people
- Sponsor
- Pfizer
Haemophilia A, Haemophilia B
Impact of Conservative Treatment by Custom-made Orthoses in Patients With Haemophilic Ankle Arthropathy
The purpose of this study intend to evaluate the effectiveness of functional orthoses (including custom-made insoles and shoes) for preventing and controlling repetitive haemarthrosis in patients suffering of haemophilic ankle…
- Trial name
- NCT00638001
- Testing
- custom-molded insoles and custom-molded shoes
- Where
- Belgium
- Volunteers
- 70 people
- Sponsor
- Cliniques universitaires Saint-Luc- Université Catholique de Louvain
Hemophilia A
Health Related Quality of Life of Youth and Young Adults With Hemophilia A
The relevance of the study will be in establishing whether Health Related Quality of Life (HRQoL) measures obtained repeatedly during hemophilia care are sensitive to significant changes in health or personal circumstances…
- Trial name
- NCT01034904
- Where
- 2 sites in Canada
- Volunteers
- 48 people
- Sponsor
- CSL Behring
Hemophilia
The Dynamic Interplay Between Bleeding Phenotype and Baseline Factor Level in Moderate and Mild Hemophilia A and B
There are large inter-individual differences in the bleeding pattern of patients with moderate or mild hemophilia. The major determinant of bleeding phenotype is the level of coagulant factor VIII or IX.
- Trial name
- DYNAMO (NCT03623295)
- Testing
- Blood sample, Questionnaire, MRI-imaging
- Where
- 17 sites in Australia, Austria and 8 more
- Volunteers
- 304 people
- Sponsor
- Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Hemophilia A, Hemophilia B
Females With Severe or Moderate Hemophilia A or B: an International Multi-center Study
This is a multi-center, international study designed to collect clinical, genetic and quality of life information on females with hemophilia, an inherited bleeding disorder.
- Trial name
- NCT00936312
- Where
- 11 sites in United States, Australia and 7 more
- Volunteers
- 200 people
- Sponsor
- Weill Medical College of Cornell University
Hemophilia A
Efficacy and Safety Study of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Pediatric Patients in Canada With Hemophilia A - A Continuation of Baxter Study 060101
The purpose of this study is to evaluate whether rAHF-PFM is safe and effective in the treatment of children with hemophilia A. The study is open to pediatric patients in Canada who completed Baxter Study 060101.
- Trial name
- NCT00189982
- Testing
- Antihemophilic Factor Manufactured and Formulated without Added Human or Animal Proteins (rAHF-PFM)
- Where
- Canada
- Volunteers
- 4 people
- Sponsor
- Baxalta now part of Shire
Hemophilia
Canadian CHO-KLAT/H-FIT Study - Quality of Life of, and Burden of Caring for, Persons With Hemophilia
The primary goal of this observational study is to determine if: health-related quality of life of boys with moderate/severe hemophilia A who are followed in Canadian pediatric hemophilia treatment centres is significantly…
- Trial name
- NCT06191068
- Where
- Canada
- Volunteers
- 190 people
- Sponsor
- The Hospital for Sick Children
Hemophilia A
Efficacy and Safety of ADVATE Standard Prophylaxis to Hemophilia A
Hemophilia A is an X-linked recessive, congenital bleeding disorder caused by deficient or defective coagulation factor VIII (FVIII).
- Trial name
- NCT02280265
- Testing
- Recombinant Human Coagulation Factor VIII for injection
- Volunteers
- 15 people
- Sponsor
- Nanjing Medical University
Severe Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor
An Observational Study to Evaluate Physical Activity, Bleeding Incidence and Health Related Quality of Life, in Participants With Haemophilia A Without Inhibitors Receiving Standard of Care Treatment
This multicenter, non-interventional, prospective study will collect information about activity status, bleeds, health-related quality of life (HRQoL), health status, and safety in participants with moderate or severe haemophilia…
- Conditions
- Severe Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor, Haemophilia A
- Trial name
- POWER (NCT04165135)
- Testing
- Standard of Care for Haemophilia A
- Where
- 17 sites in Italy
- Volunteers
- 103 people
- Sponsor
- Hoffmann-La Roche
Hemophilia B
Dose Confirmation Trial of AAV5-hFIXco-Padua
This is an open-label, single-dose, single-arm, multi-center trial, with a screening, a treatment + post-treatment follow-up phase, and a long-term follow-up phase.
- Trial name
- NCT03489291
- Testing
- AAV5-hFIXco-Padua (AMT-061)
- Where
- 4 sites in United States
- Volunteers
- 3 people
- Sponsor
- CSL Behring
Hemophilia
Resistance Training With Blood Flow Restriction in Hemophilia
The main objectives of this study are to evaluate safety and chronic effects of a low-load resistance exercise intervention (elbow flexions and extensions, and knee extensions) with blood flow restriction in people with…
- Trial name
- HemoBFR (NCT05568524)
- Testing
- BFRT, Control
- Where
- Spain
- Volunteers
- 20 people
- Sponsor
- University of Valencia