Clinical trials: Hemophilia a
Hemophilia B
Study Evaluating of Recombinant Human Factor IX (BeneFIX) and a New Formulation of BeneFIX (rFIX-R) in Moderate to Severe Hemophilia B
The primary objective of this clinical research study is to establish the bioequivalence of 2 treatments, rFIX and rFIX-R, when given as a 10-minute intravenous bolus infusion.
- Trial name
- NCT00093210
- Testing
- rFIX, rFIX-R
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Haemophilia
Functional and Proprioceptive Status in Patients With Hemophilic Ankle Arthropathy
Introduction: Hemophilic ankle arthropathy is manifested by functional degenerative alterations (muscle strength deficit, mobility and proprioception), intra-articular and chronic pain. Design: Case-control study.
- Trial name
- NCT05425888
- Testing
- Hemophilia group
- Where
- Spain
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Investigación en Hemofilia y Fisioterapia
Hemophilia A
Kogenate FS Regulatory Post-Marketing Surveillance
To obtain data on safety, efficacy, and tolerability of KOGENATE FS under real-life conditions in its registered indications. The observation period for each patient is up to 6 months.
- Trial name
- KG0910KR (NCT01386268)
- Testing
- Recombinant Factor VIII (Kogenate FS, BAY14-2222)
- Where
- South Korea
- Volunteers
- 64 people
- Sponsor
- Bayer
Severe Hemophilia A Without Inhibitor
A Study on the Bone-health Effectiveness of Applying Recombinant Factor VIII Fc (rFVIIIFc) to Patients With Hemophilia A (Prototype A)
Study Objectives* 1. Provide a systematic evaluation of the treatment outcomes in patients with hemophilia A 2. Emphasize the importance and clinical benefits of rFVIII-Fc in joint and bone health. 3.
- Trial name
- NCT05981274
- Testing
- serum biomarker.
- Where
- Taiwan
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- Tri-Service General Hospital (TSGH)
Hemophilia
Learning to Live With Non-severe Haemophilia
While the burden of standard treatment may be reduced through the use of gene therapy, converting those with severe haemophilia to a mild or moderate phenotype, the long-term sequelae of previous joint bleeds and associated…
- Trial name
- NCT05314751
- Testing
- Questionnaire and interview
- Where
- United Kingdom
- Volunteers
- 165 people
- Sponsor
- Haemnet
Hemophilia B
Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B
A long-term follow-up study to evaluate the safety, tolerability, and efficacy of DTX101 in adult males with moderate/severe to severe hemophilia B.
- Trial name
- NCT02971969
- Where
- 5 sites in United States, United Kingdom
- Volunteers
- 6 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Hemophilia A, Hemophilia B
Understanding Hemophilia A and B Drug Dosage Administration Patterns
Study Design A prospective observational, cross-sectional epidemiological study in U.S. site-based clinical practice settings. 30 sites will enroll approximately 300 patients Participating patients - or their caregiver in the…
- Trial name
- NCT03248141
- Testing
- Hemophilia B standard half-life, Hemophilia B extended half-life, Hemophilia A standard half-life
- Where
- 3 sites in United States
- Volunteers
- 11 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
Evaluation Of Acceptability And Satisfaction Of Hemophilia Patients Treated With FusENGO
This study aims to investigate prospectively throughout a period of 3- 6 months and not to exceed 12 months (according to local therapeutic plans) patients experiences of treatment with Factor VIII in the new device named…
- Trial name
- NCT01959919
- Testing
- Refacto FusENGO
- Where
- 19 sites in Italy
- Volunteers
- 86 people
- Sponsor
- Pfizer
Haemophilia A
A Study Looking at How Different Doses of Study Medicine (Inno8) Works in the Body of Healthy Men
This study will test how different doses of study medicine (Inno8) work in the healthy men. The purpose of this study is to prove safety of Inno8 in healthy men, which will support further development of Inno8 in people with…
- Trial name
- VOYAGER1 (NCT06649630)
- Testing
- NNC0442-0344 A
- Where
- 2 sites in Germany
- Volunteers
- 95 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
Hemophilia A
Dose-Response Study of Recombinant Factor VIII Manufactured Protein-Free (rAHF-PFM) in Patients With Hemophilia A
The purpose of this study is to determine the effect of 3 doses of ADVATE rAHF-PFM on initial recovery (% increase [IU/dL] per IU/kg infused) and major single-infusion pharmacokinetic parameters.
- Trial name
- NCT00289536
- Testing
- Antihemophilic factor, recombinant, manufactured protein-free
- Where
- 8 sites in United States
- Volunteers
- 38 people
- Sponsor
- Baxalta now part of Shire
Haemophilia B
Post-marketing Surveillance (Use Result Surveillance) With Refixia®
The participants are invited to take part in this study because they have Haemophilia B. The purpose of this study is to assess the safety and effectiveness of Refixia® about long-term routine use in patients with Haemophilia B.
- Trial name
- NCT03875547
- Testing
- Refixia®
- Where
- 23 sites in Denmark, Japan
- Volunteers
- 28 people
- Sponsor
- Novo Nordisk A/S
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
Hemophilia A, Hemophilia B
Web Accessible Population Pharmacokinetics Service - Hemophilia: Sources of Variability
Using anonymized patient data collected as part of the WAPPS-Hemo project to explore the sources of variability in individual pharmacokinetics (PK); use the sources of variability to improve the performance of the WAPPS-Hemo…
- Trial name
- WAPPS-Hemo (NCT03533504)
- Testing
- Variability in individual PK
- Where
- Canada
- Volunteers
- 1,000 people
- Sponsor
- McMaster University
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 A
Biomarkers Serum Collection Methodology Pilot Study
The purpose of this study is to compare the standard collection of whole blood in tiger top tubes for serum collection versus a collection method of added FVIII to whole blood samples in the hemophilia population for routine…
- Trial name
- NCT01318707
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Georgetown 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
Severe Haemophilia
Quality of Life and Adjustment Among Siblings of Children and Adolescents With Severe Hemophilia
Severe hemophilia is a rare and chronic disease characterized by spontaneous bleedings from early childhood, which may lead to various complications especially in joints.
- Trial name
- FRATHEMO (NCT03299699)
- Where
- France
- Volunteers
- 30 people
- Sponsor
- Assistance Publique Hopitaux De Marseille
Hemophilia
Motor Proficiency of People With Bleeding Disorders Using the BOT-2 (TM)
The purpose of this study is to find out if gross motor skills of children and young adults with bleeding disorders are different from those without bleeding disorders.
- Trial name
- NCT02279199
- Where
- 2 sites in United States
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- Oregon Health and Science University
Hemophilia A
Study Evaluating ReFacto in Hemophilia A
To identify the causative mutations in previously untreated patients with hemophilia A enrolled in the ReFacto® clinical safety and efficacy study CTN 93-R833-0XX/C9741-28, using two established hemophilia mutation testing…
- Trial name
- NCT00038909
- Testing
- BDDrFVII
- Volunteers
- 103 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer