Clinical trials: Hemophilia A and B
Liver Cirrhosis, Hemophilia
Alterations in Coagulation Factor Levels in Patients With End Stage Liver Disease
The goal of this observational study is to learn about the changes in coagulation factor VIII and IX levels in patients undergoing liver transplantation to help guide future management of coagulation factor replacement in…
- Conditions
- Liver Cirrhosis, Hemophilia, Hemophilia A, Hemophilia B
- Trial name
- NCT06014320
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Stanford University
Hemophilia, Hemophilia A
Viscoelastic Properties of Lower Extremity Muscles in Patients With Hemophilia
The aim of our study is to evaluate the viscoelastic properties of lower extremity muscles in patients with hemophilia A and hemophilia B secondarily to compare them with their healthy peers.
- Conditions
- Hemophilia, Hemophilia A, Hemophilia B
- Trial name
- NCT05981313
- Where
- Turkey (Türkiye)
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Hasan Kalyoncu University
Hemophilia
Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX
Primary Objective: To characterize the long-term safety and tolerability of fitusiran Secondary Objectives: To characterize the efficacy and long-term efficacy of fitusiran as assessed by the frequency of: Bleeding episodes…
- Trial name
- ATLAS-OLE (NCT03754790)
- Testing
- Fitusiran
- Where
- 79 sites in United States, Australia and 18 more
- Volunteers
- 281 people
- Sponsor
- Genzyme, a Sanofi Company
Hemophilia B
Study Evaluating BeneFIX in Patients With Haemophilia B, Previously Treated With Plasma Derived Factor IX
The purpose of this study is to collect data around the period of the conversion from plasma-derived Factor IX (pdFIX) to BeneFIX.
- Trial name
- NCT00749476
- Testing
- Factor IX recovery
- Where
- 8 sites in France
- Volunteers
- 1 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Hemophilia B, Allergic Reactions
Study to Describe the Allergic Reactions to Factor IX in Patients With Hemophilia B
Retrospective, multicenter study in patients with hemophilia B. This study will be placed at 20 to 50 hemophilia treatment centers in North America and the EU, and is designed to estimate the frequency of Class II and III…
- Trial name
- NCT00195221
- Where
- 38 sites in United States, Austria and 7 more
- Volunteers
- 166 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Hemophilia A, Hemophilia B
A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors
Primary Objective: -To evaluate the efficacy of fitusiran compared to on-demand treatment with factor concentrates, as determined by the frequency of bleeding episodes.
- Trial name
- NCT03417245
- Testing
- fitusiran, factor concentrates
- Where
- 64 sites in United States, Australia and 17 more
- Volunteers
- 120 people
- Sponsor
- Genzyme, a Sanofi Company
Hemophilia B
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
This study will evaluate the efficacy and safety of PF-06838435 (a gene therapy drug) in adult male participants with moderately severe to severe hemophilia B (participants that have a Factor IX circulating activity of 2% or…
- Trial name
- BENEGENE-2 (NCT03861273)
- Testing
- PF-06838435/ fidanacogene elaparvovec
- Where
- 65 sites in United States, Australia and 14 more
- Volunteers
- 51 people
- Sponsor
- Pfizer
Hemophilia, Menstrual Flow Excessive
Extended Half Life Factor (EHF) Products For Heavy Menstrual Bleeding in Hemophilia Carriers
The purpose of this feasibility study is to find out if two clotting factor products, Eloctate [hemophilia A] and Alprolix [hemophilia B], can reduce the amount of menstrual bleeding in female hemophilia A and B carriers (14…
- Trial name
- NCT03272568
- Testing
- Recombinant FVIII Fc fusion product Eloctate, Recombinant FIX Fc fusion product Alprolix, Patient-operated diagnostic device for anemia AnemoCheck.
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Emory University
Cystic Fibrosis, Hemophilia A
Medication Adherence and Non-adherence in Adults With Rare Disease
The purpose of this study is to use the Medication Adherence Reasons Scale (MAR-Scale) to determine the extent of non-adherence to specific medications indicated to treat cystic fibrosis, hemophilia (A or B), idiopathic pulmonary…
- Conditions
- Cystic Fibrosis, Hemophilia A, Hemophilia B, Idiopathic Pulmonary Fibrosis
- Trial name
- NCT04541875
- Testing
- MAR-Scale
- Volunteers
- healthy volunteers welcome
- Sponsor
- Xperiome
Hemophilia a, Hemophilia B
Measurement Properties in People with Hemophilia
The aim of this project is to determine the measurement properties (namely reliability and validity) of a set of clinical assessments, measuring different aspects of physical function.
- Conditions
- Hemophilia a, Hemophilia B, Musculoskeletal Complication, Measurement Error
- Trial name
- HEMOPRO (NCT06565481)
- Testing
- Measurement Properties (Reliability and Validity) of a Set of Assessments evaluating Physical Function in adult Participants with Hemophilia
- Where
- Switzerland
- Volunteers
- 50 people
- Sponsor
- University of Zurich
Hemophilia B
Dose-escalation Study to Investigate the Safety, PK, and PD of ISU304/CB2679d in Hemophilia B Patients
This study is a phase 1, open-label, multi-center, dose-escalation study to investigate the safety, pharmacokinetics and pharmacodynamics of ISU304/CB2679d in previously treated hemophilia B patients.
- Trial name
- NCT03186677
- Testing
- ISU304/CB2679d/Dalcinonacog alfa 75~150 IU/kg, BeneFIX
- Where
- 3 sites in South Korea
- Volunteers
- 11 people
- Sponsor
- ISU Abxis Co., Ltd.
Hemophilia B
Hemophilia B Gene Therapy With AAV8 Vector
Hemophilia B is a bleeding disease in males due to very low levels of coagulation factor IX (FIX) in the blood. The current treatment is intravenous injection of FIX clotting factor concentrates, in response to bleeding.
- Trial name
- NCT01620801
- Testing
- AAV8-hFIX19
- Where
- 3 sites in United States, Australia
- Volunteers
- 4 people
- Sponsor
- Spark Therapeutics, Inc.
Hemophilia A and B, Liver Transplantation
ATHN 11: Liver Transplantation Outcomes Study
This study is designed as a multi-center, observational cohort study of participants with hemophilia A and B who have and have not undergone liver transplantation.
- Trial name
- HOT (NCT04055051)
- Where
- 15 sites in United States
- Volunteers
- 86 people
- Sponsor
- American Thrombosis and Hemostasis Network
Hemophilia A, Hemophilia B
The Effect of Patient Counseling on Adolescent Hemophilia Patient Compliance With Bleeding Logs
The study will see if counselling adolescents with severe or moderate Hemophilia A or B results in increased compliance in the maintenance of bleeding logs.
- Trial name
- NCT00212459
- Testing
- Counseling, Control
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- New York Presbyterian Hospital
Hemophilia A, Hemophilia B
Treatment Patterns And Outcomes In Patients Treated With Benefix Or Refacto/Refacto AF - A Swedish Cohort Study
The overall aim of the study is to describe demographic and clinical characteristics, treatment patterns and outcomes, in the populations of hemophilia patients treated with BeneFIX and ReFacto/ReFacto AF in Sweden
- Trial name
- NCT02740413
- Volunteers
- 86 people
- Sponsor
- Pfizer
Healthy Participants, Hemophilia A
A Study of Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SR604 in Two Participants Groups (Part A: Healthy Participants, and Part B: Participants With Hemophilia A or Hemophilia B or Factor VII Deficiency)
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamic (PD) of SR604 in healthy participants (Part A) and to evaluate the safety, tolerability, PK, PD, and efficacy of SR604…
- Conditions
- Healthy Participants, Hemophilia A, Hemophilia B, Factor VII Deficiency
- Trial name
- NCT06349473
- Testing
- SR604
- Where
- 10 sites in United States, Canada
- Volunteers
- 31 people, healthy volunteers welcome
- Sponsor
- Equilibra Bioscience LLC
Hemophilia
A Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia
This is a multicenter, multinational, open-label, one-way cross-over, Phase 3, single-arm study for treatment of hemophilia. The purpose of this study is to measure the frequency of treated bleeding episodes with fitusiran in…
- Trial name
- ATLAS-NEO (NCT05662319)
- Testing
- Fitusiran, Clotting factor concentrates (CFC) or bypassing agents (BPA), Antithrombin concentrate (ATIIIC)
- Where
- 50 sites in United States, Canada and 15 more
- Volunteers
- 91 people
- Sponsor
- Sanofi
Congenital Bleeding Disorder, Haemophilia A
Investigation of the Pharmacokinetics of NNC172-2021, at Two Different Dose Levels, in Healthy Japanese Subjects
This trial is conducted in Europe. The aim of this trial is to investigate the pharmacokinetics (how the trial drug is distributed in the body) of NNC172-2021 administered subcutaneously, at two different dose levels, in healthy…
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia B, Healthy
- Trial name
- NCT01555749
- Testing
- NNC172-2021
- Where
- United Kingdom
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
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 B
A Safety and Efficacy Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B
This study will examine the safety, pharmacokinetics and efficacy of rIX-FP for the control and prevention of bleeding episodes in subjects who have previously received factor replacement therapy for hemophilia B.
- Trial name
- NCT01496274
- Testing
- rIX-FP
- Where
- 30 sites in United States, Austria and 8 more
- Volunteers
- 63 people
- Sponsor
- CSL Behring