Clinical trials: Hemophilia a
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
Telemedicine, Exercise
Feasibility of a Blended Therapy Approach
Exercise is an important part of therapy guidelines in the rehabilitation of rare diseases (RDs) as Haemophilia and Myositis. The aim of this study is not to evaluate a new therapy intervention, but to evaluate the delivery of…
- Conditions
- Telemedicine, Exercise, Feasibility, Myositis
- Trial name
- bletheRD (NCT03713151)
- Testing
- Dividat FIT
- Where
- Switzerland
- Volunteers
- 30 people
- Sponsor
- University of Zurich
Hemophilia
Impact of Moderate Intensity Physical Activities on PK-guided EHL FVIII Concentrates Prophylaxis Severe HA Patients
To compare clinical outcomes before and after using combination between moderate to vigorous-intensity physical activities and pharmacokinetic-guided extended half-life FVIII concentrates prophylaxis in moderate to severe…
- Trial name
- NCT05728528
- Testing
- combination between moderate to vigorous-intensity physical activities and pharmacokinetic-guided extended half-life FVIII concentrates prophylaxis, pharmacokinetic-guided extended half-life FVIII concentrates prophylaxis alone
- Where
- Thailand
- Volunteers
- 13 people
- Sponsor
- Chulalongkorn University
Hemophilia A, Hemophilia B
Psychometric Validation of the Hemophilia Functional Ability Scoring Tool (Hemo-FAST)
The purpose of this study is to validate the newly developed Hemophilia Functional Ability Scoring Tool (Hemo-FAST), which is a fast and simple scoring in haemophilia able to assess patient-reported functional mobility.
- Trial name
- Hemo-FAST (NCT04731701)
- Testing
- Hemophilia Joint Health Score (HJHS)
- Where
- 16 sites in France
- Volunteers
- 180 people
- Sponsor
- Swedish Orphan Biovitrum
Hemophilia A
Assessment of the Risk of Inhibitor Formation in Previously Treated Patients With Severe Hemophilia A
Most transient inhibitor formation, if any, will develop within the first 4 weeks. The study is to further monitor whether participants with severe Hemophilia A will develop inhibitors or antibodies at the later stage when…
- Trial name
- NCT00621673
- Testing
- Kogenate (BAY 14-2222)
- Where
- 2 sites in United States
- Volunteers
- 1 people
- Sponsor
- Bayer
Hemophilia A
Impact of Emicizumab on Activated Clotting Time Using the i-STAT Alinity Analyzer
Emicizumab (Hemlibra®) is a subcutaneous hemostatic treatment approved in 2019 for the prophylaxis in severe hemophilia A. Emicizumab is a bispecific monoclonal antibody mimicking factor VIIIa that provides a constant level of…
- Trial name
- EMISTAT (NCT05856903)
- Testing
- ACT
- Where
- France
- Volunteers
- 15 people, healthy volunteers welcome
- Sponsor
- University Hospital, Grenoble
Severe Hemophilia A Without Inhibitor
Clinical Outcomes of Low Dose PK-guided EHL FVIII Concentrates Versus Standard Prophylaxis in Severe Haemophilia A
Individualised pharmacokinetic (PK)-guided dosing of extended half-life (EHL) FVIII concentrates prophylaxis may reduce hemophilia A bleeding events than previous prophylactic regimen.
- Trial name
- NCT05281185
- Testing
- Extended half-life FVIII concentrates with PK-guided dosing, standard half-life FVIII concentrates with weight-based dosing
- Where
- Thailand
- Volunteers
- 15 people
- Sponsor
- Chulalongkorn University
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
Haemophilia B
A Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B in France
Alprolix (rFIXFc) is a recombinant extended half-life coagulation factor product. The purpose of this non-interventional study is to describe the real-world usage and effectiveness of Alprolix in the on-demand and prophylactic…
- Trial name
- B-SURE (NCT03655340)
- Testing
- Alprolix
- Where
- 18 sites in France
- Volunteers
- 91 people
- Sponsor
- Swedish Orphan Biovitrum
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
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 a with Inhibitors
Individualizing Hemophilia Bypassing Agent Therapy Utilizing Thromboelastography
This pilot study will investigate the use of thromboelastograph (TEG) to determine the primary bypassing agent for the management of bleeding in children and adults severe hemophilia A patients with inhibitors.
- Trial name
- NCT03002480
- Testing
- Thromboelastography-Guided Treatment Regimen
- Sponsor
- Children's Hospital Los Angeles
Hemophilia A With Inhibitor, Hemophilia A With Anti Factor VIII
Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors
This Phase I/II clinical study will evaluate the safety and efficacy of valoctocogene roxaparvovec in patients with severe haemophilia A and inhibitors to FVIII.
- Trial name
- GENEr8-INH (NCT04684940)
- Testing
- Valoctocogene roxaparvovec
- Where
- 9 sites in United States, Brazil and 5 more
- Volunteers
- 10 people
- Sponsor
- BioMarin Pharmaceutical
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
Phase 1/2 Dose Confirmation Study of FLT180a in Hemophilia B
Study of FLT180a gene therapy in adults with Hemophilia B. Up to 9 patients will be enrolled to receive a single dose of FLT180a and be followed for 52 weeks. Results will confirm the dose for a future Phase 3 study.
- Trial name
- NCT05164471
- Testing
- verbrinacogene setparvovec
- Where
- 7 sites in United States, United Kingdom
- Volunteers
- 6 people
- Sponsor
- Spur Therapeutics
Severe Hemophilia A
Efficacy and Safety Study of Human-cl rhFVIII in PTPs With Severe Hemophilia A
This study will determine the efficacy of human-cl rhFVIII in previously treated patients with severe hemophilia A during prophylactic treatment, treatment of bleeding episodes and in surgical prophylaxis.
- Trial name
- NCT01125813
- Testing
- recombinant Factor VIII
- Where
- 11 sites in Austria, Bulgaria and 2 more
- Volunteers
- 32 people
- Sponsor
- Octapharma
Hemophilia A
Pharmacokinetic Assessment of Standard Half-Life (SHL) FVIII, Extended Half-Life (EHL) FVIII, and Efanesoctocog Alfa (BIVV001) in Severe Hemophilia A
Primary objective To assess the half-life of BIVV001, Standard Half-Life (SHL) rFVIII and Extended Half-Life (EHL) rFVIII after a single intravenous (IV) injection Secondary objectives To characterize additional pharmacokinetic…
- Trial name
- NCT05042440
- Testing
- Efanesoctocog alfa, Octocog alfa, Rurioctocog alfa pegol
- Where
- Bulgaria
- Volunteers
- 13 people
- Sponsor
- Sanofi
Hemophilia
Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B
Primary Objective: To confirm appropriate dose levels of fitusiran when administered to male pediatric participants (ages 1 to <12 years of age) with severe hemophilia A or B Secondary Objectives: To characterize the safety and…
- Trial name
- ATLAS-PEDS (NCT03974113)
- Testing
- Fitusiran
- Where
- 16 sites in United States, Canada and 4 more
- Volunteers
- 32 people
- Sponsor
- Genzyme, a Sanofi Company
Thrombosis
Reversibility of Apixaban Anticoagulation With the Four Factor Prothrombin Complex Concentrate Kcentra
Apixaban is an anticoagulant (also known as blood thinner) approved by the Food and Drug Administration (FDA) for reducing the risk of stroke and systemic embolism in patients with non-valvular atrial fibrillation.
- Trial name
- 4PAR (NCT02270918)
- Testing
- Kcentra, Apixaban
- Where
- United States
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Thomas Jefferson University
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