Clinical trials: Hemophilia a
Haemophilia A
A Study Following Males With Haemophilia A on Prophylaxis With Esperoct®
This study will collect information on side effects and how well Esperoct® (turoctocog alfa pegol (N8-GP)) works during long-term treatment (prophylaxis) in males with haemophilia A.
- Trial name
- pathfinder9 (NCT04574076)
- Testing
- Turoctocog alfa pegol (N8-GP)
- Where
- 27 sites in Austria, Bulgaria and 13 more
- Volunteers
- 60 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A
National Study of a Pharmacokinetic-Focused Educational Package for Patients With Severe Haemophilia A
Prospective, interventional study of pharmacokinetic (PK)-focused FVIII dosing discussions delivered as part of a patient education package.
- Trial name
- UK-PK (NCT02528968)
- Testing
- Educational Package
- Where
- United Kingdom
- Volunteers
- 59 people
- Sponsor
- Hampshire Hospitals NHS Foundation Trust
Acquired Hemophilia A
Trial of Acquired Haemophilia With Steroid Combined With Cyclophosphamide Versus Steroid Combined With Rituximab
Purpose: To evaluate the efficacy when administering steroid combined with single dose rituximab to eliminate the antibody in acquired hemophilia A patients compared to treatment using steroid with cyclophosphamide.
- Trial name
- NCT03384277
- Testing
- Steroid, Rituximab, Cyclophosphamide
- Where
- China
- Volunteers
- 66 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Hemophilia A
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)
This Phase III clinical study will assess the impact of BMN 270 (compared to FVIII prophylaxis) on the number of bleeding episodes irrespective of exogenous FVIII replacement treatment in the efficacy evaluation period (EEP)…
- Trial name
- BMN 270-301 (NCT03370913)
- Testing
- valoctocogene roxaparvovec
- Where
- 49 sites in United States, Australia and 11 more
- Volunteers
- 144 people
- Sponsor
- BioMarin Pharmaceutical
Hemophilia
What is the Feasibility of the ISTEP Exercise Test in Boys With Haemophilia
This study will be used to assess the feasibility and sensitivity of using the iSTEP, to assess exercise capacity in boys with haemophilia. The feasibility criteria to be explored includes: 1.
- Trial name
- NCT04076306
- Testing
- iSTEP exercise testing
- Where
- United Kingdom
- Volunteers
- 43 people
- Sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust
Hemophilia
Blood Flow Restriction in Improving Muscle Strength of Patients With Hemophilic Ankle Arthropathy
Background. The main physical sequela of patients with hemophilia is the development of a progressive, degenerative intra-articular lesion, known as hemophilic arthropathy).
- Trial name
- NCT06261593
- Testing
- Blood flow restriction
- Where
- Spain
- Volunteers
- 31 people
- Sponsor
- Investigación en Hemofilia y Fisioterapia
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 A
A Study to Evaluate Seroprevalence and Seroconversion of Antibodies to Adeno-Associated Virus (AAV) in Patients With Hemophilia A
To quantify the seroprevalence of antibodies to AAV5, AAV6, and AAV8 and the seroconversion rate over varying follow-up intervals in subjects with hemophilia A
- Trial name
- SAAVY (NCT04560933)
- Testing
- Blood sample collection
- Where
- United States
- Volunteers
- 186 people
- Sponsor
- BioMarin Pharmaceutical
Bone Mineral Density, Hemophilia A
Osteoporosis and MRI Study in Hemophilia
The purpose of this study is to evaluate bone mineral density in adult subjects with hemophilia versus a comparator population without hemophilia (non-hemophilia age- and gender-matched database) by using the following diagnostic…
- Conditions
- Bone Mineral Density, Hemophilia A, Hemophilia B
- Trial name
- NCT01460147
- Testing
- DXA scan + MRI
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Baxalta now part of Shire
Hemarthrosis, Knee, Hemophilia
Effects of Photobiomodulation in Hemophilia Patients.
Hemophilia is a genetic bleeding disorder that commonly leads to knee hemarthrosis, causing pain, swelling, and reduced joint mobility in children.
- Trial name
- NCT07579585
- Testing
- photobiomodulation
- Where
- Pakistan
- Volunteers
- 24 people
- Sponsor
- Riphah International University
Mild Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor
A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors
This is a multicenter, open-label, single-arm study designed to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of emicizumab in participants with mild or moderate hemophilia A without inhibitors against…
- Conditions
- Mild Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor, Hemophilia A
- Trial name
- HAVEN 6 (NCT04158648)
- Testing
- Emicizumab
- Where
- 22 sites in United States, Belgium and 8 more
- Volunteers
- 73 people
- Sponsor
- Hoffmann-La Roche
Hemophilia A, Hemophilia
Pharmacokinetic Characterization of the Hemophilia A Population in Spain Using myPKFiT®
The purpose of this study is to describe the pharmacokinetic profile of patients with hemophilia A in prophylaxis in Spain using myPKFit®
- Conditions
- Hemophilia A, Hemophilia, Factor VIII Deficiency
- Trial name
- NCT03006965
- Testing
- octocog alfa, rurioctocog alfa pegol
- Where
- 14 sites in Spain
- Volunteers
- 50 people
- Sponsor
- Spanish Society of Thrombosis and Haemostasis
Hemophilia A
Quality of Life Study of Helixate NexGen
The aim of this study is to describe Health Related Quality of Life (HRQoL) in adolescents and adults with Hemophilia A treated prophylactically or on-demand with Helixate NexGen.
- Trial name
- NCT01053715
- Where
- 31 sites in Austria, Belgium and 5 more
- Volunteers
- 78 people
- Sponsor
- CSL Behring
Hemophilia A
An Open Label Study to Determine the Safety and Efficacy of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection in Adolescent and Adult Patients With Hemophilia A.
The primary objectives of the study are to evaluate the efficacy of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection (FRSW107) in the Prevention and Treatment of Bleeding in patients with hemophilia A.
- Trial name
- NCT04456387
- Testing
- Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection
- Where
- 17 sites in China
- Volunteers
- 119 people
- Sponsor
- Zhengzhou Gensciences Inc
Congenital Bleeding Disorder, Haemophilia A With Inhibitors
A Trial Evaluating the Efficacy and Safety of Prophylactic Administration of Concizumab in Haemophilia A and B Patients With Inhibitors
This trial is conducted in Africa, Asia, Europe and North America. The aim of the trial is to assess the efficacy of concizumab administered s.c.
- Conditions
- Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
- Trial name
- explorer™4 (NCT03196284)
- Testing
- Concizumab, Eptacog alfa
- Where
- 22 sites in United States, Austria and 12 more
- Volunteers
- 26 people
- Sponsor
- Novo Nordisk A/S
Hemophilia B
An International Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B
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-MORE (NCT03901755)
- Testing
- Alprolix
- Where
- 29 sites in Czechia, Greece and 7 more
- Volunteers
- 151 people
- Sponsor
- Swedish Orphan Biovitrum
Hemophilia a
A Prospective Assessment of Bone Health in Patients With Severe Hemophilia A on Factor VIII vs Factor Mimetic Prophylaxis (Efa Emi Bone Health Study)
This study will compare bone mineral density in patients with severe hemophilia A receiving prophylaxis with emicizumab or efanesoctocog alfa.
- Trial name
- NCT07582276
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Arkansas Children's Hospital Research Institute
Chronic Coronary Syndrome, Heart Failure
COVID-19 Related Lockdown Effects On Chronic Diseases
The containment associated with the VIDOC-19 pandemic creates an unprecedented societal situation of physical and social isolation.
- Conditions
- Chronic Coronary Syndrome, Heart Failure, AMD and Macular Edema, Chronic Respiratory Failure
- Trial name
- CLEO-CD (NCT04390126)
- Testing
- life questionnaires, questionnaire
- Where
- France
- Volunteers
- 1,343 people
- Sponsor
- Centre Hospitalier Universitaire Dijon
Hemophilia A
GAS-Hem Feasibility Study
The purpose of this study is to investigate the feasibility and acceptability of the Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem) as a patient reported outcome (PRO) measure to monitor clinical progress in…
- Trial name
- NCT02674997
- Testing
- Factor VIII
- Where
- 4 sites in United States, Canada
- Volunteers
- 44 people
- Sponsor
- Baxalta now part of Shire
Treatment of Bleeding, Prophylaxis of Bleeding
A Study to Learn How Safe the Study Treatment BAY94-9027 is and How it Affects the Body in Previously Treated Children Aged 7 to Less Than 12 Years With Severe Hemophilia A, a Genetic Bleeding Disorder That is Caused by the Lack of a Protein Called Clotting Factor 8 (FVIII) in the Blood
Researchers are looking for a better way to treat hemophilia A. Hemophilia A is a genetic disorder where the body does not create enough of a protein called clotting factor 8 (FVIII) present in the blood.
- Conditions
- Treatment of Bleeding, Prophylaxis of Bleeding, Hemophilia A, Children
- Trial name
- Alfa-PROTECT (NCT05147662)
- Testing
- Damoctocog alfa pegol (Jivi, BAY94-9027)
- Where
- 18 sites in United States, Argentina and 5 more
- Volunteers
- 36 people
- Sponsor
- Bayer