Clinical trials: Hemophilia
Gene Therapy, Hemophilia B
Needs Assessment of Knowledge, Beliefs, and Attitudes of Patients With Hemophilia B About Gene Therapy
Gene therapy is a paradigm-shifting treatment for hemophilia B patients, particularly in resource-limited countries where factor availability remains low.
- Trial name
- NCT05044845
- Testing
- Interview
- Where
- 6 sites in United States, Nepal and 4 more
- Volunteers
- 150 people
- Sponsor
- St. Jude Children's Research Hospital
Hemophilia A, Hemophilia B
UK - EHL Outcomes Registry
Severe haemophilia A and B (SHA, SHB) are inherited bleeding disorders affecting male patients and are characterised by low levels of circulating clotting factors VIII and IX respectively.
- Trial name
- NCT02938156
- Where
- 21 sites in United Kingdom
- Volunteers
- 500 people
- Sponsor
- Royal Free Hospital NHS Foundation Trust
Severe Hemophilia A
A Study of Repeat Dosing of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection
Primary objective: To assess the pharmacokinetics, Safety and immunogenicity of Repeat Dosing of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection With Severe Hemophilia A(FRSW117) Secondary…
- Trial name
- NCT05265286
- Testing
- FRSW117
- Where
- 5 sites in China
- Volunteers
- 15 people
- Sponsor
- Hangzhou Gensciences Biopharmaceutical Co., Ltd.
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
Assessing Physician and Hemophilia A Patient Reasons and Expectations for Switching Treatment to Kovaltry & Jivi: A Nested Study Within an Existing Registry
This US study aims to assess hemophilia A patient characteristics and reasons for switching from both patient/caregiver and physician perspectives.
- Trial name
- NCT03603275
- Testing
- New FVIII products
- Where
- 27 sites in United States
- Volunteers
- 3 people
- Sponsor
- Bayer
Hemophilia A
Safety and Efficacy Study of NGGT003 in Hemophilia A Patients
This is an early phase 1, open-label, single-center, dose-escalation pilot trial to evaluate the safety and efficacy of an intravenous infusion of NGGT003 in hemophilia A patients.
- Trial name
- NCT06238908
- Testing
- NGGT003
- Where
- China
- Volunteers
- 6 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Joint Bleed, Hemophilia A
Handheld Ultrasound (HHUS) for Home Use in Hemophilia
This is a study in 3 phases that will assess the feasibility and utility of handheld home ultrasound (HHUS) in a patient's home to assess whether a painful episode in the elbow, knee or ankle is a bleed or not.
- Trial name
- HHUS (NCT04131920)
- Testing
- handheld ultrasound: Philips Lumify integrated tele-ultrasound powered by Reacts collaborative platform
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Washington Institute for Coagulation
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
Von Willebrand Diseases, Hemophilia A
BT200 in Hereditary Bleeding Disorders
BT200 is a PEGylated aptamer that binds to the A1 domain of human von Willebrand factor (VWF). At low doses, BT200 blocks the clearance of VWF antigen (VWF Ag) from the circulation and causes an increase in concentrations of both…
- Trial name
- NCT04677803
- Testing
- BT200
- Where
- Austria
- Volunteers
- 26 people
- Sponsor
- Medical University of Vienna
MRI, Hemophilia
MRI Role in Knee Hemophilic Arthopathy
The aim of this study is to assess role of MRI in detecting synovial, cartilaginous , osseous abnormalities ، bleeding inside knee joint and to use a system for assessing HA as support for therapeutic regimes and for monitoring…
- Conditions
- MRI, Hemophilia, Arthropathy Hemophilic, Arthropathy of Knee
- Trial name
- NCT07233122
- Volunteers
- 95 people
- Sponsor
- Assiut University
Hemophilia B
Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222)
The primary purpose of this study is to assess the long-term safety and efficacy in male adults with hemophilia B who were treated with CSL222 (CSL222) in parent studies CSL222_2001 (NCT03489291) or CSL222_3001 (NCT03569891).
- Trial name
- IX-TEND 3003 (NCT05962398)
- Testing
- AAV5-hFIXco-Padua
- Where
- 28 sites in United States, Belgium and 6 more
- Volunteers
- 56 people
- Sponsor
- CSL Behring
Hemophilia Arthropathy
Pain and Joint Status With Psychosocial Factors in Patients With Hemophilia
Background. The recurrence of hemarthrosis in patients with congenital coagulopathies favors the development of a progressive, degenerative intra-articular lesion (hemophilic arthropathy) that mainly affects the knees, ankles and…
- Trial name
- NCT04715100
- Testing
- Observational group
- Where
- Spain
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Investigación en Hemofilia y Fisioterapia
Healthy
A Study to Assess the Effects of 2 Different Prothrombin Complex Concentrates on the Pharmacodynamics of Rivaroxaban in Healthy Adult Volunteers
The purpose of this study is to assess the use of 2 different Prothrombin Complex Concentrates (PCCs) on their ability to reverse (normalize) the pharmacodynamic effects of rivaroxaban in healthy adult volunteers.
- Trial name
- NCT01656330
- Testing
- Rivaroxaban 20 mg twice daily, Rivaroxaban 20 mg once daily, Profilnine SD
- Where
- Belgium
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- Janssen Research & Development, LLC
Hemophilia A and B, Periodontitis
Effect of Non-Surgical Periodontal Treatment on Oral Health in Hemophilia Patients
This study aimed to evaluate the effect of non-surgical periodontal treatment (NSPT) on oral health-related quality of life in patients with hemophilia using the Oral Health Impact Profile-14 (OHIP-14) questionnaire.
- Conditions
- Hemophilia A and B, Periodontitis, Hemophilia
- Trial name
- NCT07691437
- Testing
- Non-surgical periodontal treatment
- Where
- Turkey (Türkiye)
- Volunteers
- 146 people, healthy volunteers welcome
- Sponsor
- Karadeniz Technical University
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
Congenital Bleeding Disorder, Haemophilia A
A Multi-centre, Open Labelled, Multiple Dosing Trial Investigating Safety, Pharmacokinetics and Pharmacodynamics of NNC 0172-2021 Administered Subcutaneously to Healthy Male Subjects and Haemophilia Subjects
This trial is conducted in Europe. The aim of this trial is to investigate safety, pharmacokinetics (the exposure of the trial drug in the body) and pharmacodynamics (the effect of the investigated drug on the body) of NNC…
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Healthy
- Trial name
- explorer™2 (NCT01631942)
- Testing
- NNC172-2021
- Where
- 6 sites in Austria, France and 3 more
- Volunteers
- 4 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
Hemophilia A
A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)
The BMN 270 clinical development program consists of multiple interventional studies designed to assess the safety and efficacy of a single infusion of BMN 270 for at least 5 years post-infusion.
- Trial name
- GENEr8-LTE (NCT05768386)
- Where
- 41 sites in United States, Australia and 10 more
- Volunteers
- 172 people
- Sponsor
- BioMarin Pharmaceutical
Hemophilia, Arthropathy
PET Imaging of Hemophilic Arthropathy
Repeated hemarthroses in patients with hemophilia may lead to hemophilic arthropathy with marked inflammation and synovial hypertrophy.
- Conditions
- Hemophilia, Arthropathy, Inflammation
- Trial name
- NCT03485170
- Testing
- 18F-FDG PET/CT
- Where
- Taiwan
- Volunteers
- 20 people
- Sponsor
- Tri-Service General Hospital (TSGH)
Hemophilia B
Prospective Registry of European Hemophilia B Patients Receiving BeneFIX® for Usual Use
This is an open-label multi-center Registry in patients with hemophilia B receiving BeneFIX. All patients who begin treatment with BeneFIX in European Union countries, will be eligible for participation.
- Trial name
- NCT00167973
- Where
- 90 sites in Austria, Belgium and 8 more
- Volunteers
- 218 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Hemophilia a, Hemophilia B
The Efficacy and Safety of SerpinPC in Participants with Severe Hemophilia a or Moderately Severe to Severe Hemophilia B
The purpose of the study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of prophylactic SerpinPC administered subcutaneously (SC) to participants with severe hemophilia A (HemA) (with or without…
- Trial name
- PRESent-2 (NCT05789524)
- Testing
- SerpinPC
- Where
- 59 sites in United States, Armenia and 13 more
- Volunteers
- 60 people
- Sponsor
- ApcinteX Ltd