Clinical trials: Hemophilia a
Hemophilia, Hemophilia A, Severe
Reliability and Validity of the Turkish Version of the PedHAL
This study aimed to establish the validity and reliability of the Turkish version of the Pediatric Haemophilia Activities List (short - PedHALshort) questionnaire.
- Conditions
- Hemophilia, Hemophilia A, Severe, Hemophilia B
- Trial name
- NCT05889754
- Where
- Turkey (Türkiye)
- Volunteers
- 50 people
- Sponsor
- Hasan Kalyoncu University
Hemophilia A, Factor VIII
POCUS: Hemostatic Potential and Joint Health in Patients With Severe Hemophilia A on Novel Replacement Therapies
This is a prospective, randomized control trial in which each patient will be randomly assigned to receive either extended half-life factor VIII based replacement therapy or non-FVIII based replacement therapy, which are both…
- Trial name
- NCT04690322
- Testing
- Eloctate, Adynovate, Emicizumab
- Where
- United States
- Sponsor
- University of Texas Southwestern Medical Center
Sonography, Haemophilia
Sonography and Haemophilia
The aim of this study is to evaluate the possibility of establishing a patient-based ultrasound monitoring of the knee (representing a target joint accessible with any hand) with a handheld device: The following questions will be…
- Trial name
- NCT04550988
- Testing
- feasibility study
- Where
- Germany
- Volunteers
- 5 people
- Sponsor
- University Hospital, Bonn
Hemophilia A; Hemophilia B
A Study to Assess Pharmacokinetics and Pharmacodynamics Following Administration of BAY1093884 in Patients With Severe Hemophilia
The primary objective of this study is to assess the pharmacokinetics in patients with severe hemophilia. The secondary objective is to assess the pharmacodynamics of BAY1093884 based on tissue factor pathway inhibitor activity
- Trial name
- NCT03481946
- Testing
- BAY1093884
- Where
- Israel
- Volunteers
- 6 people
- Sponsor
- Bayer
Hemophilia A, Gene Therapy
Gene Therapy for Chinese Hemophilia A
IHBDH-GTHA-2020 is an open- label, non- randomized study to evaluate the safety, tolerability and kinetics of a single intravenous infusion of GS001 in hemophilia A subjects with <1 IU/dl residual FVIII levels.
- Trial name
- HA (NCT04728841)
- Testing
- Injection of GS001
- Where
- China
- Volunteers
- 12 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Hemophilia A
Nuwiq Dosing and Outcomes In the ManagEment of Women/Girls With Haemophilia A Needing FVIII Treatment for Surgery
Recombinant factor VIII for the prevention of bleeding in women/girls with haemophilia A undergoing major surgery
- Trial name
- NCT05936580
- Testing
- Nuwiq
- Where
- 16 sites in United States, Finland and 8 more
- Volunteers
- 28 people
- Sponsor
- Octapharma
Von Willebrand Disease
Pharmacokinetic, Safety and Tolerability Study of Recombinant Von Willebrand Factor / Recombinant Factor VIII Complex in Type 3 Von Willebrand Disease
The objectives of this study are to evaluate the immediate tolerability and safety of rVWF:rFVIII in subjects with Type 3 Von Willebrand Disease after administration of various dosages of VWF:RCo.
- Trial name
- NCT00816660
- Testing
- Recombinant von Willebrand factor : recombinant FVIII (rVWF:rFVIII), Marketed plasma-derived VWF/FVIII concentrate
- Where
- 25 sites in United States, Austria and 4 more
- Volunteers
- 32 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A, Hemophilia B
A Phase 2/ 3 Trial to Evaluate the Efficacy and Safety of BAY86-6150
Haemophilia is a disorder, usually genetic, affecting mostly male individuals, in which one of the proteins needed to form blood clots (FVIII) is missing or not present in sufficient levels.
- Trial name
- NCT01625390
- Testing
- BAY86-6150, eptacog alfa [activated]
- Where
- 61 sites in United States, Australia and 27 more
- Volunteers
- 10 people
- Sponsor
- Bayer
Haemophilia
Defeat Rare Disease in Asia and Pacific (APAC) DRDA - Haemophilia
This study will help to better understand the course of disease in people with haemophilia (A or B [with or without inhibitors]) and the haemophilia care scenario in the selected countries and the experience so far in dealing…
- Trial name
- NCT07163260
- Testing
- No treatment given
- Where
- 4 sites in India, Malaysia and 2 more
- Volunteers
- 244 people
- Sponsor
- Novo Nordisk A/S
Severe Hemophilia A, Moderate Hemophilia A
A Study to Evaluate Overall Health, Physical Activity, and Joint Outcomes in Participants With Severe or Moderate Hemophilia A Without Factor VIII Inhibitors on Emicizumab Prophylaxis
Study MO42623 is a Phase IV, multicenter, open-label, three cohort study designed to evaluate the impact of emicizumab prophylaxis on overall health, physical activity, and joint outcomes in participants aged ≥13 and <70 years…
- Trial name
- Beyond ABR (NCT05181618)
- Testing
- Emicizumab
- Where
- 26 sites in United States, Brazil and 10 more
- Volunteers
- 136 people
- Sponsor
- Hoffmann-La Roche
Hemophilia, Hemorrhage
Topical and Local TXA in Facelifts - A Randomized Controlled Double Blinded Study
Tranexamic acid (TXA) is a fibrinolytic inhibitor which prevents prolonged bleeding by interfering with fibrin clot breakdown by competitively binding to lysine receptors on plasminogen; this prevents the conversion of…
- Conditions
- Hemophilia, Hemorrhage, Facelift Surgery
- Trial name
- NCT06345833
- Testing
- 1%Tranexamic acid with standard local, 3% TXA, 1% TXA with local plus 3% TXA-soaked pledgets
- Where
- United States
- Sponsor
- University of Minnesota
Type 3 Von Willebrand's Disease
Type 3 Von Willebrand International Registries Inhibitor Prospective Study
International Registries and Prospective Study on Type 3 Von Willebrand's Disease (VWD3), aimed to assess number, types and risk factors for bleeding and the efficacy and safety of plasma-derived and/or recombinant Von Willebrand…
- Trial name
- 3WINTERS-IPS (NCT02460458)
- Testing
- Von Willebrand Factor
- Where
- 25 sites in Finland, France and 8 more
- Volunteers
- 265 people
- Sponsor
- Fondazione Angelo Bianchi Bonomi
Hemophilia
Individualized Prophylaxis for Severe Hemophilia A Children
A multi centre two year long term escalating dose tertiary prophylaxis study on the efficacy and cost saving of individualized low dose prophylaxis regimens for boys with severe hemophilia A in China staring with a low dose…
- Trial name
- NCT02116855
- Testing
- according to the efficacy of AJBRs
- Volunteers
- 50 people
- Sponsor
- Beijing Children's Hospital
Hemophilia A
A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia A
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of NXT007 prophylaxis compared with Factor VIII (FVIII) prophylaxis in participants with severe or moderate congenital…
- Trial name
- ZEBRHA 1 (NCT07416526)
- Testing
- NXT007, Human Coagulation Factor VIII
- Where
- 36 sites in United States, Argentina and 16 more
- Volunteers
- 138 people
- Sponsor
- Hoffmann-La Roche
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
Severe Haemophilia A
Gene Therapy Study in Severe Haemophilia A Patients (270-201)
This study is being conducted by BioMarin Pharmaceutical Inc. as an open label, dose escalation study in order to determine the safety and efficacy of valoctocogene roxaparvovec (an Adenovirus-Associated Virus based gene therapy…
- Trial name
- NCT02576795
- Testing
- valoctocogene roxaparvovec
- Where
- 5 sites in United Kingdom
- Volunteers
- 15 people
- Sponsor
- BioMarin Pharmaceutical
Hemophilia, Inhibitors
PCC Treatment for Hemophilia Patients With Inhibitor(2022PCC-A)
This study is a multicenter, prospective, single-arm exploratory clinical trial designed to evaluate the efficacy and safety of prothrombin complex concentrate (PCC) in the treatment of bleeding episodes in patients with…
- Trial name
- 2022PCC-A (NCT07406139)
- Testing
- PCC
- Where
- China
- Volunteers
- 30 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
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 A With Inhibitor, Haemophilia A Without Inhibitor
ATHN 7: Hemophilia Natural History Study
This is a real-world study of the safety of the treatments used for people with hemophilia. The study will follow people with hemophilia A or B from across the country for about 4 years as they receive treatment.
- Conditions
- Hemophilia A With Inhibitor, Haemophilia A Without Inhibitor, Hemophilia B With Inhibitor, Haemophilia B Without Inhibitor
- Trial name
- ATHN 7 (NCT03619863)
- Where
- 29 sites in United States
- Volunteers
- 395 people
- Sponsor
- American Thrombosis and Hemostasis Network
Hemophilia A
BAX 855 Dose-Escalation Safety Study
The objectives of this study are to assess the tolerability and safety after single dose treatments of BAX 855 in previously treated patients (PTPs) with severe hemophilia A, to determine the pharmacokinetic (PK) parameters of…
- Trial name
- NCT01599819
- Testing
- Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method, PEGylated Recombinant Factor VIII
- Where
- 8 sites in Bulgaria, Germany and 2 more
- Volunteers
- 19 people
- Sponsor
- Baxalta now part of Shire