Clinical trials: Hemophilia a
Hemophilia a, Hemophilia B
Study of TU7710 in Warfarin Anti-coagulated Healthy Male Subjects
This is a Phase 1a, double-blind, randomized, placebo- controlled, SAD study to assess safety, tolerability, PK, and PD of TU7710 in warfarin treated healthy male participants.
- Conditions
- Hemophilia a, Hemophilia B, Blood Coagulation Disorders
- Trial name
- NCT06025552
- Testing
- TU7710, Normal saline
- Where
- South Korea
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- TiumBio Co., Ltd.
Hemophilia A
Optimizing the Use of Prophylaxis in Patients With Severe Haemophilia A
MyPKFiT is a web-based application recently developed by Baxalta for the use in patients treated with Advate. MyPkFit has its basis in Bayesian forecasting, which allows estimation of individual PK parameters by a sparse sampling…
- Trial name
- myPKFiT (NCT03915080)
- Testing
- Oktokog alpha
- Where
- Sweden
- Volunteers
- 14 people
- Sponsor
- Lund University
Hemophilia A
Study Evaluating B-Domain Deleted Recombinant Factor VIII (BDDrFVIII, ReFacto AF) in Patients With Hemophilia A Undergoing Elective Major Surgery
This study will examine the efficacy and safety of ReFacto AF in patients with severe and moderately severe hemophilia A undergoing elective major surgery when administered by either bolus injections (BI) or continuous infusion…
- Trial name
- NCT00243659
- Testing
- ReFacto AF
- Where
- 17 sites in United States, Australia and 6 more
- Volunteers
- 30 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
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
Study of a pd vWF/FVIII, Biostate®, in Subjects With Haemophilia A
The aim of this study are to assess the efficacy of Biostate® [Study Product (SP)] in subjects with Haemophilia A compare the pharmacokinetics of Biostate® [SP] with the previously marketed product Biostate® (here referred to as…
- Trial name
- NCT00879541
- Testing
- Biostate® [SP], Biostate® [RP]
- Where
- 14 sites in Bulgaria, North Macedonia and 2 more
- Volunteers
- 81 people
- Sponsor
- CSL Behring
Hemophilia A
Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults
C3731003 is a pivotal Phase 3 study to evaluate the clinical efficacy and safety of a single IV infusion of PF-07055480 / giroctocogene fitelparvovec (Recombinant AAV2/6 Human Factor VIII Gene Therapy) in adult male participants…
- Trial name
- AFFINE (NCT04370054)
- Testing
- PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy
- Where
- 45 sites in United States, Australia and 14 more
- Volunteers
- 77 people
- Sponsor
- Pfizer
Congenital Bleeding Disorder, Haemophilia A
To Quantify the Range of Main Psychosocial Factors Affecting Patients and Caregivers in Their Daily Lives
This survey is conducted in South America. The purpose is to identify the key psychosocial issues affecting patients with haemophilia.
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia B, Haemophilia A With Inhibitors
- Trial name
- HERO (NCT02241694)
- Testing
- No treatment given
- Where
- Brazil
- Volunteers
- 200 people
- Sponsor
- Novo Nordisk A/S
Severe Hemophilia A, Osteoporosis
Bone Microarchitecture in Men With Hemophilia
Hemophilia A and B are hereditary sex-linked deficiencies of coagulation factors VIII and IX characterized by bleeding. Their modern therapy increases life expectancy and risk of age-related diseases, e.g., osteoporosis.
- Trial name
- HEMOS (NCT05127681)
- Testing
- HR-pQCT, Blood sample, Dual energy X-ray absorptiometry
- Where
- France
- Volunteers
- 10 people
- Sponsor
- Hospices Civils de Lyon
Hemophilia A
Phase 3 Efficacy and Safety Study of BAX 855 in Severe Hemophilia A Patients Undergoing Surgical Procedures
The purpose of the study is to evaluate the efficacy and safety of BAX 855 in severe hemophilia A previously treated (PTP) males, 12 to 65 years of age who are undergoing elective surgical or other invasive procedures.
- Trial name
- NCT01913405
- Testing
- PEGylated Recombinant factor VIII (rFVIII)
- Where
- 22 sites in United States, Bulgaria and 7 more
- Volunteers
- 30 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A, Hemophilia B
Study of Coagulation Faction VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia
This multi-center, open label Phase 1 study will evaluate the pharmacokinetics, pharmacodynamics, and safety of a single IV dose of MarzAA followed by ascending single SC doses of MarzAA in adult subjects with moderate or severe…
- Conditions
- Hemophilia A, Hemophilia B, Hemophilia A With Inhibitor, Hemophilia B With Inhibitor
- Trial name
- NCT04072237
- Testing
- MarzAA (marzeptacog alfa [activated])
- Where
- 5 sites in Bulgaria, Russia
- Volunteers
- 11 people
- Sponsor
- Catalyst Biosciences
Hemophilia a
EVALUATION of the OVERALL HAEMOSTATIC CAPACITY of MIM8 with GLOBAL HAEMOSTASIS ASSAYS and FIBRIN CLOT ULTRASTRUCTURE
Hemophilia A is an X linked disorder characterized by a deficiency in Factor VIII. The clinical hallmark of this disease is increased tendency to spontaneous bleeding with hemarthrosis accounting for 90% of the hemorrhages.
- Trial name
- Mim8-TGT (NCT06212505)
- Testing
- blood test
- Where
- France
- Volunteers
- 6 people
- Sponsor
- Hospices Civils de Lyon
Hemophilia A, Hemophilia A, Congenital
Searching Patterns In the Robustness of Immunological FVIII Tolerance
Children with hemophilia A lack clotting factor VIII (FVIII) due to a genetic mutation. It is well known that administration of FVIII concentrate leads to immunological tolerance for the FVIII protein in the majority of children.
- Trial name
- SPIRIT (NCT07539402)
- Where
- Netherlands
- Volunteers
- 500 people
- Sponsor
- Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Multiple Sclerosis, Relapse
Observational Trial Evaluating Elevated Factor VIII Related Labs as a Biomarker for Incomplete Relapse Recovery
Nine Multiple Sclerosis (MS) patients suffering an acute relapse from the outpatient or inpatient settings will be consented to be followed prospectively for three months post relapse, in an effort to identify markers of…
- Trial name
- NCT05926167
- Testing
- blood tests drawn days 1, 8, 22 and 90 of the study
- Volunteers
- 9 people
- Sponsor
- Dignity Health Medical Foundation
Congenital Bleeding Disorder, Haemophilia B
A Trial Comparing Nonacog Beta Pegol (N9-GP) and ALPROLIX® in Patients With Haemophilia B
This trial is conducted in Europe and the United States of America. The aim of this trial is to compare the pharmacokinetics (the exposure of the trial drug in the body) of nonacog beta pegol (N9-GP) and ALPROLIX® in patients…
- Trial name
- paradigm™7 (NCT03075670)
- Testing
- N9-GP, ALPROLIX®
- Where
- 12 sites in United States, Germany and 1 more
- Volunteers
- 15 people
- Sponsor
- Novo Nordisk A/S
Von Willebrand Disease (VWD), Inherited Bleeding Disorders in Children
Clinical Spectrum and Management of Von Willebrand Disease Among Children in Assiut Governorate
Von Willebrand disease (VWD) is the most common inherited bleeding disorder in children. It occurs due to a deficiency or dysfunction of von Willebrand factor, a protein that plays an essential role in blood clotting.
- Conditions
- Von Willebrand Disease (VWD), Inherited Bleeding Disorders in Children, Pediatric Hemostatic Disorders
- Trial name
- VWD-ASSIUT (NCT07410130)
- Testing
- Tranexamic Acid, Von Willebrand Factor-Containing Concentrates
- Where
- Egypt
- Volunteers
- 25 people
- Sponsor
- Assiut University
Hemophilia
A Study to Learn About the Study Medicine (Called Marstacimab) in People With Hemophilia A or B
The purpose of this non-interventional, observational, real-world study is to find out what are the real-world experiences of people living with hemophilia A or B[OK1.1] who start the study medicine (called marstacimab) in…
- Trial name
- NCT07859527
- Testing
- Marstacimab
- Volunteers
- 100 people
- Sponsor
- Pfizer
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
Does the Thrombin Generation Test Performed During the Pharmacokinetic Profile of the Substitutive Factor VIII Bring Benefits to the Personalized Treatment of Pediatric Patients and Adult Hemophilia A Patients Under Prophylaxis ?
In the context of hemophilia, it is well know that the level of factor VIII alone does not reflect the clinical phenotype of the patients in an accurate way.
- Trial name
- NCT02803502
- Testing
- Chronometric method, Chromogenic method, Thrombin generation test (TGT)
- Where
- 5 sites in Belgium
- Volunteers
- 50 people
- Sponsor
- Brugmann University Hospital
Hemophilia A
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg
This Phase III clinical study will assess the efficacy of BMN 270 defined as FVIII activity, during weeks 49-52 following intravenous infusion of BMN 270 and assess the impact of BMN 270 on usage of exogenous FVIII replacement…
- Trial name
- GENEr8-2 (NCT03392974)
- Testing
- Valoctocogene Roxaparvovec
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- BioMarin Pharmaceutical
Hemophilia
Restricting Blood Flow in Improving Muscle Strength in Patients With Hemophilic 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
- NCT05039008
- Testing
- Blood flow restriction
- Where
- Spain
- Volunteers
- 23 people, healthy volunteers welcome
- Sponsor
- Investigación en Hemofilia y Fisioterapia