Clinical trials: Severe Hemophilia A
Hemophilia A
A Trial Investigating Safety and Efficacy of Treatment With BAY94-9027 in Severe Hemophilia A
Haemophilia A is an inherited disorder in which one of the proteins, Factor VIII, needed to form blood clots is missing or not present in sufficient levels.
- Trial name
- PROTECT-VIII (NCT01580293)
- Testing
- BAY94-9027
- Where
- 59 sites in United States, Austria and 18 more
- Volunteers
- 145 people
- Sponsor
- Bayer
Hemophilia
In Vitro Correction of Thrombin Generation by Concizumab (Anti-TFPI) for Severe Hemophilia Patients
Studies have shown, in haemophilia patients, the effectiveness of Anti TFPI antibody (aTFPIAb) to prevent joint and muscular bleeding (tissues poor in Tissue Factor (TF)).
- Trial name
- NCT05617209
- Testing
- thrombin generation measurement
- Where
- France
- Volunteers
- 20 people
- Sponsor
- Centre Hospitalier Universitaire de Saint Etienne
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
Safety/Efficacy Study to Assess Whether FVIII/VWF Concentrate Can Induce Immune Tolerance in Haemophilia A Patients
The purpose of this study is to assess the role of a FVIII/VWF complex concentrate (Emoclot) in successfully inducing immune tolerance (I.T.I.) in patients with Haemophilia A with inhibitors, including patients at high risk of…
- Trial name
- ITI (NCT02479087)
- Testing
- Plasma-derived FVIII/VWF concentrate
- Where
- 4 sites in Egypt, India
- Volunteers
- 20 people
- Sponsor
- Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Hemophilia, Arthropathy of Knee
Evaluation of Static Postural Balance in Children With Hemophilia and Its Relationship With Joint Health
Hemophilia is a rare hematological disorder characterized by a partial or complete deficiency of clotting factor VIII or IX.Bleeding disorders mainly affect the musculoskeletal system.
- Conditions
- Hemophilia, Arthropathy of Knee, Arthropathy of Ankle or Foot
- Trial name
- NCT04406519
- Where
- Turkey (Türkiye)
- Volunteers
- 42 people, healthy volunteers welcome
- Sponsor
- Necmettin Erbakan University
Hemophilia A
Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A
Primary Objective: To evaluate the long-term safety of BIVV001 in previously treated subjects with hemophilia A Secondary Objectives: To evaluate the efficacy of BIVV001 as a prophylaxis treatment.
- Trial name
- XTEND-ed (NCT04644575)
- Testing
- efanesoctocog alfa (BIVV001)
- Where
- 85 sites in United States, Argentina and 21 more
- Volunteers
- 261 people
- Sponsor
- Bioverativ, a Sanofi company
Hemophilia A
A Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously Received Preventive Treatment With Emicizumab
This is an exploratory, single group, Phase 4, study to assess treatment with fitusiran prophylaxis after switching from emicizumab prophylaxis.
- Trial name
- NCT06145373
- Testing
- Fitusiran (SAR439774), Clotting factor concentrates (CFC) or bypassing agents (BPA), Antithrombin concentrate (ATIIIC)
- Where
- 3 sites in United States, Taiwan
- Volunteers
- 20 people
- Sponsor
- Sanofi
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 B
A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B
Participants in this study have a genetic mutation, specifically in the coagulation (blood clotting) Factor 9 gene that causes severe or moderately severe hemophilia B.
- Trial name
- BEYOND-9 (NCT06379789)
- Testing
- REGV131, LNP1265
- Where
- 52 sites in United States, Australia and 7 more
- Volunteers
- 130 people
- Sponsor
- Regeneron Pharmaceuticals
Severe Hemophilia A Without Inhibitor, Joint Bleed
Comparison of Outcomes Between Low Dose Emicizumab and Factor VIII in Clinically Severe Hemophilia A
To outcome between low dose Emicizumab and low dose prophylaxis with FVIII concentration To study pharmacokinetic, side effect of low dose Emicizumab
- Trial name
- NCT06155955
- Testing
- "Emicizumab", "HEMLIBRA®"
- Where
- Thailand
- Volunteers
- 15 people, healthy volunteers welcome
- Sponsor
- Chulalongkorn University
Hemophilia A
Evaluate Efficacy and Safety of Recombinant Factor VIII （rFVIII）Treatment of Severe or Moderately Severe Hemophilia A
Efficacy, Safety and Pharmacokinetics Study of a rFVIII in Chinese subjects with Hemophilia A.To assess efficacy and safety of rFVIII administered as treatment and as on-demand therapy in adult and adolescent (12-65 years)…
- Trial name
- NCT02930317
- Testing
- Recombinant Factor VIII (50 IU/kg), Recombinant Factor VIII (On-demand treatment)
- Where
- 4 sites in China
- Volunteers
- 60 people
- Sponsor
- Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
Haemophilia A
Efficacy and Safety Evaluation Study of SCT800 in Previously Untreated Hemophilia A Patients.
This is an Open Label, Uncontrolled Study to Evaluate the the Safety and Efficacy of SCT800 for Prophylaxis Treatment in Severe Previously Untreated Hemophilia Patients.
- Trial name
- NCT07437404
- Testing
- SCT800 prophylaxis and treatment for bleeding events
- Where
- China
- Volunteers
- 36 people
- Sponsor
- Sinocelltech Ltd.
Severe Hemophilia A
Pharmacokinetics of rFVIIIFc at Two Vial Strengths
The primary objective of the study is to characterize the pharmacokinetics (PK) of rFVIIIFc administered at vial strengths of 1000 and 3000 IU in subjects with severe hemophilia A.
- Trial name
- NCT02083965
- Testing
- rFVIIIFc
- Where
- 8 sites in United States, Australia and 1 more
- Volunteers
- 19 people
- Sponsor
- Bioverativ Therapeutics Inc.
Congenital Bleeding Disorder, Haemophilia A
A Multi-centre, Comparative, Double Blind, Randomised Cross-over Trial Investigating Single Dose Pharmacokinetics and Safety of Turoctocog Alfa Pegol From the Pivotal Process and Turoctocog Alfa Pegol From the Commercial Process in Patients With Severe Haemophilia A
Investigating single dose pharmacokinetics and safety of turoctocog alfa pegol from the pivotal process and turoctocog alfa pegol from the commercial process in patients with severe haemophilia A
- Trial name
- pathfinder™7 (NCT02920398)
- Testing
- turoctocog alfa pegol
- Where
- 14 sites in United States, Denmark and 4 more
- Volunteers
- 21 people
- Sponsor
- Novo Nordisk A/S
Severe Hemophilia A
Study of Efficacy and Safety of FRSW107 in Pediatric Patients With Severe Hemophilia A
This study was divided into four stages: screening period, main trial period, extension period and follow-up period. In the main trial, both groups received FRSW107 prophylactic therapy.
- Trial name
- NCT06136507
- Testing
- FRSW107
- Where
- 13 sites in China
- Volunteers
- 76 people
- Sponsor
- Hangzhou Gensciences Biopharmaceutical Co., Ltd.
Hemophilia A
A Post Approval Commitment Study to Gain More Information on How Safe and Effective KOVALTRY is in Chinese Children, Adolescents /Adults With Severe Hemophilia A
The goal of this study is to gather more information on safety and efficacy of Kovaltry for the prevention and treatment of bleeds in Chinese children, adolescents/adults with severe hemophilia A.
- Trial name
- NCT04565236
- Testing
- Recombinant Factor VIII (Kovaltry, BAY81-8973) Treatment Group 1, Recombinant Factor VIII (Kovaltry, BAY81-8973) Treatment Group 2, Recombinant Factor VIII (Kovaltry, BAY81-8973) Treatment Group 3
- Where
- 10 sites in China
- Volunteers
- 45 people
- Sponsor
- Bayer
Hemophilia A, Severe Hemophilia A
An Open-label Safety and Efficacy Study of Recombinant FVIII in Patients With Severe Hemophilia A
This multicenter, open-label, phase 3 extension study will investigate the safety and efficacy of rVIII-SingleChain for prophylaxis and on-demand treatment of bleeding episodes in at least 200 previously treated patients (PTPs)…
- Trial name
- NCT02172950
- Testing
- rVIII-SingleChain
- Where
- 64 sites in United States, Australia and 23 more
- Volunteers
- 246 people
- Sponsor
- CSL Behring
Hemophilia B
Study of Recombinant Factor IX Product, IB1001, in Previously Treated Subjects With Hemophilia B
To evaluate the safety (acute adverse effects associated with infusions, and inhibitor development), pharmacokinetics (PK), and efficacy with respect to breakthrough bleeding and control of hemorrhaging during prophylaxis of…
- Trial name
- NCT02048111
- Testing
- IB1001
- Where
- 2 sites in United States, United Kingdom
- Sponsor
- Cangene Corporation
Severe Hemophilia A With an Inhibitor
The FEIBA NovoSeven Comparative Study
FENOC is a prospective, open-label, randomized, cross-over, multi-center study to investigate and compare the hemostatic effect and cost-efficacy of two different by-passing agents in the treatment of joint hemorrhages in…
- Trial name
- NCT00166309
- Testing
- FEIBA and NovoSeven
- Where
- Sweden
- Volunteers
- 60 people
- Sponsor
- Skane University Hospital
Hemophilia A, Hemophilia B
A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B
The purpose of this clinical trial is to learn about the safety and effects of the study medicine (called marstacimab) for the potential treatment of hemophilia in pediatric patients.
- Trial name
- BASIS KIDS (NCT05611801)
- Testing
- marstacimab
- Where
- 63 sites in United States, Argentina and 21 more
- Volunteers
- 100 people
- Sponsor
- Pfizer