Clinical trials: Hemophilia a
Congenital Bleeding Disorder, Haemophilia A
Evaluation of Safety and Efficacy, Including Pharmacokinetics, of NNC 0129-0000-1003 When Administered for Treatment and Prophylaxis of Bleeding in Subjects With Haemophilia A
This trial is conducted globally. The aim of the trial is to evaluate the safety and efficacy, including pharmacokinetics (the exposure of the trial drug in the body) of NNC 0129-0000-1003 (N8-GP) in subjects with Haemophilia A.
- Trial name
- pathfinder™2 (NCT01480180)
- Testing
- turoctocog alfa pegol
- Where
- 93 sites in United States, Australia and 22 more
- Volunteers
- 186 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A
BAX 855 PK-guided Dosing
1. To compare the efficacy and safety of pharmacokinetic (PK)-guided treatment with BAX 855 targeting FVIII trough levels of 1-3% and approximately 10% (8-12%) 2.
- Trial name
- PROPEL (NCT02585960)
- Testing
- PEGylated Recombinant Factor VIII
- Where
- 83 sites in United States, Australia and 20 more
- Volunteers
- 135 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A
UPLC-MS/MS Monitoring of Emicizumab Therapy
Emicizumab is a monoclonal bispecific antibody with a terminal half-life of 28 days which is now licensed in the treatment of severe haemophilia A with or without inhibitors.
- Trial name
- EMICARE (NCT04472169)
- Where
- 2 sites in France
- Volunteers
- 100 people
- Sponsor
- University Hospital, Lille
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
Healthy
A Phase I Study of Monoclonal Antibody TB-402 in Healthy Male Volunteers
Dose escalation study to assess the safety and tolerability of TB-402, a monoclonal antibody directed against FVIII, versus placebo in healthy male volunteers.
- Trial name
- NCT00612196
- Testing
- TB-402
- Where
- Denmark
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- ThromboGenics
Hemophilia A
Immune Tolerance Induction in Haemophilia A Patients Using Wilate or Nuwiq
Uncontrolled, multi-centre, non-interventional study with a prospective and a retrospective cohort, to evaluate the efficacy of Wilate or Nuwiq in achieving complete or partial immune tolerance induction (ITI) success in severe…
- Trial name
- PREVAIL (NCT03344003)
- Testing
- Wilate or Nuwiq
- Where
- 3 sites in Canada
- Volunteers
- 14 people
- Sponsor
- Octapharma
Hemophilia A
A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A
This is a prospective, observational, multi-center longitudinal cohort study to describe the real-world effectiveness, safety and treatment usage of efanesoctocog alfa in patients with hemophilia A treated per standard of care in…
- Trial name
- NCT05911763
- Testing
- Efanesoctocog Alfa BIVV001
- Where
- 28 sites in United States
- Volunteers
- 200 people
- Sponsor
- Sanofi
Hemophilia A
Evaluation of the Safety and Efficacy of Hemophilia A Gene Therapy Drugs
This is a multi-center, Phase 1/2/3, single-arm, open-label, single-dose treatment clinical study to evaluate the safety, tolerability and efficacy of BBM-H803 injection in severe Hemophilia A subjects.
- Trial name
- NCT06111638
- Testing
- Single dose intravenous injection of BBM-H803
- Where
- 10 sites in China
- Volunteers
- 55 people
- Sponsor
- Shanghai Xinzhi BioMed Co., Ltd.
Hemophilia A
Study Evaluating The Safety Of Xyntha In Usual Care Settings
The purpose of this study is to collect safety information associated with the use of Xyntha in a usual care setting. Upon meeting eligibility criteria, patients will be required to have approximately 5 study visits over a 2 year…
- Trial name
- NCT00765726
- Testing
- Moroctocog alfa(AF-CC), Blood draw for laboratory testing
- Where
- 6 sites in United States, New Zealand
- Volunteers
- 12 people
- Sponsor
- Pfizer
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
Hemophilia A
PF-05208756, Moroctocog Alfa (AF-CC), Xyntha For Hemophilia A
An open-label, single-arm, post- authorization pragmatic clinical trial on the safety and efficacy of Xyntha (Moroctocog-alfa (AF-CC), Recombinant FVIII) in subjects with hemophilia A in usual care settings in China for…
- Trial name
- NCT02492984
- Testing
- Intravenous infusions of Xyntha
- Where
- 15 sites in China
- Volunteers
- 85 people
- Sponsor
- Pfizer
Hemophilia A
Combination Therapy of Low Doses of rFVIIa and FEIBA for Severe Hemophilia A Patients With an Inhibitor to Factor VIII
Patients with severe hemophilia and inhibitors can be treated effectively by Activated Prothrombin Complex Concentrates (APCC, eg. FEIBA) or High dose recombinant factor VIIa (rFVIIa).
- Trial name
- NCT00284193
- Testing
- rFVIIa-FEIBA therapy for hemophilia A inhibitors, FEIBA- Activated Prothrombin Complexes
- Volunteers
- 5 people
- Sponsor
- Sheba Medical Center
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
Study Evaluating ReFacto AF in Severe Hemophilia A
To characterize the safety and efficacy of ReFacto AF in treating acute bleeding episodes during prophylaxis treatment, including neoantigenicity.
- Trial name
- NCT00037544
- Testing
- ReFacto AF
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Hemophilia A
Covalent Tolerance Induction to Factor VIII-Prediction of Inhibitors in Hemophilia
To correlate the Human Leukocyte Antigen type and genetic defect with hemophilia A.
- Trial name
- NCT00178607
- Where
- United States
- Volunteers
- 99 people
- Sponsor
- The University of Texas Health Science Center, Houston
Hemophilia A
Cartilage Biomarkers and HEAD-US Score in Severe Hemophilia A Patients Receiving FVIII Prophylaxis
Patients with severe hemophilia A often develop joint complication requiring careful monitoring. This study aimed to compare the effectiveness of standard low dose weight-based FVIII concentrates (CFCs) prophylaxis with…
- Trial name
- HEADUS_SHA8 (NCT06816953)
- Testing
- HEAD-US, Urinary carboxy-terminal cross linked telopeptide of type II collagen, Inflammatory markers
- Where
- Thailand
- Volunteers
- 19 people
- Sponsor
- Chulalongkorn University
Haemophilia A Without Inhibitors, Haemophilia B Without Inhibitors
Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia Without Inhibitors
This study will test how well a new medicine called concizumab works in the body of people with haemophilia A or B without inhibitors. The purpose is to show that concizumab can prevent bleeds in the body and is safe to use.
- Trial name
- explorer8 (NCT04082429)
- Testing
- Concizumab
- Where
- 113 sites in United States, Algeria and 31 more
- Volunteers
- 156 people
- Sponsor
- Novo Nordisk A/S
Hemophilia, Factor XIII Deficiency
Clinical Trial of Factor XIII (FXIII) Concentrate
Congenital deficiency of Factor XIII is a rare but potentially life threatening disorder. It is inherited in an autosomal recessive fashion.
- Trial name
- NCT00640289
- Testing
- Fibrogammin P
- Where
- United States
- Volunteers
- 72 people
- Sponsor
- Children's Hospital of Orange County
Hemophilia A
Evaluate Efficacy and Safety of ADVATE in the Standard Prophylaxis Treatment of Severe or Moderately Severe Hemophilia A
Based on the successful pioneer work of Dr Nilsson and her colleagues in Sweden that started in the late 1950's, prophylaxis is recommend as the standard of care for boys with severe haemophilia by WHO and World Federation of…
- Trial name
- NCT02282410
- Testing
- ADVATE
- Volunteers
- 15 people
- Sponsor
- The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School
Hemophilia A
A Study of PEGylated Recombinant Factor VIII (BAX855) in Previously Untreated Young Children With Severe Hemophilia A
This study is for young children with severe hemophilia A who have previously not been treated with BAX855 or other FVIII concentrates. The main aim of the study is to check for side effects from treatment with BAX855.
- Trial name
- NCT02615691
- Testing
- PEGylated Recombinant Factor VIII, ITI
- Where
- 91 sites in United States, Austria and 21 more
- Volunteers
- 120 people
- Sponsor
- Baxalta now part of Shire