Clinical trials: Haemophilia
Hemophilia A With Inhibitor, Hemophilia B With Inhibitor
Study of Coagulation Factor VIIa Marzeptacog Alfa (Activated) in Subjects With Hemophilia A or B
The purpose of the trial is to evaluate the safety and efficacy of MarzAA for on-demand treatment and control of bleeding episodes in hemophilia A or B patients with inhibitors compared with their standard of care (SOC).
- Trial name
- NCT04489537
- Testing
- MarzAA
- Where
- 43 sites in United States, Armenia and 14 more
- Volunteers
- 18 people
- Sponsor
- Catalyst Biosciences
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
Congenital Bleeding Disorder, Haemophilia A
Project to Update the Study of Congenital Haemophilia in Spain
This study is conducted in Europe. The aim of the study is to update the knowledge about the prevalence and severity of congenital haemophilia (A and B) in Spain.
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia B
- Trial name
- PUCHS (NCT01959555)
- Testing
- No treatment given
- Where
- Spain
- Volunteers
- 1,157 people
- Sponsor
- Novo Nordisk A/S
Hemophilia B, Hemophilia A
A Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%)
To establish baseline prospective efficacy data of current FIX prophylaxis replacement therapy in the usual care setting of hemophilia B subjects, who are negative for nAb to AAV-Spark100, prior to the Phase 3 gene therapy study.
- Trial name
- NCT03587116
- Testing
- Standard of Care FIX Replacement therapy, Standard of Care FVIII Replacement therapy
- Where
- 75 sites in United States, Australia and 16 more
- Volunteers
- 212 people
- Sponsor
- Pfizer
Hemophilia B
Safety and Dose Finding Study of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B
A Phase 1/2, open-label, dose-finding safety study of single ascending doses of DTX101 in adult males with moderate/severe to severe hemophilia B.
- Trial name
- NCT02618915
- Testing
- DTX101
- Where
- 9 sites in United States, Bulgaria and 1 more
- Volunteers
- 6 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Blood Coagulation Disorders, Hemophilia A
Effekt-2 - Efficacy and Safety of Long-term Treatment With KOGENATE® FS in Latin America
The aim of this international prospective post-marketing surveillance study is to obtain data on treatment procedures, long-term safety and efficacy and patient acceptance of KOGENATE Bayer/FS in treatment of patients with…
- Trial name
- NCT00969319
- Testing
- Recombinant Factor VIII (Kogenate FS, BAY14-2222)
- Where
- 2 sites in Mexico, Venezuela
- Volunteers
- 105 people
- Sponsor
- Bayer
Congenital Bleeding Disorder, Haemophilia B
Safety and Efficacy of NNC-0156-0000-0009 in Haemophilia B Patients
This trial is conducted in Africa, Asia, Europe, Japan and North America. The aim of this trial is to evaluate the safety and efficacy, including pharmacokinetics (the rate at which the body eliminates the trial drug), of…
- Trial name
- paradigm™ 2 (NCT01333111)
- Testing
- nonacog beta pegol
- Where
- 43 sites in United States, Canada and 13 more
- Volunteers
- 74 people
- Sponsor
- Novo Nordisk A/S
Hemophilia B
Study Evaluating rFIX; BeneFIX® in Hemophilia B
The primary objective of this clinical research study is to assess the safety and efficacy of rFIX for a minimum of 6 months in previously treated patients (PTPs) with hemophilia B (FIX:C ≤2%) during standard-of-care treatment…
- Trial name
- NCT00093171
- Testing
- rFIX
- Where
- 7 sites in United States
- Volunteers
- 23 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Severe Hemophilia A
rFVIII-Fc (Produced by AryoGen Pharmed Co.) Pharmacokinetic Study
The study is designed as a randomized, two-armed, double-blind, single-dose, crossover, two-sequence, active-controlled, multi-center, bioequivalence clinical trial with a primary endpoint of dose-normalized area under the curve…
- Trial name
- NCT06137092
- Testing
- Factor VIII, recombinant human with Fc fusion (rFVIII-Fc)
- Where
- 5 sites in Iran
- Volunteers
- 50 people
- Sponsor
- AryoGen Pharmed Co.
Haemophilia A
Turoctocog Alfa in Haemophilic Italian Patients: Protection and Engagement in Recreational Activities
This is a study on Italian patients with haemophilia A in prophylaxis treatment with Turoctocog alfa under routine clinical conditions.
- Trial name
- THIRd (NCT04584892)
- Testing
- Turoctocog alfa
- Where
- 10 sites in Italy
- Volunteers
- 18 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A
BAY79-4980 Compared to rFVIII-FS in Previously Treated Patients With Severe Hemophilia A
A study to assess treatment with a new formulation of recombinant factor VIII reconstituted with liposomes (BAY79-4980) to evaluate whether a once-a-week treatment is safe and can prevent bleeds in subjects with severe…
- Trial name
- NCT00623727
- Testing
- rFVIII-FS/pegylated liposomes (BAY79-4980), rFVIII-FS/WFI (BAY14-2222)
- Where
- 95 sites in United States, Argentina and 24 more
- Volunteers
- 143 people
- Sponsor
- Bayer
Hemophilia B
Post-Marketing Surveillance (Usage Results Study) of RIXUBIS in Adult and Pediatric Patients With Haemophilia B in South Korea
Primary: To characterize the safety of RIXUBIS when used under normal clinical care in South Korea. Secondary: To describe hemostatic effectiveness in subjects receiving RIXUBIS under normal clinical care in South Korea.
- Trial name
- NCT02922231
- Testing
- RIXUBIS
- Where
- 5 sites in South Korea
- Volunteers
- 57 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A
Long-term Study Evaluating Joint Health in People With Haemophilia A Receiving Real-world Prophylactic Treatment With Efanesoctocog Alfa
The rationale for this study is to further understand and describe the long-term prophylactic effectiveness of efanesoctocog alfa in preventing joint bleeds in a real-life setting.
- Trial name
- NCT06940830
- Testing
- Efanesoctocog alfa
- Where
- 31 sites in Croatia, Czechia and 6 more
- Volunteers
- 252 people
- Sponsor
- Swedish Orphan Biovitrum
Hemophilia B
Study Evaluating Allergic Reactions To Benefix In Hemophilia B Patients
Based on the finding that anaphylactic reactions to Benefix ("FIX") are associated with a specific IgE, a Basophil histamine release assay was selected to evaluate and demonstrate subject sensitization to FIX.
- Trial name
- NCT00244114
- Testing
- blood draw
- Volunteers
- 20 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Congenital Bleeding Disorder, Haemophilia A
Safety and Efficacy of Turoctocog Alfa in Previously Treated Male Children With Haemophilia A
This trial is conducted in Asia, Europe, and North and South America. The aim of this clinical trial is to investigate the safety and efficacy of turoctocog alfa (recombinant factor VIII, rFVIII (N8)) in male previously treated…
- Trial name
- guardian™ 3 (NCT01138501)
- Testing
- turoctocog alfa
- Where
- 38 sites in United States, Brazil and 11 more
- Volunteers
- 65 people
- Sponsor
- Novo Nordisk A/S
Haemophilia A
Adverse Event Data Collection From the EUHASS Registry on Turoctocog Alfa Pegol
This non-interventional study concerns a safety data collection based on adverse event data from a third-party registry (European Haemophilia Safety Surveillance System, EUHASS) that includes information about adverse events from…
- Trial name
- NCT04682145
- Testing
- Turoctocog alfa pegol
- Where
- Denmark
- Volunteers
- 50 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A
Efficacy and Safety Study of a Recombinant Protein-Free Manufactured Factor VIII (rAHF-PFM) in Previously Untreated Hemophilia A Patients
The purpose of this study is to evaluate whether Antihemophilic factor, recombinant, manufactured protein-free (rAHF-PFM) is effective and safe in the treatment of hemophilia A patients who have not been treated with factor VIII…
- Trial name
- NCT00157157
- Testing
- Recombinant Antihemophilic Factor Manufactured and Formulated without Added Human or Animal Proteins (rAHF-PFM)
- Where
- 35 sites in United States, Austria and 8 more
- Volunteers
- 66 people
- Sponsor
- Baxalta now part of Shire
Acute Heart Failure
Study of Safety, Tolerability and Pharmacokinetics of Serelaxin in Japanese Acute Heart Failure (AHF) Patients
This is a multicenter, randomized, double-blind, placebo-controlled study to assess safety, tolerability and pharmacokinetics and to explore efficacy of IV infusion of 10 µg/kg/day and 30 µg/kg/day serelaxin for 48 hours compared…
- Trial name
- NCT02002702
- Testing
- Serelaxin
- Where
- 15 sites in Japan
- Volunteers
- 46 people
- Sponsor
- Novartis Pharmaceuticals
Hemophilia A With Inhibitor, Hemophilia B With Inhibitor
Efficacy and Safety of KN057 Prophylaxis in Patients With Haemophilia A or B With Inhibitors
The purpose of this study is to show that KN057 can prevent bleeds in patients with haemophilia A or B with inhibitors and is safe to use.
- Trial name
- NCT06312475
- Testing
- KN057
- Where
- China
- Volunteers
- 53 people
- Sponsor
- Suzhou Alphamab Co., Ltd.
Hemophilia B
Post Marketing Observational Study of Reformulated BeneFIX
The primary objective of this observational study is to collect safety data on reformulated BeneFIX as prescribed in routine clinical practice conditions in France.
- Trial name
- NCT00835068
- Testing
- No intervention
- Where
- 17 sites in France
- Volunteers
- 58 people
- Sponsor
- Pfizer