Clinical trials: Disorders, Blood Coagulation
Thrombotic Thrombocytopenic Purpura (TTP)
A Study to Learn More About the Treatment of People With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Who Received Recombinant ADAMTS13 (rADAMTS13) as Part of the Early Access Program
Congenital thrombotic thrombocytopenic purpura (cTTP) is a rare blood disorder that some people are born with. It is caused by inherited changes in the ADAMTS13 gene that reduce the body's ability to produce the ADAMTS13 enzyme.
- Trial name
- NCT07429942
- Testing
- No Intervention
- Where
- 8 sites in Germany, Poland
- Volunteers
- 94 people
- Sponsor
- Takeda
Healthy Volunteers
Safety and Tolerability of ZE74-0282 in Healthy Volunteers
This first-in-human, randomized, double-blind, placebo-controlled Phase 1 study evaluates the safety, tolerability, and pharmacokinetics of single, split, and multiple oral doses of ZE74-0282 under fasted or fed conditions in…
- Trial name
- NCT07527221
- Where
- Australia
- Volunteers
- 64 people, healthy volunteers welcome
- Sponsor
- Eilean Therapeutics AU Pty Ltd
Hemophilia A
A Study of Standard and Individualized Prophylactic Treatment With Advate in Men and Boys With Severe Hemophilia A
The main aim of this study is to learn about changes in the lowest blood levels of Factor VIII in men and boys when upgraded from standard prophylaxis with Advate to individualized prophylaxis with Advate.
- Trial name
- HAPKIDO (NCT04953000)
- Where
- 13 sites in Russia
- Volunteers
- 94 people
- Sponsor
- Takeda
Von Willebrand Disease
Study of Voncento® in Subjects With Von Willebrand Disease
This is a multi-centre, open-label, single-arm, phase 4, post-marketing study to further investigate the efficacy and safety of Voncento in subjects with Von Willebrand Disease (VWD) in whom treatment with a Von Willebrand Factor…
- Trial name
- NCT02552576
- Testing
- Voncento
- Where
- 12 sites in Austria, Germany and 3 more
- Volunteers
- 26 people
- Sponsor
- CSL Behring
Congenital Bleeding Disorder, Haemophilia A
To Investigate Safety and Efficacy of NovoEight® (rFVIII) During Long-term Treatment of Haemophilia A in Japan
This study is conducted in Asia. The aim of this study is to evaluate the safety and efficacy of NovoEight® (recombinant factor VIII) in patients with haemophilia A in Japan in the setting of routine clinical practice.
- Trial name
- NCT02207218
- Testing
- turoctocog alfa
- Where
- 2 sites in Japan
- Volunteers
- 40 people
- Sponsor
- Novo Nordisk A/S
Essential Thrombocythemia
French Observational Xagrid (FOX) Study In Adult Patients With Essential Thrombocythemia
This is an observational study to explore how different treatment regimens affect continuation with treatment in the first 6 months following initiation of XAGRID into adult patients' essential thrombocythemia therapy.
- Trial name
- FOX (NCT01192347)
- Where
- 46 sites in France
- Volunteers
- 177 people
- Sponsor
- Shire
Essential Thrombocythaemia
A Study to Evaluate the Efficacy and Safety of Ropeginterferon Alfa-2b in Essential Thrombocythaemia Patients
The primary objective of this study is to assess the efficacy of ropeginterferon alfa-2b in patients with ET who need cytoreductive treatment but are intolerant or refractory to, and/or ineligible for cytoreductive treatments…
- Trial name
- NCT06514807
- Testing
- Ropeginterferon alfa-2b (BESREMi®)
- Where
- 36 sites in Austria, Czechia and 8 more
- Volunteers
- 117 people
- Sponsor
- AOP Orphan Pharmaceuticals AG
Hemophilia A
Long-Term Safety and Efficacy of rFVIIIFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia A
The primary objective of the study is to evaluate the long-term safety of recombinant human Factor VIII Fc fusion protein (rFVIIIFc) in participants with hemophilia A.
- Trial name
- ASPIRE (NCT01454739)
- Testing
- rFVIIIFc
- Where
- 78 sites in United States, Australia and 20 more
- Volunteers
- 240 people
- Sponsor
- Bioverativ Therapeutics Inc.
Healthy
Study to Evaluate CAY001 in Healthy Volunteers
This is a Phase 1, first-in-human, randomized, double-blind, placebo-controlled, single ascending dose, sequential group study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of CAY001 when…
- Trial name
- NCT06845839
- Testing
- CAY001
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Cayuga Biotech, Inc.
Hemophilia A, Hemophilia B
A Phase 1 Study of an Investigational Drug, ALN-AT3SC, in Healthy Volunteers and Hemophilia A or B Patients
The purpose of this study is to determine the safety, tolerability, and pharmacokinetics of ALN-AT3SC in healthy volunteers and Hemophilia A or B patients.
- Trial name
- NCT02035605
- Testing
- ALN-AT3SC, Sterile Normal Saline (0.9% NaCl)
- Where
- 14 sites in United States, Bulgaria and 3 more
- Volunteers
- 51 people, healthy volunteers welcome
- Sponsor
- Sanofi
Immune Thrombocytopenia
Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)
This was a randomized, double-blind study of rilzabrutinib in patients with persistent or chronic ITP, with an average platelet count of <30,000/μL (and no single platelet count >35,000/μL) on two counts at least 5 days apart in…
- Trial name
- LUNA 3 (NCT04562766)
- Testing
- Rilzabrutinib
- Where
- 155 sites in United States, Argentina and 24 more
- Volunteers
- 232 people
- Sponsor
- Principia Biopharma, a Sanofi Company
Focal Segmental Glomerulosclerosis, Minimal Change Disease
Study of Sparsentan Treatment in Pediatrics With Proteinuric Glomerular Diseases
To evaluate the safety, efficacy and tolerability of sparsentan oral suspension and tablets, and assess changes in proteinuria after once-daily dosing over 108 weeks.
- Conditions
- Focal Segmental Glomerulosclerosis, Minimal Change Disease, Immunoglobulin A Nephropathy, IgA Vasculitis
- Trial name
- EPPIK (NCT05003986)
- Testing
- Sparsentan
- Where
- 47 sites in United States, Germany and 6 more
- Volunteers
- 67 people
- Sponsor
- Travere Therapeutics, Inc.
Hemophilia A
Prophylaxis Study of Recombinant Factor VIII Manufactured Protein-Free (rAHF-PFM) in Patients With Hemophilia A
The primary purpose of this randomized, two-arm parallel clinical study in 66 previously treated patients with severe or moderately severe hemophilia A is to compare the rate of bleeding episodes for standard prophylaxis (20-40…
- Trial name
- NCT00243386
- Testing
- Antihemophilic factor, recombinant, manufactured protein-free
- Where
- 29 sites in United States, Austria and 8 more
- Volunteers
- 82 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A
Patient-reported Outcomes in Subjects Treated With ReFacto AF Routine Prophylaxis
This NIS aims to assess the patient-reported outcomes (PROs) in enrolled subjects
- Trial name
- NCT02718677
- Testing
- Refacto AF
- Sponsor
- Pfizer
Primary Immune Thrombocytopenia
A Study to Investigate the Long-term Safety, Tolerability, and Efficacy of Rozanolixizumab in Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)
The purpose of this study is to assess the long-term safety, tolerability and clinical efficacy of treatment with rozanolixizumab.
- Trial name
- myOpportunITy3 (NCT04596995)
- Testing
- Rozanolixizumab
- Where
- 27 sites in United States, China and 12 more
- Volunteers
- 43 people
- Sponsor
- UCB Biopharma SRL
Idiopathic Thrombocytopenic Purpura
A Study in Healthy Subjects to Evaluate Bioavailability of 4 Formulations of E5501
This is a study in healthy subjects. There are two parts to the study. In the first part of the study each subject will receive a single 10mg dose of each of the four formulations of E550.
- Trial name
- NCT01549054
- Testing
- 10-mg dose of E5501 2G tablet, 10-mg dose of E5501 cyclodextrin oral solution, 10-mg dose of E5501-P21% powder
- Where
- United Kingdom
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- Eisai Inc.
Breast Neoplasms
Immunostimulating Interstitial Laser Thermotherapy in Breast Cancer
Thermotherapy is a technology aiming at destroying tissue, for example tumor tissue. Immunostimulating Interstitial Laser Thermotherapy (imILT) is a specific form of thermotherapy, which, in addition to destroying tumor tissue…
- Trial name
- NCT03039127
- Testing
- Immunostimulating Interstitial Laser Thermotherapy
- Where
- United Kingdom
- Volunteers
- 3 people
- Sponsor
- Clinical Laserthermia Systems AB
Haemophilia A, Haemophilia B
Post-Marketing Surveillance (All Case Surveillance) on Treatment With Alhemo® in Patients With Haemophilia A or Haemophilia B With Inhibitors
The purpose of the study is to investigate the safety and effectiveness of Alhemo® in all participants under real-world clinical practice in Japan. Participants will get Alhemo® as prescribed by the study doctor.
- Trial name
- NCT06285071
- Testing
- Concizumab
- Where
- 11 sites in Japan
- Volunteers
- 23 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A With Inhibitor, Hemophilia A
Real-life Clinical FEIBA Samples Measured Using the Version A of the HemA EnzySystem
The goal of this observational study is to assess if the version A of the HemA EnzySystem, a novel portable coagulation testing platform, can be used in patients with hemophilia A treated with Factor VIII Bypassing Agent (FEIBA).
- Trial name
- NCT06357572
- Testing
- Thrombin generation assay (EnzySystem HemA version A - whole blood), Thrombin generation assay (Ceveron s100 (Technoclone) - fresh plasma), Additional coagulation tests
- Where
- Poland
- Volunteers
- 6 people
- Sponsor
- Enzyre B.V.
Hemophilia A, Hemophilia B
A Study of a Long-Acting r-Factor 7a (Factor VIIa) in Adult Men With Hemophilia A or B
The purpose of the current Phase 1/2a single dose, dose-escalating study is to evaluate the acute safety, pharmacokinetics (PK) and pharmacodynamics (PD) properties of MOD-5014 in adult subjects with moderate/severe congenital…
- Trial name
- NCT02418793
- Testing
- MOD-5014
- Where
- 11 sites in United States
- Volunteers
- 27 people
- Sponsor
- OPKO Health, Inc.
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