Clinical trials: Bleeding Diathesis
Hemophilia B, Blood Coagulation Disorders, Inherited
AAV Gene Therapy Screening/Observational Protocol (ECLIPSE)
Freeline is developing adeno-associated virus (AAV) vector based gene therapies for a number of diseases and is actively advancing a programme in Haemophilia B (HB).
- Conditions
- Hemophilia B, Blood Coagulation Disorders, Inherited, Blood Coagulation Disorder, Haematologic Disease
- Trial name
- ECLIPSE (NCT04272554)
- Where
- 56 sites in United States, Australia and 10 more
- Volunteers
- 37 people
- Sponsor
- Spur Therapeutics
Immune Thrombocytopenic Purpura
Study to Evaluate Safety and Efficacy in Adult Subjects With ITP
The purpose of this study is to assess the safety and tolerability of BMS-986004 when administered in subjects with ITP.
- Trial name
- ITP (NCT02273960)
- Testing
- BMS-986004 75 mg IV, BMS-986004 225 mg IV, BMS-986004 675 mg IV
- Where
- 20 sites in United States, Australia and 6 more
- Volunteers
- 46 people
- Sponsor
- Bristol-Myers Squibb
MPN (Myeloproliferative Neoplasms)
Phase 2 Study of Ruxolitinib Versus Anagrelide in Subjects With Essential Thrombocythemia Who Are Resistant to or Intolerant of Hydroxyurea (RESET-272)
The purpose of this study is to evaluate the efficacy and safety of ruxolitinib versus anagrelide in subjects with essential thrombocythemia who are resistant to or intolerant of hydroxyurea.
- Trial name
- NCT03123588
- Testing
- Ruxolitinib, Anagrelide
- Where
- 26 sites in United States
- Volunteers
- 12 people
- Sponsor
- Incyte Corporation
Essential Thrombocythemia, Polycythemia Vera
Open Label Study to Evaluate the Activity of Imetelstat in Patients With Essential Thrombocythemia or Polycythemia Vera
This is a phase II open-label study of single agent imetelstat in patients with essential thrombocytopenia or with polycythemia vera who have failed or are intolerant to at least one prior therapy, or who refuse standard therapy.
- Trial name
- ET/PV (NCT01243073)
- Testing
- Imetelstat
- Where
- 12 sites in United States, Germany and 1 more
- Volunteers
- 20 people
- Sponsor
- Geron Corporation
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.
Idiopathic Thrombocytopenic Purpura
A Study Evaluating the Addition of MabThera (Rituximab) to Standard Treatment in Patients With Idiopathic Thrombocytopenic Purpura (ITP)
This study will compare the efficacy, safety, and pharmacokinetics of standard treatment versus standard treatment plus MabThera in patients with ITP.
- Trial name
- NCT00770562
- Testing
- rituximab, Dexamethasone
- Where
- 22 sites in Italy
- Volunteers
- 103 people
- Sponsor
- Hoffmann-La Roche
Von Willebrand Disease
Study of Biostate® in Children With Von Willebrand Disease
This is an open-label study to investigate the pharmacokinetics (PK), efficacy, and safety of a von Willebrand Factor/Factor VIII (VWF/FVIII), Biostate, in children with Von Willebrand disease (VWD) in whom treatment with a VWF…
- Trial name
- NCT01213446
- Testing
- Biostate
- Where
- 7 sites in Belarus, Georgia and 4 more
- Volunteers
- 17 people
- Sponsor
- CSL Behring
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.
Haemophilia A
Patient Preferences in Treatment of Haemophilia A With and Without Inhibitors: a Discrete Choice Experiment in Germany
This study aims to generate structured patient preference evidence on prophylactic treatment options for haemophilia A for adults with and without inhibitors to support health technology assessment and benefit evaluation…
- Trial name
- NCT07653139
- Testing
- No treatment given
- Where
- Germany
- Volunteers
- 150 people
- Sponsor
- Novo Nordisk A/S
Idiopathic Thrombocytopenic Purpura
Safety Study of GMA161 in Patients With Idiopathic Thrombocytopenic Purpura (ITP)
This study is designed to investigate the safety of a single infusion of GMA161 in patients with idiopathic thrombocytopenic purpura, as well as, the way the drug enters and leaves the body.
- Trial name
- NCT00244257
- Testing
- GMA161
- Where
- 6 sites in United States
- Volunteers
- 10 people
- Sponsor
- Genzyme, a Sanofi Company
Hemophilia A
Pharmacokinetic Study of ADVATE Reconstituted in 2 mL Sterile Water for Injection
The purpose of this study is to determine the pharmacokinetics and safety of Antihemophilic factor, recombinant, manufactured protein-free (rAHF-PFM) reconstituted in 2 mL sterile water for injection (SWFI) and compare with those…
- Trial name
- NCT00952822
- Testing
- Antihemophilic factor, recombinant, manufactured protein-free (rAHF-PFM). (Antihemophilic factor is also known as Factor VIII)
- Where
- 11 sites in United States
- Volunteers
- 52 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A or B With Inhibitors
A Single-dose, Dose-escalation Study of a Long-acting MOD-5014 in Healthy Adult Male
A Phase 1, randomized, single-blind, placebo-controlled, single dose, dose-escalation study to assess the safety, pharmacokinetic and pharmacodynamic profile of subcutaneous administration of a long-acting recombinant factor VIIa…
- Trial name
- NCT02919800
- Testing
- MOD-5014
- Where
- Israel
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- OPKO Health, Inc.
ITP - Immune Thrombocytopenia
Mesenchymal Stem Cells in the Treatment of Refractory Primary Immune Thrombocytopenia
This study is a clinical study initiated by single-arm, single-center, multiple administration researchers. The main purpose of this study is to evaluate the safety and efficacy of mesenchymal stem cells in refractory ITP…
- Trial name
- MSC、ITP (NCT06813157)
- Testing
- MSC
- Where
- China
- Sponsor
- Guangzhou Bio-gene Technology Co., Ltd
Haemophilia A
A Study Looking at How Different Doses of Study Medicine (Inno8) Works in the Body of Healthy Men
This study will test how different doses of study medicine (Inno8) work in the healthy men. The purpose of this study is to prove safety of Inno8 in healthy men, which will support further development of Inno8 in people with…
- Trial name
- VOYAGER1 (NCT06649630)
- Testing
- NNC0442-0344 A
- Where
- 2 sites in Germany
- Volunteers
- 95 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
Hemophilic Arthropathy
Rofecoxib Efficacy and Safety Evaluation Trial in Hemophilic Arthropathy
This is a multicenter, randomized, double-blind study to evaluate the efficacy and safety of TRM-201 (rofecoxib) versus Placebo in the treatment of patients with hemophilic arthropathy (HA) over a 12-week period (Part I) that is…
- Trial name
- RESET-HA (NCT04684511)
- Testing
- TRM-201 (Rofecoxib)
- Where
- 46 sites in United States, Australia and 6 more
- Volunteers
- 2 people
- Sponsor
- Tremeau Pharmceuticals, Inc.
Congenital Bleeding Disorder, Glanzmann's Disease
A Study to Evaluate Safety and Efficacy of NovoSeven® in Patients With Glanzmann's Thrombasthenia in Japan
This study is conducted in Asia. The aim of the study is to evaluate the safety and efficacy of NovoSeven® (activated recombinant factor VII, eptacog alfa (activated)) during bleeding episodes and for the prevention of bleeding…
- Trial name
- NCT01876745
- Testing
- eptacog alfa (activated)
- Where
- Japan
- Volunteers
- 4 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A
GAS-Hem Feasibility Study
The purpose of this study is to investigate the feasibility and acceptability of the Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem) as a patient reported outcome (PRO) measure to monitor clinical progress in…
- Trial name
- NCT02674997
- Testing
- Factor VIII
- Where
- 4 sites in United States, Canada
- Volunteers
- 44 people
- Sponsor
- Baxalta now part of Shire
Essential Thrombocythaemia
Anagrelide Retard vs. Placebo: Efficacy and Safety in "At-risk" Patients With Essential Thrombocythaemia
This is a multicenter, phase III, randomized, subject and sponsor-blinded, placebo-controlled study to determine the treatment effect of "Anagrelide retard" in subjects with Essential Thrombocythaemia (ET) at "defined risk"…
- Trial name
- ARETA (NCT01230775)
- Testing
- Anagrelide retard
- Where
- 37 sites in Austria, Bulgaria and 7 more
- Volunteers
- 146 people
- Sponsor
- AOP Orphan Pharmaceuticals AG
Hemophilia A or B
A Study to Describe Patient REal-world FitusiRan ExpeRience
This is a real-world, mixed-methods observational study conducted among adolescent (ages 12-17 years) and adult participants diagnosed with hemophilia A or B, with or without Factor VIII (FVIII) or Factor IX (FIX) inhibitors, who…
- Trial name
- PREFER (NCT07895381)
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Sanofi
Polycythemia Vera
A Two-part Study to Assess the Safety and Preliminary Efficacy of Givinostat in Patients With Polycythemia Vera
This is a two-part, multicenter, open label, non-randomized, phase Ib/II study to assess the safety and tolerability, Maximum Tolerated Dose and preliminary efficacy of Givinostat in patients with JAK2V617F positive Polycythemia…
- Trial name
- NCT01901432
- Testing
- Givinostat
- Where
- 25 sites in France, Germany and 3 more
- Volunteers
- 48 people
- Sponsor
- Italfarmaco