Clinical trials: Disorders, Blood Coagulation
Albinism, Inborn Errors of Metabolism
Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome
Hermansky-Pudlak Syndrome (HPS) is an inherited disease that results in decreased pigmentation (oculocutaneous albinism), bleeding problems due to a platelet abnormality (platelet storage pool defect), and storage of an abnormal…
- Conditions
- Albinism, Inborn Errors of Metabolism, Oculocutaneous Albinism, Platelet Storage Pool Deficiency
- Trial name
- NCT00001596
- Testing
- Pirfenidone
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- William Gahl, M.D.
Idiopathic Thrombocytopenic Purpura, Purpura, Thrombocytopenic, Idiopathic
Clinical Evaluation of Eltrombopag in Chronic Idiopathic Thrombocytopenic Purpura (ITP)
An open-label, dose-adjustment extension study to evaluate the safety and efficacy of eltrombopag for treatment of subjects with ITP who have previously been enrolled in the eltrombopag trial TRA108109 (NCT00540423).
- Trial name
- NCT00828750
- Testing
- Eltrombopag oral tablets
- Where
- 6 sites in Japan
- Volunteers
- 19 people
- Sponsor
- GlaxoSmithKline
Idiopathic Thrombocytopenic Purpura, Thrombocytopenia
An Open Label Study of Romiplostim in Adult Thrombocytopenic Subjects With ITP
This protocol will provide open label romiplostim to adult thrombocytopenic subjects. Romiplostim will be administered by subcutaneous injection once per week.
- Conditions
- Idiopathic Thrombocytopenic Purpura, Thrombocytopenia, Thrombocytopenic Purpura
- Trial name
- NCT00508820
- Testing
- Romiplostim
- Volunteers
- 407 people
- Sponsor
- Amgen
Severe Hemophilia B
Long-Term Safety and Efficacy of rFIXFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia B
The primary objective of the study is to evaluate the long-term safety of rFIXFc in participants with hemophilia B. The secondary objective of this study is to evaluate the efficacy of rFIXFc in the prevention and treatment of…
- Trial name
- B-YOND (NCT01425723)
- Testing
- rFIXFc
- Where
- 49 sites in United States, Australia and 16 more
- Volunteers
- 120 people
- Sponsor
- Bioverativ Therapeutics Inc.
Severe Hemophilia A
Hemophilia Adult Prophylaxis Study: Factor VIII in Severe Hemophilia A
The purpose of this pilot R34 trial is to determine the feasibility of a large single dose Phase III study of hemophilia adult prophylaxis comparing once weekly with thrice-weekly recombinant factor VIII.
- Trial name
- NCT01405742
- Testing
- rF.VIII
- Where
- 6 sites in United States
- Volunteers
- 4 people
- Sponsor
- University of Pittsburgh
Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis
Long-term Safety and Efficacy of Momelotinib in Subjects With Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, Post-essential Thrombocythemia Myelofibrosis, Polycythemia Vera or Essential Thrombocythemia
This open-label study is to determine the long-term safety and tolerability of momelotinib in previously enrolled study participants with primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (post-PV MF)…
- Conditions
- Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, Post-Essential Thrombocythemia Myelofibrosis, Polycythemia Vera
- Trial name
- NCT02124746
- Testing
- Momelotinib
- Where
- 22 sites in United States, Australia and 3 more
- Volunteers
- 87 people
- Sponsor
- Sierra Oncology LLC - a GSK company
Purpura, Thrombocytopenic, Idiopathic
Daily Prednisone Versus Pulsed Dexamethasone in Treatment-naïve Adult Patients With Immune Thrombocytopenia
Patients above age 18 with a first episode of immune thrombocytopenia are randomized 1:1 between 2-4 weeks of daily prednisone (1 mg/kg/d) with subsequent dose tapering (arm A) and six 3-week cycles of pulsed dexamethasone (0.6…
- Trial name
- EIS2002 (NCT02334813)
- Testing
- Prednisone, Dexamethasone
- Volunteers
- 26 people
- Sponsor
- University Hospital, Essen
Hemophilia A, Hemophilia B
An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B
Primary Objective: To evaluate the long-term safety and tolerability of fitusiran in male patients with moderate or severe hemophilia A or B Secondary Objectives: To investigate the long-term efficacy of fitusiran To characterize…
- Trial name
- NCT02554773
- Testing
- Fitusiran (SAR439774)
- Where
- 12 sites in United States, Bulgaria and 3 more
- Volunteers
- 34 people
- Sponsor
- Genzyme, a Sanofi Company
Severe Hemophilia A
Human Cell Line-derived Recombinant Factor VIII (Human-cl-rhFVIII) in Previously Untreated Patients
Investigate the inhibitor development rate of Human cl rhFVIII in previously untreated patients with severe Hemophilia A.
- Trial name
- NCT01712438
- Testing
- Human cl rhFVIII
- Where
- 37 sites in United States, Belarus and 15 more
- Volunteers
- 110 people
- Sponsor
- Octapharma
Thrombocytopenic Purpura
Amgen Megakaryopoiesis Protein 2 (AMG 531) in Thrombocytopenic Subjects With Immune Thrombocytopenic Purpura (ITP)
The purpose of this study is to assess the safety and tolerability of AMG 531 (romiplostim), a novel thrombopoiesis-stimulating peptibody, and its effect on platelet counts in adults with immune thrombocytopenic purpura.
- Trial name
- NCT00117143
- Testing
- Romiplostim
- Volunteers
- 16 people
- Sponsor
- Amgen
End Stage Renal Disease
A Study to Investigate the Effect of Triferic Plus Heparin Infusion Compared to Heparin Alone on Coagulation Parameters in Hemodialysis Patients
This study will investigate the effect of co-administration of Triferic and heparin on the ability to maintain circuit anti-coagulation and iron delivery when compared to control conditions when each treatment is administered via…
- Trial name
- NCT04042324
- Testing
- Triferic, Heparin
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Rockwell Medical Technologies, Inc.
Von Willebrand Disease
Efficacy, PK, Immunogenicity and Safety of Wilate in Severe Von Willebrand Disease (VWD) Patients <6 Years of Age
The WIL-33 study aimed to determine the efficacy, pharmacokinetics, immunogenicity and safety of wilate as routine prophylaxis in up to 12 paediatric patients (eight evaluable) with severe von Willebrand Disease VWD (defined as…
- Trial name
- NCT04953884
- Testing
- Wilate
- Where
- 9 sites in United States, Czechia and 5 more
- Volunteers
- 12 people
- Sponsor
- Octapharma
Hemophilia A
A Clinical Trial to Evaluate Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants Without Inhibitors
This is a randomized, global, multicenter, open-label, Phase 3 clinical study in participants with severe hemophilia A without inhibitors against Factor VIII (FVIII) who are 12 years or older.
- Trial name
- HAVEN 3 (NCT02847637)
- Testing
- Emicizumab, Factor VIII (FVIII)
- Where
- 39 sites in United States, Australia and 12 more
- Volunteers
- 152 people
- Sponsor
- Hoffmann-La Roche
Severe Haemophilia A
Gene Therapy Study in Severe Haemophilia A Patients (270-201)
This study is being conducted by BioMarin Pharmaceutical Inc. as an open label, dose escalation study in order to determine the safety and efficacy of valoctocogene roxaparvovec (an Adenovirus-Associated Virus based gene therapy…
- Trial name
- NCT02576795
- Testing
- valoctocogene roxaparvovec
- Where
- 5 sites in United Kingdom
- Volunteers
- 15 people
- Sponsor
- BioMarin Pharmaceutical
Anticoagulant Therapy
In Vitro Diagnostic Test for DOAC in Urine
This trial is conducted to assess the performance and handling of the in vitro diagnostic (IVD) device for oral direct factor Xa and thrombin inhibitors from urine samples of patients on treatment with direct oral anticoagulants…
- Trial name
- PADOASU (NCT03182829)
- Testing
- DOAC Dipstick
- Where
- 18 sites in Germany
- Volunteers
- 880 people
- Sponsor
- Doasense GmbH
Primary Immunodeficiency, Common Variable Hypogammaglobulinemia
Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases.
The main objective of this study is to see if GAMMAPLEX is efficacious with respect to Food and Drug Administration (FDA) minimal requirements (no more than 1 serious, acute, bacterial infection per subject per year) in subjects…
- Conditions
- Primary Immunodeficiency, Common Variable Hypogammaglobulinemia, X-linked Hypogammaglobulinemia, Hypogammaglobulinemia
- Trial name
- NCT00278954
- Testing
- Gammaplex (Intravenous immunoglobulin)
- Where
- 7 sites in United States
- Volunteers
- 50 people
- Sponsor
- Bio Products Laboratory
Idiopathic Thrombocytopenic Purpura
Phase 2, Parallel Group, Rollover Study of AKR-501 in Patients With ChronicITP Who Completed 28 Days of Study Treatment in Protocol 501-CL-003
The purpose of this study is to determine the safety and efficacy of AKR-501 (avatrombopag) administered in participants with chronic Idiopathic Thrombocytopenic Purpura (ITP) who were enrolled into and completed 28 days of study…
- Trial name
- NCT00625443
- Testing
- Open Label (Avatrombopag tablets), Blinded (Avatrombopoag tablets)
- Where
- 12 sites in United States
- Volunteers
- 53 people
- Sponsor
- Eisai Inc.
Purpura, Thrombocytopenic, Idiopathic
Multiple Rising Does Study (Subcutaneous Doses) of BI 655064 in Male and Female Patients With Chronic Primary Immune Thrombocytopenic Purpura (ITP).
BI 655064 will be administered subcutaneously once weekly in patients with immune thrombocytopenic purpura (ITP) for up to 12 weeks.
- Trial name
- NCT02009761
- Testing
- BI 655064
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Boehringer Ingelheim
Factor XIII Deficiency
A Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
Congenital deficiency of factor XIII (FXIII) is an extremely rare inherited disorder associated with potentially life-threatening bleeding. Factor XIII Concentrate is given to patients whose blood is lacking factor XIII.
- Trial name
- NCT00885742
- Testing
- FXIII Concentrate (Human)
- Where
- 23 sites in United States, Spain
- Volunteers
- 41 people
- Sponsor
- CSL Behring
Hemophilia B
Study of Recombinant Factor IX Product, IB1001, in Subjects With Hemophilia B
Primary Objective: To evaluate the safety (acute effects associated with infusions, and inhibitor development), pharmacokinetics (PK), and efficacy with respect to breakthrough bleeding during prophylaxis and with respect to…
- Trial name
- NCT00768287
- Testing
- IB1001, nonacog alfa
- Where
- 23 sites in United States, France and 5 more
- Volunteers
- 77 people
- Sponsor
- Medexus Pharma, Inc.