Clinical trials: Bleeding Diathesis
Hemophilia A, Hemophilia B
A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors
The purpose of this study was to determine the frequency of bleeding episodes in participants receiving fitusiran as prophylactic treatment of hemophilia compared to participants who were assigned to continue with their regular…
- Trial name
- ATLAS-INH (NCT03417102)
- Testing
- fitusiran, Bypassing agents
- Where
- 58 sites in United States, Australia and 15 more
- Volunteers
- 60 people
- Sponsor
- Genzyme, a Sanofi Company
Primary Immune Deficiency Disorders, Common Variable Immunodeficiency
A Study to Find Out How Safe and Effective Gammaplex® is in Young People With Primary Immunodeficiency
The main objective is to determine the efficacy of Gammaplex by measuring the number of serious acute bacterial infections during treatment with Gammaplex over a 12 month period.
- Conditions
- Primary Immune Deficiency Disorders, Common Variable Immunodeficiency, X-linked Agammaglobulinemia, Hyper-IgM Syndrome
- Trial name
- NCT01289847
- Testing
- Gammaplex
- Where
- 10 sites in United States, Chile and 1 more
- Volunteers
- 25 people
- Sponsor
- Bio Products Laboratory
Immune Thrombocytopenic Purpura
A Efficacy and Safety Study of R935788 in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura (ITP)
The purpose of this study is to determine whether fostamatinib is safe and effective in the treatment of persistent/chronic Immune Thrombocytopenic Purpura (ITP).
- Trial name
- FIT (NCT02076399)
- Testing
- Fostamatinib disodium
- Where
- 52 sites in United States, Australia and 6 more
- Volunteers
- 76 people
- Sponsor
- Rigel Pharmaceuticals
Immune Thrombocytopenia, Platelet Disorder
Safety and Efficacy of Eltrombopag at Escalated Doses
Study rationale is based on the data that in previous clinical studies of eltrombopag in ITP there are some patients who have been reported as non responders at the maximal approved dose of 75 mg daily.
- Trial name
- NCT01880047
- Testing
- Eltrombopag
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- Weill Medical College of Cornell University
Disseminated Intravascular Coagulation
Recombinant Human Antithrombin (ATryn®) in the Treatment of Patients With DIC Associated With Severe Sepsis
The primary objective of the study is to explore the efficacy and safety of ATryn® (antithrombin alfa) for the treatment of disseminated intravascular coagulation (DIC) associated with severe sepsis, when administered by…
- Trial name
- NCT00506519
- Testing
- Antithrombin alfa (INN name), Control (Standard treatment)
- Volunteers
- 25 people
- Sponsor
- LEO Pharma
Severe Hemophilia B
Study of Recombinant Factor IX Fc Fusion Protein (rFIXFc) in Participants With Hemophilia B
The primary objectives of the study were: to evaluate the safety and tolerability of rFIXFc; to evaluate the efficacy of rFIXFc in all treatment arms; to evaluate the effectiveness of prophylaxis over on-demand (episodic) therapy…
- Trial name
- NCT01027364
- Testing
- Factor IX (rFIXFc), rFIX
- Where
- 51 sites in United States, Australia and 15 more
- Volunteers
- 123 people
- Sponsor
- Bioverativ Therapeutics Inc.
Primary Myelofibrosis, Thrombocytosis
A Phase Ib/II Dose-finding Study to Assess the Safety and Efficacy of LDE225 + INC424 in Patients With MF
The purpose of this phase Ib/II clinical trial was to: a) evaluate the safety of the co-administration of LDE225 and INC424 in myelofibrosis patients and establish a maximum tolerated dose and/or Recommended Phase II dose of the…
- Conditions
- Primary Myelofibrosis, Thrombocytosis, Essential Thrombocythemia, Polycythemia Vera
- Trial name
- NCT01787552
- Testing
- LDE225, INC424
- Where
- 18 sites in Australia, Belgium and 9 more
- Volunteers
- 50 people
- Sponsor
- Novartis Pharmaceuticals
Idiopathic Thrombocytopenic Purpura, Thrombocytopenia
Retrospective & Prospective Observational Study of Patients With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
This is a multi-center prospective observational descriptive study complemented by a retrospective chart review. Patients diagnosed with ITP and currently treated for ITP by a hematologist or hematologist-oncologist will be…
- Conditions
- Idiopathic Thrombocytopenic Purpura, Thrombocytopenia, Thrombocytopenia in Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP), Thrombocytopenic Purpura
- Trial name
- NCT00454857
- Testing
- Retrospective Chart Review, Patient-reported Outcome Questionnaires, Physician Survey
- Volunteers
- 326 people
- Sponsor
- Amgen
Hemophilia B
A Safety and Efficacy Study of a Recombinant Factor IX in Patients With Severe Hemophilia B
This study will examine the safety and efficacy of a Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) for the control and prevention of bleeding episodes in subjects who have previously received factor…
- Trial name
- NCT01361126
- Testing
- Recombinant Coagulation Factor IX Albumin Fusion Protein
- Where
- 2 sites in Bulgaria, Israel
- Volunteers
- 17 people
- Sponsor
- CSL Behring
Congenital Afibrinogenemia
Pharmacokinetics, Efficacy, and Safety of Human Plasma-Derived Fibrinogen (FIB Grifols) in Participants With Congenital Afibrinogenemia
The main objective of this study was to evaluate the pharmacokinetics (PK), efficacy, and safety of human plasma-derived fibrinogen concentrate FIB Grifols after a single-dose 70 milligrams/kilogram (mg/kg) body weight…
- Trial name
- IG0902 (NCT02281500)
- Testing
- Human Plasma-Derived Fibrinogen Concentrate
- Where
- 6 sites in United States, India and 2 more
- Volunteers
- 24 people
- Sponsor
- Grifols Therapeutics LLC
Thrombotic Thrombocytopenic Purpura (TTP)
A Study of BAX 930 in Children, Teenagers, and Adults Born With Thrombotic Thrombocytopenic Purpura (TTP)
Thrombotic thrombocytopenic purpura (or TTP for short) is a condition where blood clots form in small blood vessels throughout the body.
- Trial name
- NCT03393975
- Testing
- TAK-755, Standard of care
- Where
- 33 sites in United States, Austria and 7 more
- Volunteers
- 52 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A
Early Prophylaxis Immunologic Challenge (EPIC) Study
The purpose of the study was to assess if a once-weekly prophylactic regimen of 25 IU/kg ADVATE started at or before 1 year of age and before the onset of a severe bleeding phenotype (ie, joint bleeding), together with the…
- Trial name
- EPIC (NCT01376700)
- Testing
- Recombinant antihemophilic factor, plasma/albumin-free method (rAHF-PFM)
- Where
- 19 sites in United States, Austria and 10 more
- Volunteers
- 22 people
- Sponsor
- Baxalta now part of Shire
Accelerated Phase Chronic Myelogenous Leukemia, Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative
3-AP and Fludarabine in Treating Patients With Myeloproliferative Disorders, Chronic Myelomonocytic Leukemia, or Accelerated Phase or Blastic Phase Chronic Myelogenous Leukemia
This phase II trial is studying how well giving 3-AP together with fludarabine works in treating patients with myeloproliferative disorders (MPD), chronic myelomonocytic leukemia (CMML), or accelerated phase or blastic phase…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative, Blastic Phase Chronic Myelogenous Leukemia, Chronic Eosinophilic Leukemia
- Trial name
- NCT00381550
- Testing
- fludarabine phosphate, triapine, laboratory biomarker analysis
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- National Cancer Institute (NCI)
Myeloproliferative Diseases
Phase IIA Study of the HDAC Inhibitor ITF2357 in Patients With JAK-2 V617F Positive Chronic Myeloproliferative Diseases
Primary Objective: To evaluate efficacy and safety of ITF2357 in the treatment of patients with JAK2V617F positive myeloproliferative diseases [Polycythemia Vera (PV), Essential Thrombocytosis (ET), Myelofibrosis (MF)].
- Trial name
- NCT00606307
- Testing
- ITF2357
- Where
- 2 sites in Italy
- Volunteers
- 29 people
- Sponsor
- Italfarmaco
Atherosclerosis
Arterial Function and Atherosclerosis in Essential Thrombocythemia
The aim of the study is to examine (a) whether patients with JAK2 V617F positive ET in comparison to age-and sex-matched, apparently healthy control subjects show more advanced progression of arterial stiffness, pulse-wave…
- Trial name
- NCT03828422
- Testing
- imaging
- Volunteers
- 82 people, healthy volunteers welcome
- Sponsor
- University Medical Centre Ljubljana
Haemophilia A
BAY81-8973 Pediatric Safety and Efficacy Trial
The primary objective was to evaluate the safety and efficacy of the treatment with BAY81-8973 for prophylaxis and treatment of breakthrough bleeds in children with severe hemophilia A.
- Trial name
- NCT01311648
- Testing
- Recombinant Factor VIII (Kovaltry, BAY81-8973)
- Where
- 43 sites in United States, Argentina and 15 more
- Volunteers
- 94 people
- Sponsor
- Bayer
Hemophilia A
Rituximab to Treat Severe Hemophilia A
Hemophilia A is a serious blood clotting disorder caused by a lack of factor VIII, a specialized protein needed for normal blood clotting to occur.
- Trial name
- RICH (NCT00331006)
- Testing
- Rituximab
- Where
- 13 sites in United States
- Volunteers
- 23 people
- Sponsor
- Carelon Research
Cancer
Fentanyl Sublingual Spray in Treating Patients With Breakthrough Cancer Pain
This is a phase III, randomized, double-blind, placebo-controlled, multicenter study of the clinical response to fentanyl sublingual spray as a treatment for breakthrough cancer pain.
- Trial name
- NCT00538850
- Testing
- Fentanyl sublingual spray
- Where
- United States
- Volunteers
- 130 people
- Sponsor
- INSYS Therapeutics Inc
Hemophilia A
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg
This Phase III clinical study will assess the efficacy of BMN 270 defined as FVIII activity, during weeks 49-52 following intravenous infusion of BMN 270 and assess the impact of BMN 270 on usage of exogenous FVIII replacement…
- Trial name
- GENEr8-2 (NCT03392974)
- Testing
- Valoctocogene Roxaparvovec
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- BioMarin Pharmaceutical
Immune Thrombocytopenic Purpura
A Safety Study of Intravenous Immunoglobulin in Patients With Chronic Immune Thrombocytopenic Purpura (ITP)
It is known that intravenous immunoglobulins can induce hemolysis, but the mechanism is not known in detail. The primary objective of this study was to investigate the specificity of antigens on red blood cells in patients with…
- Trial name
- NCT01390649
- Testing
- IgPro10
- Where
- 17 sites in Bulgaria, Romania and 1 more
- Volunteers
- 57 people
- Sponsor
- CSL Behring