Clinical trials: Coagulation Defect
Immune Deficiency Disorders, Severe Combined Immunodeficiency
Immune Disorder HSCT Protocol
This study hypothesizes that a reduced intensity immunosuppressive preparative regimen will establish engraftment of donor hematopoietic cells with acceptable early and delayed toxicity in patients with immune function disorders.
- Conditions
- Immune Deficiency Disorders, Severe Combined Immunodeficiency, Chronic Granulomatous Disease, X-linked Agammaglobulinemia
- Trial name
- NCT01821781
- Testing
- Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Washington University School of Medicine
Hemophilia A With Inhibitor, Hemophilia A
INdividualized ITI Based on Fviii(ATE) Protection by VWF
The primary goal of the INITIATE trial is to compare the clinical outcome of individualized lot selection to random lot selection utilizing one plasma-derived von Willebrand factor (VWF)/coagulation factor (FVIII) complex…
- Trial name
- INITIATE (NCT03204539)
- Testing
- Wilate
- Where
- 3 sites in United States
- Volunteers
- 1 people
- Sponsor
- University of California, Davis
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 A
Study Evaluating On-Demand Treatment Of Xyntha In Chinese Subjects
This study will evaluate the safety and efficacy of on-demand treatment with Xyntha in Chinese hemophilia A subjects.
- Trial name
- NCT00868530
- Testing
- Xyntha
- Where
- 6 sites in China
- Volunteers
- 53 people
- Sponsor
- Pfizer
Von Willebrand Disease
Efficacy of Alphanate FVIII/VWF Concentrate in Type 3 Von Willebrand Patients
To assess the efficacy of FVIII/VWF Complex (Human), Alphanate® as replacement therapy in preventing excessive bleeding in subjects with congenital Type 3 von Willebrand Disease (VWD) who undergo surgical procedures.
- Trial name
- NCT00555555
- Testing
- Alphanate SD/HT
- Volunteers
- 4 people
- Sponsor
- Grifols Biologicals, LLC
Hemophilia A
Study Evaluating The Efficacy And Safety Of Xyntha In Children Less Than 6 Years Of Age
This study will be investigating the safety and efficacy of Xyntha (moroctocog alfa (AF-CC)) in male patients less than 6 years old.
- Trial name
- NCT00759655
- Testing
- Moroctocog alfa
- Volunteers
- 1 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Hemophilia
LR769 in Congenital Hemophilia Patients With Inhibitors Undergoing Elective Surgery or Invasive Procedures
The purpose of this study, PerSept 3, is to evaluate LR769 for the prevention of excessive bleeding and achievement of hemostasis in congenital hemophilia A or B patients who have inhibitors to Factor VIII or Factor IX , are aged…
- Trial name
- PerSept3 (NCT02548143)
- Testing
- Coagulation Factor VIIa (Recombinant)
- Where
- 9 sites in United States, Mexico and 4 more
- Volunteers
- 12 people
- Sponsor
- Laboratoire français de Fractionnement et de Biotechnologies
Essential Thrombocythemia
Study of Bomedemstat in Participants With Essential Thrombocythemia (IMG-7289-CTP-201/MK-3543-003)
This is a Phase 2b open label study of an orally administered LSD1 inhibitor, Bomedemstat (MK-3543, formerly called IMG-7289), in patients with essential thrombocythemia.
- Trial name
- NCT04254978
- Testing
- Bomedemstat
- Where
- 26 sites in United States, Australia and 5 more
- Volunteers
- 73 people
- Sponsor
- Imago BioSciences, Inc., a subsidiary of Merck & Co., Inc., (Rahway, New Jersey USA)
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
Congenital Bleeding Disorder, Haemophilia B
Safety, Efficacy and Pharmacokinetics of NNC-0156-0000-0009 in Previously Treated Children With Haemophilia B.
This trial is conducted in Asia, Europe and North America. The aim of the trial is to evaluate safety, efficacy and pharmacokinetics (the exposure of the trial drug in the body) of NNC-0156-0000-0009 (nonacog beta pegol, N9-GP)…
- Trial name
- paradigm™5 (NCT01467427)
- Testing
- nonacog beta pegol
- Where
- 51 sites in United States, Brazil and 10 more
- Volunteers
- 25 people
- Sponsor
- Novo Nordisk A/S
Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission
A Two-Step Approach to Reduced Intensity Bone Marrow Transplant for Patients With Hematological Malignancies
This phase II trial studies how well reduced intensity donor stem cell transplant works in treating patients with hematologic malignancies.
- Conditions
- Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q)
- Trial name
- NCT01384513
- Testing
- Fludarabine, Busulfan, Total Body Irradiation (TBI)
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
Thrombocytopenia, Idiopathic Thrombocytopenic Purpura
Open Label Extension Study of Romiplostim (AMG 531) in Thrombocytopenic Patients With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
The purpose of this study is to determine the safety of romiplostim as a long-term treatment in thrombocytopenic subjects with ITP, to evaluate the long-term platelet response to romiplostim, and to evaluate changes in patient…
- Trial name
- NCT00116688
- Testing
- Romiplostim
- Volunteers
- 313 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.
Polycythemia Vera, Essential Thrombocythemia
Myeloproliferative Neoplasms: an In-depth Case-control Study
There is a paucity of data on the aetiology of myeloproliferative neoplasms (MPNs). The investigators conducted a systematic review of the literature which identified several cohort and case-control studies that have investigated…
- Conditions
- Polycythemia Vera, Essential Thrombocythemia, Primary Myelofibrosis
- Trial name
- MOSAICC (NCT01831635)
- Testing
- Methodological pens, Methodological compensation, Methodological trolley tokens
- Where
- 3 sites in United Kingdom
- Volunteers
- 234 people, healthy volunteers welcome
- Sponsor
- Queen's University, Belfast
Neutropenia, Sickle Cell Disease
Decitabine for High-Risk Sickle Cell Disease
Background: In sickle cell disease (SCD), the proteins in the red blood cells that carry oxygen do not behave normally. In parts of the body where there are low levels of oxygen or where oxygen is used more, the sickle hemoglobin…
- Trial name
- NCT01375608
- Testing
- Decitabine
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
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
Hemophilia A
Clinical Study to Investigate the Pharmacokinetics, Efficacy, Safety and Immunogenicity of a Recombinant FVIII in Patients With Severe Hemophilia A
This is a clinical study to investigate the pharmacokinetics, efficacy, safety and immunogenicity of human-cl rhFVIII, a newly developed human cell-line derived recombinant FVIII concentrate in previously treated patients with…
- Trial name
- NCT00989196
- Testing
- Human-cl rhFVIII, Kogenate FS
- Where
- 9 sites in United States, Bulgaria and 1 more
- Volunteers
- 22 people
- Sponsor
- Octapharma
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
Acute Myeloid Leukemia, Essential Thrombocythemia
Study of LY573636-sodium in Essential Thrombocythemia and Acute Myeloid Leukemia
The purpose of this study is to determine a safe dose of LY573636-sodium to be given to patients with acute myeloid leukemia and to determine any side effects that may be associated with LY573636-sodium in this patient…
- Trial name
- NCT00718159
- Testing
- LY573636-sodium
- Where
- 5 sites in United States
- Volunteers
- 23 people
- Sponsor
- Eli Lilly and Company
Severe Hemophilia A
Efficacy and Safety Study of Human-cl rhFVIII in PTPs With Severe Hemophilia A
This study will determine the efficacy of human-cl rhFVIII in previously treated patients with severe hemophilia A during prophylactic treatment, treatment of bleeding episodes and in surgical prophylaxis.
- Trial name
- NCT01125813
- Testing
- recombinant Factor VIII
- Where
- 11 sites in Austria, Bulgaria and 2 more
- Volunteers
- 32 people
- Sponsor
- Octapharma