Clinical trials: Blood Coagulation Disorder
Von Willebrand Diseases
Minimize Menorrhagia in Women With Von Willebrand Disease
This is an outpatient, 24-week Phase III prospective, randomized, crossover trial comparing recombinant von Willebrand factor (rVWF) and tranexamic acid (TA, Lysteda®) to minimize menorrhagia in women with von Willebrand disease…
- Trial name
- VWDMin (NCT02606045)
- Testing
- recombinant von Willebrand factor, tranexamic acid
- Where
- 19 sites in United States
- Volunteers
- 39 people
- Sponsor
- Margaret Ragni
Hemophilia A
Prophylaxis Versus on Demand Treatment for Children With Hemophilia A
Comparison of the effect of three times a week prophylaxis on all bleeds with on-demand treatment for children with severe Hemophilia A.
- Trial name
- NCT01810666
- Testing
- Recombinant Factor VIII (Kogenate FS, BAY14-2222)
- Where
- 5 sites in China
- Volunteers
- 30 people
- Sponsor
- Bayer
Hemophilia B
A Gene Therapy Study for Hemophilia B
A Phase 1/2, Open-Label, Non-Randomized, Dose-Escalation Study of SPK-9001 in Subjects with Hemophilia B.
- Trial name
- NCT02484092
- Testing
- SPK-9001
- Where
- 10 sites in United States, Australia
- Volunteers
- 15 people
- Sponsor
- Pfizer
Chronic Myeloproliferative Disorders, Leukemia
Allogeneic Hematopoietic Cell Transplantation for Patients With Busulfex-based Regimen
RATIONALE: Giving chemotherapy, such as fludarabine and busulfan, before a donor peripheral stem cell transplant helps stop the growth of cancer or abnormal cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- LCCC0510 (NCT00448357)
- Testing
- rabbit anti-thymocyte globulin (ATG), therapeutic allogeneic lymphocytes, busulfan
- Where
- United States
- Volunteers
- 54 people
- Sponsor
- UNC Lineberger Comprehensive Cancer Center
Hemophilia B
A Safety and Efficacy Extension Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B
This study will examine the long-term safety and efficacy of rIX-FP for the control and prevention of bleeding episodes in children and adults with severe hemophilia B.
- Trial name
- NCT02053792
- Testing
- rIX-FP
- Where
- 42 sites in United States, Australia and 13 more
- Volunteers
- 97 people
- Sponsor
- CSL Behring
Haemophilia A
Efficacy and Safety of Turoctocog Alfa Pegol (N8-GP) for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Chinese Patients With Haemophilia A (pathfinder10)
The study investigates how well the medicine called turoctocog alfa pegol (N8-GP) works in previously treated Chinese patients with severe haemophilia A. Participants will be treated with N8-GP.
- Trial name
- Pathfinder10 (NCT05082116)
- Testing
- turoctocog alfa pegol (N8-GP)
- Where
- 11 sites in China
- Volunteers
- 36 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A
Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Pediatric Subjects With Hemophilia A
The primary objective of the study is to evaluate the safety of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc) in previously treated pediatric subjects with hemophilia A.
- Trial name
- Kids ALONG (NCT01458106)
- Testing
- BIIB031 (rFVIIIFc), FVIII (PK subgroup only)
- Where
- 23 sites in United States, Australia and 6 more
- Volunteers
- 71 people
- Sponsor
- Bioverativ Therapeutics Inc.
Hemophilia A
Study of Modified Recombinant Factor VIII (OBI-1) in Subjects With Congenital Hemophilia A
This study is to test whether the study drug (OBI-1) is safe and effective for the treatment of serious bleeding episodes in people with congenital hemophilia A.
- Trial name
- NCT01434511
- Testing
- OBI-1
- Where
- 3 sites in United States, South Africa and 1 more
- Volunteers
- 1 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A
A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants With Inhibitors
This multicenter, open-label study will evaluate the safety, efficacy and pharmacokinetics of prophylactic emicizumab treatment in participants previously treated with episodic or prophylactic bypassing agents.
- Trial name
- HAVEN 1 (NCT02622321)
- Testing
- Emicizumab, rFVIIa, aPCC
- Where
- 44 sites in United States, Australia and 12 more
- Volunteers
- 113 people
- Sponsor
- Hoffmann-La Roche
Bone Marrow Failure Syndrome, Thalassemia
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) to determine the minimum…
- Conditions
- Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia
- Trial name
- NCT03513328
- Testing
- Thiotepa--single daily dose, Thiotepa--escalated dose
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of Florida
Purpura, Thrombocytopaenic, Idiopathic
RAISE: Randomized Placebo-Controlled Idiopathic Thrombocytopenic Purpura (ITP) Study With Eltrombopag
The rationale for this Phase III study is to evaluate the 6 month safety and efficacy of eltrombopag in the treatment of previously treated subjects with chronic ITP.
- Trial name
- RAISE (NCT00370331)
- Testing
- eltrombopag
- Where
- 115 sites in United States, Austria and 23 more
- Volunteers
- 197 people
- Sponsor
- GlaxoSmithKline
Immune Thrombocytopenia (ITP)
A Study to Investigate the Efficacy and Safety of Lusutrombopag (S-888711) Tablets Administered to Adults With Immune Thrombocytopenia (ITP)
The primary objective of this study was to assess the efficacy of 3 dose levels of lusutrombopag (0.5 mg, 0.75 mg, and 1.0 mg) and placebo on platelet count.
- Trial name
- NCT01054443
- Testing
- Lusutrombopag
- Where
- 19 sites in United States
- Volunteers
- 20 people
- Sponsor
- Shionogi
Idiopathic Thrombocytopenic Purpura
Evaluating the Safety and Efficacy of Romiplostim (AMG 531) in Thrombocytopenic Subjects With Immune Thrombocytopenic Purpura (ITP)
The primary objective of this study is to evaluate the safety and tolerability of romiplostim in thrombocytopenic patients with ITP.
- Trial name
- NCT00111475
- Testing
- Romiplostim
- Volunteers
- 45 people
- Sponsor
- Amgen
Von Willebrand Disease
IL-11 in Women With Von Willebrand Disease and Refractory Menorrhagia
The purpose of this study is to determine the efficacy and safety of rhIL-11, when given subcutaneously for six consecutive months, in reducing menstrual blood loss in women with type 1 von Willebrand disease and refractory…
- Trial name
- NCT00524342
- Testing
- Oprelvekin, Interleukin 11, IL-11
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Margaret Ragni
Idiopathic Thrombocytopenic Purpura
Interventional Study in Adults With Immune Thrombocytopenia Purpura (ITP) Receiving Romiplostim
The purpose of this study is to describe the number of months with a platelet response over a 12 month treatment period and to describe ITP remission rates in adults with ITP receiving romiplostim.
- Trial name
- NCT01143038
- Testing
- Romiplostim
- Where
- 48 sites in United States, Australia and 7 more
- Volunteers
- 75 people
- Sponsor
- Amgen
Factor X Deficiency
Safety & Efficacy of BPL's High Purity FACTOR X in Treatment of Factor X Deficient Subjects Undergoing Surgery
To primary efficacy variable is to assess the presence or absence of excessive blood loss during and after surgery. The secondary efficacy endpoints are as follows: 1.
- Trial name
- Ten03 (NCT01086852)
- Testing
- FACTOR X
- Where
- 7 sites in United States, Spain and 2 more
- Volunteers
- 4 people
- Sponsor
- Bio Products Laboratory
Idiopathic Thrombocytopenic Purpura, Thrombocytopenia in Pediatric Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
Safety and Efficacy Study of Romiplostim (AMG 531) to Treat ITP in Pediatric Subjects
The purpose of this study is to evaluate the safety and tolerability of romiplostim (AMG 531) in the treatment of thrombocytopenia in pediatric subjects with chronic ITP.
- Conditions
- Idiopathic Thrombocytopenic Purpura, Thrombocytopenia in Pediatric Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP), Thrombocytopenia in Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
- Trial name
- NCT00515203
- Testing
- AMG 531
- Volunteers
- 22 people
- Sponsor
- Amgen
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
Hemophilia A
Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A
Primary Objective: To evaluate the safety of BIVV001 in previously treated pediatric participants with hemophilia A. Secondary Objectives: To evaluate the efficacy of BIVV001 as a prophylaxis treatment.
- Trial name
- XTEND-Kids (NCT04759131)
- Testing
- efanesoctocog alfa (BIVV001)
- Where
- 40 sites in United States, Australia and 13 more
- Volunteers
- 74 people
- Sponsor
- Bioverativ, a Sanofi company
Hemophilia A Without Inhibitor
The Hemophilia Inhibitor Prevention Trial
This is a multi-center randomized phase III clinical trial, the Inhibitor Prevention Trial, in which Eloctate will be compared with Emicizumab, using adaptive design, to prevent inhibitors in patients with severe hemophilia A.
- Trial name
- NCT04303559
- Testing
- Eloctate Injectable Product, Emicizumab Injection [Hemlibra]
- Where
- 3 sites in United States
- Volunteers
- 1 people
- Sponsor
- Margaret Ragni