Clinical trials: Bleeding Diathesis
Hemophilia A
A Study of PEGylated Recombinant Factor VIII (BAX855) in Previously Untreated Young Children With Severe Hemophilia A
This study is for young children with severe hemophilia A who have previously not been treated with BAX855 or other FVIII concentrates. The main aim of the study is to check for side effects from treatment with BAX855.
- Trial name
- NCT02615691
- Testing
- PEGylated Recombinant Factor VIII, ITI
- Where
- 91 sites in United States, Austria and 21 more
- Volunteers
- 120 people
- Sponsor
- Baxalta now part of Shire
Thrombotic Thrombocytopenic Purpura
Study of Cyclosporine or Corticosteroids as an Adjunct to Plasma Exchange in Thrombotic Thrombocytopenic Purpura (TTP)
This research involves the use of immune base therapy as an adjunct to plasma exchange, the present standard of care for thrombotic thrombocytopenic purpura (TTP). Funding source -FDA OOPD
- Trial name
- NCT00713193
- Testing
- Cyclosporine, Prednisone
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- Ohio State University
Hemophilia A
Pharmacokinetic Comparison of Advate rAHF-PFM With Recombinate rAHF in Patients With Severe Hemophilia A
The purpose of this study is to compare the pharmacokinetic parameters and safety of Advate rAHF-PFM versus Recombinate rAHF in well described previously treated patients with severe hemophilia A (factor VIII level < 1%).
- Trial name
- NCT00666406
- Testing
- Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method (rAHF-PFM), Recombinant Factor VIII (rAHF)
- Where
- Germany
- Volunteers
- 9 people
- Sponsor
- Baxalta now part of Shire
Hemarthrosis, Hemophilia
Low-Cost Platelet-Rich Plasma for Hemarthropathy
Orthopedic complications can be injuries or diseases that affect the bones, muscles, and joints. Hemarthropathy is due to bleeding disorders, most commonly hemophilia, and can result in severe issues related to chronic amounts of…
- Trial name
- NCT06543368
- Testing
- Intra articular PRP Injection
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- University of Utah
Essential Thrombocythemia (ET)
Exploratory Multi-centre Trial In Patients With ET Treated With XAGRID®
This study is hypothesis-generating to explore the impact of JAK2 (V617F) mutation status on the treatment response to anagrelide hydrochloride
- Trial name
- EMIX (NCT01352585)
- Testing
- Anagrelide hydrochloride
- Where
- 10 sites in Italy
- Volunteers
- 47 people
- Sponsor
- Shire
Thrombotic Thrombocytopenic Purpura
Evaluating the Effectiveness of Adding Rituximab to Standard Treatment for Thrombotic Thrombocytopenic Purpura (TTP)
Thrombotic thrombocytopenic purpura (TTP) is a rare disorder that causes blood clots to form in blood vessels. The main treatment for TTP is plasma exchange, in which affected patients receive transfusions of plasma, the liquid…
- Trial name
- STAR (NCT00799773)
- Testing
- Rituximab, Plasma exchange, Corticosteroids
- Where
- 23 sites in United States
- Volunteers
- 3 people
- Sponsor
- Carelon Research
Stem Cell Transplantation, Bone Marrow Transplantation
Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells
Many genetic diseases of lymphohematopoietic cells (such as sickle cell anemia, thalassemia, Diamond-Blackfan anemia, Combined Immune Deficiency (CID), Wiskott-Aldrich syndrome, chronic granulomatous disease, X-linked…
- Conditions
- Stem Cell Transplantation, Bone Marrow Transplantation, Peripheral Blood Stem Cell Transplantation, Allogeneic Transplantation
- Trial name
- NCT02512679
- Testing
- Cyclophosphamide Dose Level 1, Cyclophosphamide Dose Level 2, Cyclophosphamide Dose Level 3
- Volunteers
- 20 people
- Sponsor
- Children's Hospital Los Angeles
Primary Immune Thrombocytopenia
A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP).
This is a randomized, double-blind placebo-controlled multicenter phase 3 trial to evaluate the efficacy and safety of ARGX-113 in participants with primary ITP.
- Trial name
- ADVANCE (NCT04188379)
- Testing
- efgartigimod
- Where
- 121 sites in United States, Austria and 16 more
- Volunteers
- 131 people
- Sponsor
- argenx
Trauma/Injury Problem
Early Whole Blood in Patients Requiring Transfusion After Major Trauma
The proposal will assess if patients who require massive transfusion can be accurately predicted early after emergency department arrival and assess if the use of stored whole blood during initial resuscitation will reduce…
- Trial name
- NCT01227005
- Testing
- Transfusion of blood products
- Where
- United States
- Volunteers
- 107 people
- Sponsor
- The University of Texas Health Science Center, Houston
Immune Thrombocytopenic Purpura (ITP)
Safety and Tolerability of M254 in Healthy Volunteers and Immune Thrombocytopenic Purpura (ITP) Patients
The purpose of this study is to assess safety, tolerability, pharmacokinetics, and pharmacodynamics of M254 after administration of a single ascending dose and repeat doses in healthy volunteers and immune thrombocytopenic…
- Trial name
- NCT03866577
- Testing
- Biological: M254, Intravenous immunoglobulin (IVIg)
- Where
- 28 sites in United States, Belgium and 5 more
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Momenta Pharmaceuticals, Inc.
Chronic Myeloproliferative Disorders, Leukemia
Busulfan, Fludarabine, and Total-Body Irradiation in Treating Patients Who Are Undergoing a Donor Stem Cell Transplant for Hematologic Cancer
RATIONALE: Giving low doses of chemotherapy, such as fludarabine and busulfan, before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- Bu Flu TBI (NCT00245037)
- Testing
- therapeutic allogeneic lymphocytes, busulfan, cyclosporine
- Where
- United States
- Volunteers
- 147 people
- Sponsor
- OHSU Knight Cancer Institute
Hemophilia A
Study Evaluating Safety Of Patients Switching To ReFacto AF In Usual Care Settings
The study will be investigating safety in patients who switch to ReFacto AF from ReFacto and other Factor VIII products.
- Trial name
- NCT00884390
- Testing
- moroctocog alfa (AF-CC) (ReFacto AF), Laboratory tests
- Where
- 74 sites in Austria, Belgium and 12 more
- Volunteers
- 208 people
- Sponsor
- Pfizer
Congenital Afibrinogenemia, Congenital Hypofibrinogenemia
Pharmacokinetic, Efficacy and Safety of BT524 in Patients With Congenital Fibrinogen Deficiency
This was a prospective, open-label, multicenter, phase I/III study investigating the 14-day single-dose pharmacokinetic and pharmacodynamic properties, efficacy and safety of BT524 following intravenous administration in the…
- Trial name
- NCT02065882
- Testing
- BT524 (Part I), BT524 (Part II)
- Where
- 6 sites in Bulgaria, Egypt and 3 more
- Volunteers
- 67 people
- Sponsor
- Biotest
Adeno-Associated Virus (AAV), Blood Coagulation Disorder
Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors
SPK-8016 is in development for the treatment of patients with inhibitors to FVIII. This Phase 1/2, open-label, non-randomized, dose-finding study to evaluate the safety, efficacy, and tolerability of SPK-8016 in adult males with…
- Conditions
- Adeno-Associated Virus (AAV), Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders
- Trial name
- NCT03734588
- Testing
- SPK-8016
- Where
- 11 sites in United States
- Volunteers
- 4 people
- Sponsor
- Spark Therapeutics, Inc.
Hemophilia A
Study Evaluating Refacto For Pharmacovigilance
The purpose of this study is to investigate the effectiveness and safety of treatment with ReFacto under conditions of routine therapy. Furthermore a continuous benefit/risk assessment will be done.
- Trial name
- NCT00195442
- Testing
- Moroctocog alfa
- Where
- 26 sites in Austria, Germany
- Volunteers
- 288 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
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
Severe Haemophilia A
Assess the Efficacy and Safety of Personalized Prophylaxis Human-cl rhFVIII in Patients With Severe Haemophilia A
The rationale of this study is to further fine-tune and individualize prophylactic treatment of patients with severe Haemophilia A with the goal of keeping the trough FVIII level above 1% between doses.
- Trial name
- NCT02256917
- Testing
- Human cl rhFVIII
- Where
- 30 sites in United States, Canada and 7 more
- Volunteers
- 58 people
- Sponsor
- Octapharma
Hemophilia A, Severe Hemophilia A
An Open-label Safety and Efficacy Study of Recombinant FVIII in Patients With Severe Hemophilia A
This multicenter, open-label, phase 3 extension study will investigate the safety and efficacy of rVIII-SingleChain for prophylaxis and on-demand treatment of bleeding episodes in at least 200 previously treated patients (PTPs)…
- Trial name
- NCT02172950
- Testing
- rVIII-SingleChain
- Where
- 64 sites in United States, Australia and 23 more
- Volunteers
- 246 people
- Sponsor
- CSL Behring
Accelerated Phase Chronic Myelogenous Leukemia, Acute Undifferentiated Leukemia
Fludarabine and Total-Body Irradiation Followed By Donor Stem Cell Transplant and Cyclosporine and Mycophenolate Mofetil in Treating HIV-Positive Patients With or Without Cancer
This clinical trial studies the side effects and best dose of giving fludarabine and total-body irradiation (TBI) together followed by a donor stem cell transplant and cyclosporine and mycophenolate mofetil in treating human…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Acute Undifferentiated Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission
- Trial name
- NCT00112593
- Testing
- fludarabine phosphate, total-body irradiation, peripheral blood stem cell transplantation
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Fred Hutchinson Cancer Center
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