Clinical trials: Coagulopathy
Blood Coagulation Disorders, Hemophilia A
Trial to Evaluate the Efficacy and Safety of a New Full Length Recombinant Human FVIII for Hemophilia A
The study will assess the pharmacokinetics (part A) safety, tolerability, and efficacy of prophylaxis treatment (2 to 3 times a week) (part B) with BAY81-8973 over a one year period (split into two six month treatment periods).
- Trial name
- Leopold I (NCT01029340)
- Testing
- Recombinant Factor VIII (BAY81-8973), Recombinant Factor VIII (Kogenate FS, BAY14-2222)
- Where
- 60 sites in United States, Argentina and 20 more
- Volunteers
- 74 people
- Sponsor
- Bayer
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
Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
Open Label Extension Study of AMG 531 in Japanese Subjects With ITP
The purpose of this study is to assess the safety and efficacy of long term dosing of AMG 531 in thrombocytopenic Japanese subjects with ITP.
- Trial name
- NCT00440037
- Testing
- AMG 531
- Where
- 13 sites in Japan
- Volunteers
- 44 people
- Sponsor
- Kyowa Kirin Co., Ltd.
Severe Hemophilia
Study to Evaluate Safety and Tolerability of a Single Dose of PF-06741086 in Chinese Adult Participants With Severe Hemophilia
This Phase 1 study will be a single-arm, open-label, non-randomized, non-controlled investigation of the safety, tolerability, pharmacokinetics, and pharmacodynamics of PF-06741086 in Chinese adult participants with severe…
- Trial name
- NCT04878731
- Testing
- PF-06741086
- Where
- China
- Volunteers
- 6 people
- Sponsor
- Pfizer
Hemophilia A, Gene Therapy
Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5
This study is being conducted by BioMarin Pharmaceutical Inc. as an open label, single dose study to determine the safety of valoctocogene roxaparvovec (an Adenovirus-Associated Virus (AAV) based gene therapy vector) in severe…
- Conditions
- Hemophilia A, Gene Therapy, Clotting Disorders, Blood Disorder
- Trial name
- GENEr8-AAV5+ (NCT03520712)
- Testing
- Valoctocogene Roxaparvovec
- Where
- 9 sites in South Africa, South Korea and 2 more
- Volunteers
- 3 people
- Sponsor
- BioMarin Pharmaceutical
Hemophilia
Weight-based Dosing in Hemophilia A
Hemophilia A is an inherited (genetic) disease where a protein, factor VIII (FVIII), which promotes blood clotting is missing or does not work properly. Individuals with hemophilia A are at risk for bleeding.
- Trial name
- NCT02586012
- Testing
- rFVIII
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Craig Seaman
Primary Myelofibrosis (PMF), Post Polycythaemia Myelofibrosis (PPV MF)
Exploratory Phase II Study of INC424 Patients With Primary Myelofibrosis (PMF) or Post Polycythaemia Myelofibrosis (PPV MF) or Post Essential Thrombocythaemia Myelofibrosis (PET-MF)
The primary objective of this study is to evaluate the efficacy of INC424 in patients with PMF, PPV MF, or PET-MF using a composite measure of either an objective endpoint (> 50% reduction in splenomegaly using palpitation at 48…
- Conditions
- Primary Myelofibrosis (PMF), Post Polycythaemia Myelofibrosis (PPV MF), Post Essential Thrombocythaemia Myelofibrosis (PET-MF)
- Trial name
- MACS2030 (NCT01558739)
- Testing
- INC424
- Where
- 10 sites in United Kingdom
- Volunteers
- 48 people
- Sponsor
- Novartis Pharmaceuticals
Acute Coagulopathy
Comparison of Rapid Thrombelastography and Conventional Coagulation Testing for Haemostatic Resuscitation in Trauma
The purpose of this study is to compare rapid thrombelastography (r-TEG) with conventional coagulation testing for diagnosing and treating coagulation abnormalities in severely injured patients who are likely to require…
- Trial name
- NCT01536496
- Testing
- Blood product transfusion based on conventional coagulation tests., Blood product transfusion based on rapid thrombelastography (r-TEG) results.
- Where
- United States
- Volunteers
- 114 people
- Sponsor
- Denver Health and Hospital Authority
Burn Body Region Unspecified, Burn Shock
Enteral Resuscitation Nepal (Pilot Study)
Nepal and the South Asian sub-continent carry some of the highest rates of burn injury globally, with an associated high morbidity and mortality.
- Trial name
- NCT04732624
- Testing
- Oral Rehydration Solution, Lactated Ringer
- Where
- Nepal
- Volunteers
- 30 people
- Sponsor
- University of Washington
Thalassemia, Sickle Cell Disease
Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation
Allogeneic blood and marrow transplantation remains the only viable cure for children who suffer from many serious non-malignant hematological diseases. Transplantation, however, carries a high risk of fatal complications.
- Conditions
- Thalassemia, Sickle Cell Disease, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome
- Trial name
- NCT01319851
- Testing
- Alefacept
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Emory University
Immune Thrombocytopenia
Safety and Treatment Satisfaction in Adults With Chronic ITP After Switching to Avatrombopag From Eltrombopag or Romiplostim
Evaluate the safety and tolerability of avatrombopag given for 90 days after stopping treatment with eltrombopag or romiplostim.
- Trial name
- NCT04638829
- Testing
- Avatrombopag Oral Tablet
- Where
- 20 sites in United States
- Volunteers
- 60 people
- Sponsor
- Sobi, Inc.
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
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
Acute Heart Failure
Study of Safety, Tolerability and Pharmacokinetics of Serelaxin in Japanese Acute Heart Failure (AHF) Patients
This is a multicenter, randomized, double-blind, placebo-controlled study to assess safety, tolerability and pharmacokinetics and to explore efficacy of IV infusion of 10 µg/kg/day and 30 µg/kg/day serelaxin for 48 hours compared…
- Trial name
- NCT02002702
- Testing
- Serelaxin
- Where
- 15 sites in Japan
- Volunteers
- 46 people
- Sponsor
- Novartis Pharmaceuticals
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
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
Safety & Efficacy of Encapsulated Allogeneic FVIII Cell Therapy in Haemophilia A
SIG-001-121 is a first-in-human (FIH), phase 1/2, multi-centre, open-label, dose escalation study to assess the safety, tolerability, and preliminary efficacy of SIG-001 in adults with severe or moderately severe haemophilia A…
- Trial name
- NCT04541628
- Testing
- SIG-001
- Where
- 6 sites in United States, United Kingdom
- Volunteers
- 3 people
- Sponsor
- Eli Lilly and Company
Primary Immune Thrombocytopenia
A Study to Evaluate the Efficacy and Safety of Efgartigimod PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia
This is a phase 3, multicenter, randomized, double-blinded, placebo-controlled, parallel-group trial to evaluate the efficacy, safety, and effect on QoL/PRO of efgartigimod PH20 SC treatment in adult patients with primary ITP.
- Trial name
- ADVANCE SC (NCT04687072)
- Testing
- Efgartigimod PH20 SC
- Where
- 200 sites in United States, Argentina and 30 more
- Volunteers
- 207 people
- Sponsor
- argenx
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.
Von Willebrand Disease
Surveillance of Safety and Efficacy of Wilate in Patients With Von Willebrand Disease
This is an observational study, hence there is no study hypothesis
- Trial name
- NCT01602419
- Testing
- Patients using Wilate as standard of care
- Where
- 24 sites in United States, Argentina and 8 more
- Volunteers
- 120 people
- Sponsor
- Octapharma