Clinical trials: Coagulation Abnormalities
Hemophilia A
A Trial Investigating Safety and Efficacy of Treatment With BAY94-9027 in Severe Hemophilia A
Haemophilia A is an inherited disorder in which one of the proteins, Factor VIII, needed to form blood clots is missing or not present in sufficient levels.
- Trial name
- PROTECT-VIII (NCT01580293)
- Testing
- BAY94-9027
- Where
- 59 sites in United States, Austria and 18 more
- Volunteers
- 145 people
- Sponsor
- Bayer
Hemophilia B
Study of Recombinant Factor IX Product, IB1001, in Subjects With Hemophilia B
Primary Objective: To evaluate the safety (acute effects associated with infusions, and inhibitor development), pharmacokinetics (PK), and efficacy with respect to breakthrough bleeding during prophylaxis and with respect to…
- Trial name
- NCT00768287
- Testing
- IB1001, nonacog alfa
- Where
- 23 sites in United States, France and 5 more
- Volunteers
- 77 people
- Sponsor
- Medexus Pharma, Inc.
Congenital Bleeding Disorder, Haemophilia A
Efficacy and Safety of Turoctocog Alfa for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Chinese Patients With Haemophilia A
This trial is conducted in China. The aim of this trial is to evaluate the clinical efficacy of turoctocog alfa in treatment of bleeding episodes in Chinese patients with severe haemophilia A (FVIII≤1%).
- Trial name
- guardian TM 7 (NCT02938585)
- Testing
- turoctocog alfa
- Where
- 11 sites in China
- Volunteers
- 68 people
- Sponsor
- Novo Nordisk A/S
Von Willebrand Diseases
Bleeding Incidence in VWD Patients Undergoing On-Demand Treatment
The purpose of this study is to prospectively obtain reliable data on the bleeding and treatment pattern of patients with VWD undergoing on-demand treatment with a VWF-containing product over a period of 6 months.
- Trial name
- NCT04053699
- Testing
- Von Willebrand Factor-Containing Product
- Where
- 15 sites in United States, Belarus and 6 more
- Volunteers
- 56 people
- Sponsor
- Octapharma
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
Hemophilia A
Dose-Response Study of Recombinant Factor VIII Manufactured Protein-Free (rAHF-PFM) in Patients With Hemophilia A
The purpose of this study is to determine the effect of 3 doses of ADVATE rAHF-PFM on initial recovery (% increase [IU/dL] per IU/kg infused) and major single-infusion pharmacokinetic parameters.
- Trial name
- NCT00289536
- Testing
- Antihemophilic factor, recombinant, manufactured protein-free
- Where
- 8 sites in United States
- Volunteers
- 38 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A
Study Evaluating Pharmacovigilance Of Refacto AF
The purpose of this observational study is to describe the incidence of adverse events among patients treated with Refacto AF in usual health care settings in Germany and Austria.
- Trial name
- NCT00895037
- Testing
- ReFacto AF (Moroctocog alfa)
- Where
- 24 sites in Austria, Germany
- Volunteers
- 101 people
- Sponsor
- Pfizer
Hemophilia A
Pharmacokinetic Study of ADVATE Reconstituted in 2 mL Sterile Water for Injection
The purpose of this study is to determine the pharmacokinetics and safety of Antihemophilic factor, recombinant, manufactured protein-free (rAHF-PFM) reconstituted in 2 mL sterile water for injection (SWFI) and compare with those…
- Trial name
- NCT00952822
- Testing
- Antihemophilic factor, recombinant, manufactured protein-free (rAHF-PFM). (Antihemophilic factor is also known as Factor VIII)
- Where
- 11 sites in United States
- Volunteers
- 52 people
- Sponsor
- Baxalta now part of Shire
Congenital Bleeding Disorder, Haemophilia A
Safety and Efficacy of Turoctocog Alfa in Previously Treated Male Children With Haemophilia A
This trial is conducted in Asia, Europe, and North and South America. The aim of this clinical trial is to investigate the safety and efficacy of turoctocog alfa (recombinant factor VIII, rFVIII (N8)) in male previously treated…
- Trial name
- guardian™ 3 (NCT01138501)
- Testing
- turoctocog alfa
- Where
- 38 sites in United States, Brazil and 11 more
- Volunteers
- 65 people
- Sponsor
- Novo Nordisk A/S
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
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
Study Evaluating Safety And Efficacy Of Moroctocog Alfa (AF-CC) In Previously Treated Hemophilia A Patients
The study will be investigating pharmacokinetics, safety and efficacy in patients less than 12 years of age with severe hemophilia A that have been previously treated with Factor VIII products ( including blood products).
- Trial name
- NCT00914459
- Testing
- Moroctocog alfa ( AF-CC), Laboratory tests
- Where
- 17 sites in Finland, Georgia and 7 more
- Volunteers
- 37 people
- Sponsor
- Pfizer
Hemophilia A, Hemophilia B
Study of Ataluren (PTC124) in Hemophilia A and B
Hemophilia A (HA) and hemophilia B (HB) are inherited bleeding disorders caused by mutations in the gene for factor VIII (FVIII) and factor IX (FIX), respectively. These proteins are essential for blood clotting.
- Trial name
- NCT00947193
- Testing
- Ataluren
- Where
- 13 sites in United States, Canada and 2 more
- Volunteers
- 13 people
- Sponsor
- PTC Therapeutics
Wiskott-Aldrich Syndrome, Thrombocytopenia
Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients
The purpose of this project is to describe the pathophysiology of thrombocytopenia and bleeding in patients with Wiskott-Aldrich Syndrome (WAS) and determine the response to thrombopoietic agents in vitro and in vivo.
- Conditions
- Wiskott-Aldrich Syndrome, Thrombocytopenia, Bleeding
- Trial name
- WAS (NCT00909363)
- Testing
- Promacta, blood drawing in patients with WAS, blood drawing in healthy controls
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Weill Medical College of Cornell University
Hemophilia A With Inhibitor
The Hemophilia Inhibitor Eradication Trial
This is a multi-center randomized phase III clinical trial, the Inhibitor Eradication Trial, in which Eloctate ITI plus Emicizumab will be compared with Eloctate ITI alone to eradicate inhibitors in severe hemophilia A.
- Trial name
- NCT04303572
- Testing
- Eloctate ITI, Emicizumab
- Where
- 2 sites in United States
- Volunteers
- 1 people
- Sponsor
- Margaret Ragni
Hemophilia B
Dose-escalation Study to Investigate the Safety, PK, and PD of ISU304/CB2679d in Hemophilia B Patients
This study is a phase 1, open-label, multi-center, dose-escalation study to investigate the safety, pharmacokinetics and pharmacodynamics of ISU304/CB2679d in previously treated hemophilia B patients.
- Trial name
- NCT03186677
- Testing
- ISU304/CB2679d/Dalcinonacog alfa 75~150 IU/kg, BeneFIX
- Where
- 3 sites in South Korea
- Volunteers
- 11 people
- Sponsor
- ISU Abxis Co., Ltd.
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
Hemophilia B
Trial of AAV5-hFIX in Severe or Moderately Severe Hemophilia B
This study evaluates how safe gene therapy treatment with AAV5-hFIX is in adult patients with severe or moderately severe hemophilia B and severe bleeding type.
- Trial name
- NCT02396342
- Testing
- AAV5-hFIX
- Where
- 7 sites in Denmark, Germany and 1 more
- Volunteers
- 10 people
- Sponsor
- CSL Behring
Hemophilia B
Nonacog Alfa Prophylaxis And Treatment Of Bleeding Episodes In Previously Treated Patients With Hemophilia B
Nonacog alfa is indicated for the control and prevention of hemorrhagic episodes and for routine and surgical prophylaxis in patients with hemophilia B.
- Trial name
- NCT04286412
- Testing
- Nonacog alfa
- Where
- 5 sites in India
- Volunteers
- 25 people
- Sponsor
- Pfizer
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.