Clinical trials: Clotting Factor Deficiency
Health Services Accessibility, Blood Coagulation Factor Deficiencies
Accessibility of Prophylaxis and On-demand Treatment for Persons With Haemophilia and Other Coagulation Deficiencies
The current treatment of people with haemophilia and other bleeding deficiencies is largely based on clotting factor replacement therapy. The injections can be repeated several times a week according to a personalized schedule.
- Trial name
- PHAREO (NCT04775888)
- Where
- 34 sites in France
- Volunteers
- 500 people
- Sponsor
- RESCUe - RESeau Cardiologie Urgence / RESUVal - RESeau des Urgences de la vallée du Rhône
Hemophilia A
Efficacy and Safety Study of a Recombinant Protein-Free Manufactured Factor VIII (rAHF-PFM) in Previously Untreated Hemophilia A Patients
The purpose of this study is to evaluate whether Antihemophilic factor, recombinant, manufactured protein-free (rAHF-PFM) is effective and safe in the treatment of hemophilia A patients who have not been treated with factor VIII…
- Trial name
- NCT00157157
- Testing
- Recombinant Antihemophilic Factor Manufactured and Formulated without Added Human or Animal Proteins (rAHF-PFM)
- Where
- 35 sites in United States, Austria and 8 more
- Volunteers
- 66 people
- Sponsor
- Baxalta now part of Shire
Atrial Fibrillation, Venous Thromboses
Pharmacogenetic Dosage Algorithm for Acenocoumarol
The use of coumarins has been a challenge for doctors because of its narrow therapeutic range and they show great inter and intra-individual variability in the dose necessary to achieve an international normalized ratio (INR)…
- Conditions
- Atrial Fibrillation, Venous Thromboses, Cardiac Valve Disease
- Trial name
- NCT03015025
- Testing
- Acenocoumarol
- Where
- Spain
- Volunteers
- 340 people
- Sponsor
- Instituto de Investigación Hospital Universitario La Paz
Inherited Bleeding Disorders
Study to Evaluate the Use of Ultrasound in the Diagnosis of Hemophilic Joint Disease
The purpose of this study is to see whether power Doppler sonography (PDS) can be used to diagnose synovitis in patients with inherited bleeding disorders.
- Trial name
- NCT00212485
- Testing
- Power Doppler Sonography, MRI
- Where
- United States
- Volunteers
- 43 people
- Sponsor
- New York Presbyterian Hospital
Factor VIII Deficiency
A Phase 3 Open-label Interventional Study of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein, Efanesoctocog Alfa (BIVV001), in Patients With Severe Hemophilia A
Primary Objective: To evaluate the efficacy of BIVV001 as a prophylaxis treatment in prophylaxis treatment arm. Secondary Objectives: To evaluate the efficacy of BIVV001 as a prophylaxis treatment.
- Trial name
- XTEND-1 (NCT04161495)
- Testing
- efanesoctocog alfa (BIVV001)
- Where
- 47 sites in United States, Argentina and 17 more
- Volunteers
- 159 people
- Sponsor
- Bioverativ, a Sanofi company
Hemophilia A
Pharmacokinetics, Efficacy and Safety Study of IMMUNATE SD (Human Plasma-Derived Coagulation Factor VIII Concentrate) in Hemophilia A Patients
The purpose of this study is to evaluate whether IMMUNATE S/D is effective and safe in the treatment of hemophilia A patients. The study consists of 3 parts: Part 1 is a pharmacokinetic comparison of IMMUNATE S/D and its…
- Trial name
- NCT00162019
- Testing
- Human Plasma-Derived Coagulation Factor VIII Concentrate (Virus Inactivated by Polysorbate 80 Treatment and Vapor Heat Treatment)
- Where
- 5 sites in Bulgaria, Czechia and 2 more
- Volunteers
- 56 people
- Sponsor
- Baxalta now part of Shire
Von Willebrand Disease, Congenital Coagulation Factors Deficiency
Evaluation of Menses in Congenital Bleeding Disorders
Menorrhagia is the term used to define excessive menstrual blood losses, as often based on a subjective judgment of women. Many arbitrary values, expressed in milliliters of blood, have been proposed to define this symptom: 60 on…
- Conditions
- Von Willebrand Disease, Congenital Coagulation Factors Deficiency, Haemophilia Carriers
- Trial name
- MEVA-3 (NCT01261936)
- Where
- 3 sites in Italy
- Volunteers
- 30 people
- Sponsor
- University of L'Aquila
Thromboembolism
Applying Pharmacogenetic Algorithms to Individualize Dosing of Warfarin
The purpose of this study is to determine whether DNA analysis improves the efficiency of dosing and safety in patients who are being started on warfarin therapy.Warfarin, a blood thinner (anticoagulant) prescribed to 1-2 million…
- Trial name
- Coumagen-II (NCT00927862)
- Testing
- IWPC adapted genotype-guided dosing algorithm for warfarin, Modified IWPC genetic-guided warfarin dosing algorithm, Standard of care treatment
- Where
- United States
- Volunteers
- 2,415 people
- Sponsor
- Intermountain Health Care, Inc.
Adverse Drug Events, Adverse Drug Reactions
YouScript IMPACT Registry
This multicenter observational study aims to investigate the benefits of providing pharmacogenetic testing with the YouScript Personalized Prescribing System which includes a clinical decision support tool and individualized…
- Conditions
- Adverse Drug Events, Adverse Drug Reactions, Drug Interaction Potentiation, Drug Metabolism, Poor, CYP2D6-RELATED
- Trial name
- IMPACT (NCT02191358)
- Where
- 7 sites in United States
- Volunteers
- 800 people
- Sponsor
- Genelex Corporation
Liver Decompensation, Hepatocellular Carcinoma
The Second Multicenter Hemophilia Cohort Study
The Second Multicenter Hemophilia Cohort Study (MHCS-II) will evaluate and prospectively follow approximately 4500 persons with hemophilia who were exposed to hepatitis C virus (HCV).
- Conditions
- Liver Decompensation, Hepatocellular Carcinoma, Non-Hodgkin Lymphoma
- Trial name
- NCT00341705
- Where
- 48 sites in United States, Brazil and 6 more
- Volunteers
- 2,565 people
- Sponsor
- National Cancer Institute (NCI)
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
Factor VII Deficiency
Study Comparing the Pharmacokinetic of Biosimilar Eptacog Alfa With Novoseven®, in Patients With Congenital Factor VII Deficiency
The purpose of this multicentre, randomized, double blinded, single dose, two-way cross-over study, is to compare the pharmacokinetics (PK) of biosimilar eptacog alfa (activated) with Novoseven in 24 patients, adult and children…
- Trial name
- NCT03079063
- Testing
- Eptacog alfa, biosimilar, Novoseven
- Where
- 2 sites in Iran
- Volunteers
- 24 people
- Sponsor
- AryoGen Pharmed Co.
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.
HIV Infections, Hemophilia A
The Effects of AZT and Ibuprofen on HIV-Infected Patients With Hemophilia
To determine if platelet dysfunction and/or pharmacologic drug interaction occurs in patients taking both AZT and ibuprofen, which might account for enhanced bleeding tendency.
- Trial name
- NCT00002276
- Testing
- Ibuprofen, Zidovudine
- Where
- United States
- Sponsor
- University of Pittsburgh
Factor X Deficiency
A Study to Investigate Bio Product Laboratory Ltd (BPL's) Factor X in the Prophylaxis of Bleeding in Children <12 Years
The primary objective of the study is to assess the efficacy of FACTOR X in the prevention of bleeding when given as routine prophylaxis over 12 months. The secondary objectives of the study are: 1.
- Trial name
- NCT01721681
- Testing
- FACTOR X
- Where
- 3 sites in United Kingdom
- Volunteers
- 9 people
- Sponsor
- Bio Products Laboratory
HIV Infections, Hepatitis C
Treatment of Hepatitis C in Hemophilic Patients With HIV
Pegylated interferon (PEG-interferon) and ribavirin are accepted treatments for hepatitis C virus (HCV) infection. However, HCV infection progresses differently in patients who are coinfected with HIV and in hemophiliacs.
- Conditions
- HIV Infections, Hepatitis C, Hemophilia
- Trial name
- NCT00055341
- Testing
- Peginterferon alfa-2a, Ribavirin
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Factor XIII Deficiency, ST-Elevation Myocardial Infarction
Factor XIII and Other Biomarkers in ST Segment Elevation Myocardial Infarction
In medical practice, a combination of clinical exam, electrocardiograms, circulating biomarkers, and imaging is used to gain insights on the prognosis after myocardial infarction.
- Conditions
- Factor XIII Deficiency, ST-Elevation Myocardial Infarction, Myocardial Haemorrhage, Coronary Microvascular Disease
- Trial name
- eXIST (NCT03523624)
- Where
- Italy
- Volunteers
- 35 people
- Sponsor
- University of Padova
Congenital Bleeding Disorder, Congenital FXIII Deficiency
Comparison of a Blood Clotting Drug (Recombinant Factor XIII) Produced by Two Different Manufacturers in Healthy Male Subjects
This trial is conducted in Europe. The aim of this clinical trial is to compare the metabolism of a blood-clotting drug (recombinant Factor XIII)) produced by two different manufacturers (Novo Nordisk and Avecia) in healthy male…
- Conditions
- Congenital Bleeding Disorder, Congenital FXIII Deficiency, Healthy
- Trial name
- NCT01082406
- Testing
- catridecacog, recombinant factor XIII
- Where
- United Kingdom
- Volunteers
- 51 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
Acquired Fibrinogen Deficiency
Phase 3 Study of Fibrinogen Concentrate (CSL511) in Subjects With Pseudomyxoma Peritonei Undergoing Cytoreductive Surgery
This study is a phase 3, prospective, single center, randomized, open label, controlled, parallel arm, interventional study to investigate the efficacy and safety of CSL511, in participants undergoing cytoreductive surgery (CRS)…
- Trial name
- NCT06617897
- Testing
- CSL511 Fibrinogen concentrate (human), Cryoprecipitate
- Where
- United Kingdom
- Volunteers
- 90 people
- Sponsor
- CSL Behring
Healthy Participants, Hemophilia A
A Study of Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SR604 in Two Participants Groups (Part A: Healthy Participants, and Part B: Participants With Hemophilia A or Hemophilia B or Factor VII Deficiency)
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamic (PD) of SR604 in healthy participants (Part A) and to evaluate the safety, tolerability, PK, PD, and efficacy of SR604…
- Conditions
- Healthy Participants, Hemophilia A, Hemophilia B, Factor VII Deficiency
- Trial name
- NCT06349473
- Testing
- SR604
- Where
- 10 sites in United States, Canada
- Volunteers
- 31 people, healthy volunteers welcome
- Sponsor
- Equilibra Bioscience LLC