Clinical trials: Coagulation Factor Deficiency
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
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
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
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.
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
Factor XIII Deficiency
A Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
Congenital deficiency of factor XIII (FXIII) is an extremely rare inherited disorder associated with potentially life-threatening bleeding. Factor XIII Concentrate is given to patients whose blood is lacking factor XIII.
- Trial name
- NCT00885742
- Testing
- FXIII Concentrate (Human)
- Where
- 23 sites in United States, Spain
- Volunteers
- 41 people
- Sponsor
- CSL Behring
Congenital Bleeding Disorder, Congenital FVII Deficiency
Observational Study on the Efficacy and Safety of NovoSeven® During "Real-life" Usage in Germany
This NON INTERVENTIONAL OBSERVATIONAL STUDY is conducted in Europe. The primary aim is to observe the haemostatic efficacy of NovoSeven® treatment during routine practice in German clinics.
- Conditions
- Congenital Bleeding Disorder, Congenital FVII Deficiency, Glanzmann's Disease, Acquired Bleeding Disorder
- Trial name
- WIRK (NCT00697320)
- Testing
- eptacog alfa (activated)
- Where
- Germany
- Volunteers
- 64 people
- Sponsor
- Novo Nordisk A/S
Hypofibrinogenemia, Congenital, Afibrinogenemia, Congenital
Human Fibrinogen Concentrate (FGTW) in Pediatric Patients With Congenital Fibrinogen Deficiency
The aim of the study is to evaluate clinical pharmacology, efficacy and safety of FGTW in pediatric patients with congenital fibrinogen deficiency.
- Trial name
- NCT02094430
- Testing
- biological: human fibrinogen concentrate
- Where
- 4 sites in France, Lebanon and 2 more
- Volunteers
- 16 people
- Sponsor
- Laboratoire français de Fractionnement et de Biotechnologies
Hemophilia A
Study of Pharmacokinetics, Efficacy and Safety of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Hemophilia A Patients - A Continuation of Clinical Study 069901
The purpose of this study is to evaluate whether rAHF-PFM is safe and effective in the treatment of hemophilia A subjects. The study consists of 2 parts: Part 1 is a pharmacokinetic and safety study, and Part 2 is an evaluation…
- Trial name
- NCT00157053
- Testing
- Antihemophilic factor, recombinant, manufactured protein-free
- Where
- 24 sites in United States, Austria and 7 more
- Volunteers
- 82 people
- Sponsor
- Baxalta now part of Shire
Hemophilia B
Safety of a New Type of Treatment Called Gene Transfer for the Treatment of Severe Hemophilia B
In this study a modified virus called adeno-associated virus (AAV) will be used to transfer a normal gene for human clotting factor IX into patients with severe hemophilia B (AAV human Factor IX vector).
- Trial name
- NCT00076557
- Testing
- Adeno-Associated Viral with Human Factor IX
- Where
- 3 sites in United States
- Volunteers
- 15 people
- Sponsor
- Avigen
Von Willebrand Disease
Willebrand International Non-interventional Global Surveillance
Collect information about WILLFACT or WILFACTIN in their real life clinical use and identify the therapeutic practices in an international environment.
- Trial name
- NCT01949220
- Where
- 18 sites in Belgium, Czechia and 5 more
- Volunteers
- 80 people
- Sponsor
- Laboratoire français de Fractionnement et de Biotechnologies
Hemophilia A
Study of Pharmacokinetics, Efficacy, and Safety of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Pediatric Patients With Hemophilia A
The purpose of this study is to evaluate whether rAHF-PFM is effective and safe in the treatment of children with hemophilia A. The study consists of 2 parts.
- Trial name
- NCT00157040
- Testing
- Antihemophilic factor, recombinant, manufactured protein-free
- Where
- 12 sites in United States, Canada and 1 more
- Volunteers
- 50 people
- Sponsor
- Baxalta now part of Shire
Factor XIII Deficiency
A Study of the Use of Factor XIII Concentrate in Patients With Inherited FXIII Deficiency
Congenital deficiency of Factor XIII is an extremely rare hereditary disorder associated with potentially life-threatening bleeding.
- Trial name
- NCT00883090
- Testing
- FXIII Concentrate (Human)
- Where
- 6 sites in United States, Spain
- Volunteers
- 15 people
- Sponsor
- CSL Behring
Congenital Fibrinogen Deficiency, Afibrinogenemia
Pharmacokinetic, Efficacy, and Safety Study of Octafibrin Compared to Haemocomplettan/Riastap
The purpose of this study is to investigate pharmacokinetic properties, surrogate efficacy and safety of Octafibrin compared to Haemocomplettan® P/RiaSTAPTM in patients with congenital fibrinogen deficiency
- Trial name
- NCT01575756
- Testing
- Octafibrin, Haemocomplettan® P or RiaSTAPTM
- Where
- 10 sites in United States, Bulgaria and 4 more
- Volunteers
- 22 people
- Sponsor
- Octapharma
Hemophilia A With Inhibitor, Hemophilia B With Inhibitor
A Phase I of SS109 in Hemophilia A or and B With Inhibitors
This phase I study aims to evaluate the safety, PK/PD and immunogenicity of SS109 in hemophilia A or and B with inhibitors. Twenty -seven patients are enrolled in study, and divided into five dose cohorts, from 30μg/kg to…
- Conditions
- Hemophilia A With Inhibitor, Hemophilia B With Inhibitor, Factor VII Deficiency
- Trial name
- NCT05651061
- Testing
- SS109
- Where
- 10 sites in China
- Volunteers
- 27 people
- Sponsor
- Hangzhou Gensciences Biopharmaceutical Co., Ltd.