Clinical trials: Coagulation Factor Deficiency
Hemophilia A
A Gene Transfer Study for Hemophilia A
This clinical research study is being conducted by Spark Therapeutics, Inc. to determine the safety and efficacy of the factor VIII gene transfer treatment with SPK-8011 in individuals with hemophilia A.
- Trial name
- NCT03003533
- Testing
- SPK-8011
- Where
- 16 sites in United States, Australia and 3 more
- Volunteers
- 25 people
- Sponsor
- Spark Therapeutics, Inc.
Factor VII Deficiency
A Phase II Exploratory Study of SR604 Injection Evaluating the Safety and Efficacy in the Treatment of Congenital Coagulation Factor VII Deficiency
This is an open-label, multicenter, exploratory Phase II clinical trial designed to evaluate the efficacy, safety, and pharmacokinetic characteristics of SR604 Injection in patients with congenital coagulation Factor VII…
- Trial name
- NCT07711158
- Testing
- SR604
- Where
- China
- Volunteers
- 12 people
- Sponsor
- Shanghai RAAS Blood Products Co., Ltd.
Congenital Coagulation Factor VII Deficiency
Study of the Pharmacokinetics and Safety of Recombinant Factor VIIa Fusion Protein (rVIIa-FP, CSL689) in Patients With Congenital Factor VII Deficiency
The purpose of this study is to investigate the pharmacokinetics (PK) and safety of rVIIa-FP (CSL689) in a total of 10 to 16 male or female adults with inherited coagulation factor VII (FVII) deficiency.
- Trial name
- NCT02470871
- Testing
- Eptacog alfa (activated) or pdFVII, CSL689
- Where
- 2 sites in Netherlands, Norway
- Volunteers
- 9 people
- Sponsor
- CSL Behring
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 B
A Gene Therapy Study for Hemophilia B
A Phase 1/2, Open-Label, Non-Randomized, Dose-Escalation Study of SPK-9001 in Subjects with Hemophilia B.
- Trial name
- NCT02484092
- Testing
- SPK-9001
- Where
- 10 sites in United States, Australia
- Volunteers
- 15 people
- Sponsor
- Pfizer
Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited
Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants
The aim of this prospective, observational study is to establish a dataset on the frequency of bleeding events, as well as other characteristics of bleeding events and FVIII infusions, in patients with clinically severe…
- Conditions
- Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, Hemophilia A
- Trial name
- NCT03876301
- Testing
- Standard of Care FVIII Replacement therapy
- Where
- 11 sites in United States, Australia and 2 more
- Volunteers
- 25 people
- Sponsor
- Spark Therapeutics, Inc.
Factor XIII Deficiency
An Open Enrollment Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
Congenital deficiency of factor XIII 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
- NCT00945906
- Testing
- FXIII Concentrate (Human) (FXIII)
- Where
- 25 sites in United States
- Volunteers
- 61 people
- Sponsor
- CSL Behring
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
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 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.
HIV Infections, Hemophilia A
Effect of Indinavir Plus Two Other Anti-HIV Drugs on Blood Clotting in HIV-Positive Males With Hemophilia
The purpose of this study is to see if indinavir plus two other anti-HIV drugs affect blood clotting in HIV-positive patients with hemophilia.
- Trial name
- NCT00002386
- Testing
- Indinavir sulfate, Lamivudine, Stavudine
- Where
- 8 sites in United States, Canada
- Volunteers
- 55 people
- Sponsor
- Merck Sharp & Dohme LLC
Hemophilia A, Congenital Factor VIII (FVIII) Deficiency
ADVATE 2 mL Post-Authorization Safety Surveillance (PASS)
This is a Post-Authorization Safety Surveillance (PASS) study designed to collect data on the safety and effectiveness of ADVATE reconstituted in 2 mL Sterile water for injection (SWFI) during routine clinical practice in…
- Trial name
- NCT02093741
- Testing
- Octocog alfa (recombinant human coagulation factor VIII) [ADVATE]
- Where
- 17 sites in France, Germany and 2 more
- Volunteers
- 65 people
- Sponsor
- Baxalta now part of Shire
Fibrinogen Deficiency
Human Fibrinogen - Pharmacokinetics
This study evaluated the single-dose pharmacokinetics of human fibrinogen concentrate and clot strength (maximum clot firmness [MCF]) in subjects with congenital fibrinogen deficiency.
- Trial name
- NCT00496262
- Testing
- Human Fibrinogen Concentrate
- Where
- 15 sites in United States, Italy
- Volunteers
- 15 people
- Sponsor
- CSL Behring
Hemophilia A
A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A in Taiwan
This is a national, multicenter, retrospective/prospective, observational study in Taiwan designed to assess effectiveness, safety, and usage of efanesoctocog alfa prophylaxis treatment in hemophilia A participants.
- Trial name
- PROTECT-ALT (NCT06684314)
- Testing
- Efanesoctocog alfa
- Where
- 10 sites in Taiwan
- Volunteers
- 79 people
- Sponsor
- Sanofi
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