Clinical trials: Factor VIII 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.
Hemophilia A
Evaluation Of Acceptability And Satisfaction Of Hemophilia Patients Treated With FusENGO
This study aims to investigate prospectively throughout a period of 3- 6 months and not to exceed 12 months (according to local therapeutic plans) patients experiences of treatment with Factor VIII in the new device named…
- Trial name
- NCT01959919
- Testing
- Refacto FusENGO
- Where
- 19 sites in Italy
- Volunteers
- 86 people
- Sponsor
- Pfizer
Hemophilia A With Inhibitors, Hemophilia B With Inhibitors
Phase III Study of Coagulation FVIIa (Recombinant) in Congenital Hemophilia A or B Patients With Inhibitors
The purpose of the study is to assess the safety, efficacy and pharmacokinetics of 2 separate dose regimens (75µg/kg and 225 µg/kg) of Coagulation Factor VIIa (Recombinant) for the treatment of bleeding episodes in hemophilia A…
- Trial name
- NCT02020369
- Testing
- Coagulation Factor VIIa (Recombinant)
- Where
- 17 sites in United States, Belarus and 8 more
- Volunteers
- 27 people
- Sponsor
- rEVO Biologics
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.
Hemophilia A
Study of rFVIIIFc for Immune Tolerance Induction (ITI) in Haemophilia A Patients With Inhibitors Who Have Failed Previous ITI Therapies
The primary purpose of this study is to describe the outcome of Immune Tolerance Induction (ITI) treatment performed with rFVIIIFc within a timeframe of 60 weeks in patients with haemophilia A who have failed previous attempts at…
- Trial name
- ReITIrate (NCT03103542)
- Testing
- Recombinant coagulation factor (rFVIIIFc)
- Where
- 12 sites in United States, Canada and 5 more
- Volunteers
- 16 people
- Sponsor
- Swedish Orphan Biovitrum
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, Hemophilia B
An 18-month Low-interventional Study to Assess Joint Health in Haemophilia A and B Patients on Prophylaxis With Efmoroctocog Alfa or Eftrenonacog Alfa
The goal of this low-interventional study is to describe the overall joint health in patients with haemophilia A or haemophilia B prophylactically treated with rFVIIIFc or rFIXFc.
- Trial name
- JOIN-us (NCT05856266)
- Testing
- Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US), Haemophilia Joint Health Score (HJHS)
- Where
- 56 sites in Bulgaria, Croatia and 9 more
- Volunteers
- 10 people
- Sponsor
- Swedish Orphan Biovitrum
Congenital Bleeding Disorder, Haemophilia A With Inhibitors
Pharmacogenetic Testing of Saliva Samples From Patients With Five or More Exposure Days to rFVIIa Analogue in the Adept™2 Trial
This study is conducted globally. This study describes pharmacogenetic testing of saliva samples from patients who participated in the NN1731-3562 trial (adept™2) (NCT01392547).
- Conditions
- Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
- Trial name
- NCT02541942
- Testing
- No treatment given
- Where
- 15 sites in United States, Greece and 5 more
- Volunteers
- 19 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A
BAX 802 in CHA With Inhibitors
The purpose of this study is to evaluate the efficacy and safety of BAX 802 in males with congenital hemophilia A (CHA) with inhibitors who are undergoing major or minor elective surgical, dental, or other invasive procedures.
- Trial name
- CHAWI (NCT02895945)
- Testing
- Antihemophilic Factor (Recombinant), Porcine Sequence (BAX 802)
- Where
- 24 sites in United States, Bulgaria and 10 more
- Volunteers
- 8 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A
Non-interventional Post-authorisation Study to Document the Immunogenicity, Safety, and Efficacy of NUWIQ
Prospective, multinational, non-interventional post-authorisation study to collect additional clinical data and to ensure consistency in the long-term between the outcome from pre-authorisation clinical studies (in 135 previously…
- Trial name
- NCT02962765
- Where
- 39 sites in United States, Argentina and 11 more
- Volunteers
- 80 people
- Sponsor
- Octapharma
Severe Hemophilia A
Study to Investigate the Long-term Efficacy and Safety of Human-cl rhFVIII in Previously Treated Patients (PTPs)
The purpose of the study is to study the long-term efficacy, safety and tolerability of Human-cl rhFVIII in previously treated patients with severe hemophilia A.
- Trial name
- NCT01341912
- Testing
- Human-cl rhFVIII
- Where
- 2 sites in United States, Bulgaria
- Volunteers
- 3 people
- Sponsor
- Octapharma
Severe Haemophilia A
Gene Therapy Study in Severe Haemophilia A Patients (270-201)
This study is being conducted by BioMarin Pharmaceutical Inc. as an open label, dose escalation study in order to determine the safety and efficacy of valoctocogene roxaparvovec (an Adenovirus-Associated Virus based gene therapy…
- Trial name
- NCT02576795
- Testing
- valoctocogene roxaparvovec
- Where
- 5 sites in United Kingdom
- Volunteers
- 15 people
- Sponsor
- BioMarin Pharmaceutical
Congenital Bleeding Disorder, Haemophilia A
Haemophilia Patients With Inhibitors Being Treated for Acute Joint Bleeds
This trial is conducted in Africa, Asia, Europe, Japan, and North and South America. The aim of this trial is to evaluate the safety and efficacy of activated recombinant human factor VII analogue (vatreptocog alfa (activated))…
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia B
- Trial name
- NCT00486278
- Testing
- eptacog alfa (activated), vatreptacog alfa (activated)
- Where
- 45 sites in United States, Argentina and 16 more
- Volunteers
- 51 people
- Sponsor
- Novo Nordisk A/S
Congenital Bleeding Disorder, Haemophilia A
Safety and Efficacy of Turoctocog Alfa in Haemophilia A Subjects
This trial is conducted in Asia, Europe, and North and South America. The trial consists of a main trial and a sub-trial. The main trial investigates safety and efficacy of turoctocog alfa (recombinant factor VIII, rFVIII (N8))…
- Trial name
- guardian™1 (NCT00840086)
- Testing
- turoctocog alfa
- Where
- 63 sites in United States, Brazil and 16 more
- Volunteers
- 150 people
- Sponsor
- Novo Nordisk A/S
Haemophilia A
BAY81-8973 Pediatric Safety and Efficacy Trial
The primary objective was to evaluate the safety and efficacy of the treatment with BAY81-8973 for prophylaxis and treatment of breakthrough bleeds in children with severe hemophilia A.
- Trial name
- NCT01311648
- Testing
- Recombinant Factor VIII (Kovaltry, BAY81-8973)
- Where
- 43 sites in United States, Argentina and 15 more
- Volunteers
- 94 people
- Sponsor
- Bayer
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
Mild Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor
A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors
This is a multicenter, open-label, single-arm study designed to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of emicizumab in participants with mild or moderate hemophilia A without inhibitors against…
- Conditions
- Mild Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor, Hemophilia A
- Trial name
- HAVEN 6 (NCT04158648)
- Testing
- Emicizumab
- Where
- 22 sites in United States, Belgium and 8 more
- Volunteers
- 73 people
- Sponsor
- Hoffmann-La Roche