Clinical trials: Congenital Factor VIII (FVIII) 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 VIII Deficiency, Congenital, Factor 8 Deficiency, Congenital
Post-Marketing Surveillance To Observe Safety And Efficacy Of Xyntha Solofuse Prefilled Syringe
This study aims to observe the safety and efficacy of the Xyntha Solofuse prefilled syringe in the setting of routine practice. The primary objective is to detect medically significant events (factor VIII inhibitor).
- Conditions
- Factor VIII Deficiency, Congenital, Factor 8 Deficiency, Congenital, Autosomal Hemophilia A, Classic Hemophilia
- Trial name
- NCT03034044
- Volunteers
- 106 people
- Sponsor
- Pfizer
Factor VII Deficiency, Glanzmann Thrombasthenia
Study of Coagulation Factor VIIa Marzeptacog Alfa (Activated) in Subjects With Inherited Bleeding Disorders
The purpose of the trial is to evaluate the PK, bioavailability, PD, efficacy and safety of MarzAA for on demand treatment and control of bleeding episodes in adult subjects with inherited bleeding disorders.
- Conditions
- Factor VII Deficiency, Glanzmann Thrombasthenia, Hemophilia A With Inhibitor
- Trial name
- NCT04548791
- Testing
- Coagulation Factor VIIa variant
- Where
- 20 sites in United States, India and 3 more
- Volunteers
- 19 people
- Sponsor
- Catalyst Biosciences
Hemophilia A, Hemophilia B
An Open-label, Multicenter Phase I/II Clinical Trial to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetic/Pharmacodynamic (PK/PD) Characteristics of SR604 Injection in Patients With Hemophilia A/B and Congenital Factor VII Deficiency
The purpose of this study is to evaluate the safety, tolerability, immunogenicity , PK, and PD of a single dose of SR604 in participants with Hemophilia A or Hemophilia B, with or without inhibitors （Part A）and to evaluate the…
- Conditions
- Hemophilia A, Hemophilia B, Factor VII Deficiency
- Trial name
- NCT07644832
- Testing
- SR604
- Where
- 9 sites in China
- Volunteers
- 76 people
- Sponsor
- Shanghai RAAS Blood Products Co., Ltd.
Hemophilia A
Long-term Study Evaluating Joint Health in People With Haemophilia A Receiving Real-world Prophylactic Treatment With Efanesoctocog Alfa
The rationale for this study is to further understand and describe the long-term prophylactic effectiveness of efanesoctocog alfa in preventing joint bleeds in a real-life setting.
- Trial name
- NCT06940830
- Testing
- Efanesoctocog alfa
- Where
- 31 sites in Croatia, Czechia and 6 more
- Volunteers
- 252 people
- Sponsor
- Swedish Orphan Biovitrum
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
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.