Clinical trials: Hemophilia
Hemophilia A
Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A
This Phase III clinical study will evaluate the safety and effectiveness of valoctocogene roxaparvovec in combination with prophylactic corticosteroids in patients with severe hemophilia A.
- Trial name
- GENEr8-3 (NCT04323098)
- Testing
- valoctocogene roxaparvovec
- Where
- 12 sites in United States, Australia and 2 more
- Volunteers
- 22 people
- Sponsor
- BioMarin Pharmaceutical
Haemophilia A, Haemophilia A With Inhibitors
A Research Study Looking at Long-term Treatment With Mim8 in People With Haemophilia A
This study is looking at how Mim8 works in people with haemophilia A, who either have inhibitors or do not have inhibitors. Mim8 is a new medicine that will be used to avoid bleeding episodes.
- Trial name
- FRONTIER4 (NCT05685238)
- Testing
- Mim8
- Where
- 141 sites in United States, Austria and 30 more
- Volunteers
- 451 people
- Sponsor
- Novo Nordisk A/S
Severe Hemophilia A, Moderate Hemophilia A
A Study to Evaluate Overall Health, Physical Activity, and Joint Outcomes in Participants With Severe or Moderate Hemophilia A Without Factor VIII Inhibitors on Emicizumab Prophylaxis
Study MO42623 is a Phase IV, multicenter, open-label, three cohort study designed to evaluate the impact of emicizumab prophylaxis on overall health, physical activity, and joint outcomes in participants aged ≥13 and <70 years…
- Trial name
- Beyond ABR (NCT05181618)
- Testing
- Emicizumab
- Where
- 26 sites in United States, Brazil and 10 more
- Volunteers
- 136 people
- Sponsor
- Hoffmann-La Roche
Hemophilia A With Inhibitor
Low-dose ITI Strategy for Children in Hemophilia A With High-titer Inhibitor and Poor ITI Risk in China
The study start on January 18, 2017. The Severe(FⅧ<1%) and moderate hemophilia A (FⅧ1%～5%)children with high titer inhibitor(historical peak inhibitor titer≥5BU ) combining with poor ITI risk(s) were enrolled.
- Trial name
- HA-LD-ITI (NCT03598725)
- Testing
- Coagulation Factor VIII, Prednisone, Rituximab
- Where
- China
- Volunteers
- 55 people
- Sponsor
- Beijing Children's Hospital