Here are the studies that match your search criteria. If you are interested in participating, please reach out to the contact listed for the study. If no contact is listed, contact us and we'll help you find the right person.
1
Study Matches
Gene-transfer, open-label, dose-escalation study of SPK-8011 [recombinant adeno-associated viral vector with B-domain deleted human factor VIII gene] in individuals with hemophilia A
Gene-transfer, open-label, dose-escalation study of SPK-8011 in individuals with hemophilia A. Primary Objective to evaluate the safety, tolerability and efficacy of SPK-8011 a recombinant adeno-associated viral vector with B-domain deleted human factor VIII gene.
Hemophilia A FVIII Level <1% or 1-2% with> 10 bleeds past 52 weeks or 1-2% on prophy treatment Greater than 150 exposure days to Factor VIII concentrate or cryo No evidence of Factor VIII Inhibitor Acceptable lab values as defined by protocol
Exclusion Criteria:
Active Hep B or C &/or current antiviral therapy for Hep B or C Significant underlying liver disease, cancer, GI disease, latent infectious disease HIV-1 or HIV-2 with CD4 count </=l to 200 Other investigational product in the past 12 weeks or gene therapy research within the past 52 weeks Any other significant condition/disease that in the opinion of the investigator would make the subject unsuuitable