common.study.topics.clinical

Prophylaxis Replacement Therapy in Adult Hemophilia B

common.study.values.description

Six Month lead-in Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FIX:C≤1%)

To establish a minimum of 6 months of prospective efficacy data of current FIX prophylaxis replacement therapy in the usual care setting of hemophilia B subjects, who are negative for nAb to AAV-Spark100, prior to the Phase 3 gene therapy study. To establish a minimum of 6 months of prospective efficacy data of current FVIII prophylaxis replacement therapy in the usual care setting of hemophilia A subjects, who are negative for nAb to SB-525 capsid (AAV6), prior to the Phase 3 gene therapy study. The enrollment for hemophilia B participants is completed. At this time participants are only being enrolled for hemophilia A cohort.

common.study.values.location

participant.ui.study.affiliations-map.online-study.header-virtual

participant.ui.study.affiliations-map.online-study.text

participant.ui.study.affiliations-map.legend.locations participant.ui.study.affiliations-map.legend.selected

common.study.values.methods

Pharmaceutical medication involved common.study.methods.has-drugs-yes
Recruiting patients only common.study.methods.is-healthy-yes

Drug - Standard of Care FIX Replacement therapy

There is no investigational product being administered. Subjects will be administering their own standard of care FIX replacement therapy.

Drug - Standard of Care FVIII Replacement therapy

There is no investigational product being administered. Subjects will be administering their own standard of care FVIII replacement therapy.

participant.views.study.view.additional

participant.views.study.view.scientific-title

AN OPEN-LABEL, NON-INVESTIGATIONAL PRODUCT, LEAD-IN STUDY TO EVALUATE AT LEAST 6 MONTHS OF PROSPECTIVE EFFICACY AND SAFETY DATA OF FACTOR IX OR FACTOR VIII PROPHYLAXIS REPLACEMENT THERAPY IN THE USUAL CARE SETTING OF MODERATELY SEVERE TO SEVERE ADULT HEMOPHILIA B SUBJECTS (FIX:C≤2%) WHO ARE NEGATIVE FOR nAb TO AAV VECTOR-SPARK100 AND MODERATELY SEVERE TO SEVERE HEMOPHILIA A ADULT SUBJECTS (FVIII:C≤1%) WHO ARE NEGATIVE FOR nAb to AAV VECTOR SB-525 CAPSID (AAV6), PRIOR TO THE RESPECTIVE THERAPEUTIC PH 3 GENE THERAPY STUDIES (See Detailed Description Section for Official Protocol Title)

common.study.values.clinical-trial-id

NCT03587116

participant.views.study.view.id

elYYMa