A Study to Learn About the Efficacy and Safety of Preventive Routine Replacement Treatment with Blood Clotting Factor IX (9) in Men with Hemophilia B
An Open Label, Non-Investigational Product, Multi-Center, Lead-in Study to Evaluate Prospective Efficacy and Selected Safety Data of Current Factor IX (FIX) or Factor VIII (FVIII) Prophylaxis Replacement Therapy in the Usual Care Setting of Moderately Severe to Severe Adult Hemophilia B Participants (FIX:C≤2%) Who are Negative for Neutralizing Antibodies to Adeno-Associated Virus Vector Spark100 (Benegene-1) and Moderately Severe to Severe Hemophilia A Adult Participants (FVIII:C≤1%) Who are Negative for Neutralizing Antibodies to Adeno-Associated Virus Vector 6 (AAV6), Prior to the Respective Therapeutic Phase 3 Gene Therapy Studies
Category & Conditions: Rare Diseases
Medicine: fidanacogene elaparvovec (PF-06838435)
ClinicalTrials.gov Identifier (NCT): NCT03587116
Protocol ID: C0371004
Open Plain Language Summary Result:
Click here