BEYOND-9

Conditions de participation

• Sexe:

  Male

• Âges admissibles:

  18 to 90

Critères de participation

Inclusion Criteria:
A participant must meet the following criteria to be eligible for inclusion in the study:
1. Male gender at birth
2. Confirmed diagnosis of severe or moderately severe hemophilia B.
3. Currently taking Factor IX(9) (FIX) prophylaxis and previous experience with FIX therapy: ≥150 documented exposure days to FIX.
4. Provide informed consent and willing and able to comply with clinic visits & study-related procedures.
5. Participation in the lead-in period of this interventional study.
Exclusion Criteria:
1. History of FIX inhibitor (clinical or laboratory-based assessment) on 2 or more occasions:
2. Detectable pre-existing antibodies to the Adeno Associated Virus 8.
3. History of allergy to corticosteroids
4. Known medical condition that requires chronic administration of corticosteroids
5. Unable or unwilling to take the required pretreatment or treatment for potential immune-mediated liver enzyme elevation.
6. Lack of adherence to documenting bleeds &/or prophylaxis replacement therapy administration.
7. Is positive for hepatitis B surface antigen (HbsAg) or hepatitis C virus (HCV) by HCV RNA test at screening visit.
8. History of clinically significant heart, lung, liver, kidney, gastrointestinal, endocrine, blood, psychiatric, neurological disease or bleeding or blood clotting disorder, that may confound the results of the study or poses an additional risk to the participant.
9. Received any live viral vaccinations in the 3 months prior to infusion study drug dosing.
10. Sexually active men who are unwilling to use barrier contraception, for example consistent
use of a condom, sperm donation or blood, organ, tissue, or cell donation is prohibited for 12 months following administration of study drug.