A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A

Exclusion Criteria

• •Platelet count ≥50,0000 cells/µL
• •Negative HIV test at screening, with the following exception: Individuals with a positive HIV test at screening are eligible provided they are stable on an antiretroviral treatment regimen, have a cluster of differentiation (CD4) count \>200/mm3, and undetectable viral load (\<50 gc/mL)
• •Negative hepatitis B surface antigen (HBsAg) at screening
• •Positive hepatitis surface antibody (HBsAb) at screening, or a negative HBsAb at screening accompanied by either of the following: Negative hepatitis B core antibody (HBcAb); Positive HBcAb and negative hepatitis B virus (HBV) DNA test
• •Negative hepatitis C virus (HCV) antibody test at screening, or positive HCV antibody test at screening accompanied by negative HCV RNA test
• •Otherwise appropriate medical history and physical and laboratory evaluation that are acceptable for inclusion in this clinical trial
• •Are able and willing to comply with scheduled visits, treatment plans, laboratory tests, and other study procedures, including the completion of applicable patient-reported outcome questionnaires
• •Agreement to adhere to the contraception requirements described in the protocol
• •Are currently undergoing antiviral therapy for chronic hepatitis B or chronic hepatitis C
• •Have an inherited or acquired bleeding disorder other than hemophilia A
• •Have known inherited or acquired thrombophilia, have signs of thromboembolic disease in the Investigator's judgement, or are on current treatment for thromboembolic disease. A history of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing is not considered an exclusion criterion
• •Have had prior treatment with a vector or gene transfer agent. Nucleic acid-based vaccines, such as the vaccine for coronavirus disease 2019 (COVID-19), are not considered gene transfer agents
• •Are receiving an investigational drug concurrently or have received an investigational drug within 30 days or 5 half-lives of the last investigational drug administration, whichever is longer
• •Have a major surgical procedure planned in the 15-month period following SPK-8011QQ infusion
• •Are unable (or unwilling) to receive blood or blood products (or any standard-of-care treatment for a life-threatening condition)
• •Have concurrent disease, treatment, or abnormality in clinical laboratory tests that could interfere with the conduct of the study or that would, in the opinion of the Investigator preclude the candidate's safe participation in and completion of the study, or the interpretation of the study results
• •History of malignancy within 5 years prior to screening and up to investigational study drug administration (Day 1) with the following exceptions: Participants with curatively treated basal or squamous cell carcinoma of the skin at any time prior to investigational study drug administration (Day 1) are eligible