Safety Study of SPK-8011QQ Gene Therapy in Adult Males with Severe or Moderately Severe Hemophilia A

What this Trial Is About

Researchers are evaluating the safety and tolerability of SPK-8011QQ, a gene therapy, in adult males with moderately severe to severe hemophilia A. This study is a Phase 2b, single-arm, open-label trial focusing on individuals with low factor VIII activity levels who have a history of treatment with factor VIII products. The purpose is to carefully monitor adverse events and treatment effects over an extended period. Participants will receive a single intravenous infusion of SPK-8011QQ on the first day of the study. The study is designed as a single group treatment with no placebo comparator. After the infusion, participants will be followed for up to approximately five years to assess safety and tolerability outcomes, including adverse events and laboratory abnormalities. Throughout the study, participants will attend scheduled visits where safety assessments will be conducted. These include monitoring for adverse events categorized by severity and type, laboratory tests, and antibody screenings. The primary outcomes focus on the frequency and severity of adverse events related to the treatment. The study duration may extend up to five years, allowing for long-term safety monitoring in participants.

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