A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A
ConditionHemophilia A
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexMale
Age18+
SponsorHoffmann-La Roche
About this trial
This study will assess the safety and tolerability of SPK-8011QQ in adult males with moderately severe to severe hemophilia A.
Eligibility criteria
Qualifiers
Signed Informed Consent Form (ICF)
≥18 years of age at the time of signing the ICF
Male sex assigned at birth
Severe or moderately severe hemophilia A, defined as endogenous FVIII:C activity levels ≤3%, as documented (historically or during the Screening Period) by a certified laboratory and where the FVIII:C level is measured more than 96 hours after the prior dose of an extended half-life FVIII replacement product or more than 72 hours after the prior dose of a standard half-life FVIII replacement product
Disqualifiers
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
Trial design
Treatments tested in this trial
- SPK-8011QQ
Treatment groups
5 Participants
are divided into 1 treatment groupSponsors and collaborators
Source ClinicalTrials.gov