Early stage clinical trials for the treatment of hemophilia A

Gianna M. Guzzardo, Robert Sidonio,, Michael U. Callaghan, Katherine Regling

Research output: Contribution to journalReview articlepeer-review

1 Scopus citations


Introduction: Hemophilia A is a severe bleeding disorder affecting about 1 in 5,000 males. The gold standard for prophylaxis and treatment of acute bleeding has been factor (F) VIII concentrate. A multitude of treatment modalities are now available and under clinical investigation. Areas covered: This review discusses ongoing/recently completed early-phase clinical trials registered on ClinicalTrials.gov in patients with hemophilia A through April 2022. These new pipeline therapies are focused on addressing the safety and efficacy of new factor-related products, non-factor related products, and gene therapy options for hemophilia. Expert opinion: Current standard of care effectively prevents and treats acute bleeding and has significantly improved the quality of life in hemophilia. The biggest challenges in the improvement of care are treatment-related burden and the burden of cost in developing countries. New drugs under development are likely to enter practice by the end of this decade and address many of the unmet needs particularly of those with severe disease. Data is limited in unique populations (e.g. congenital/inherited FVIII inhibitors, non-severe hemophilia A, women/girls with hemophilia and children) which are important areas for future research; additional clinical trials and long-term outcome data are necessary prior to incorporating these new therapies in our treatment arsenal.

Original languageEnglish
Pages (from-to)1169-1186
Number of pages18
JournalExpert Opinion on Investigational Drugs
Issue number11
StatePublished - 2022


  • Hemophilia A
  • early clinical trials
  • gene therapy
  • hemophilia treatment
  • phase 1 clinical trials
  • phase 2 clinical trials


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