• Join Today!

Become a member and connect with:

  • An Active Online Community
  • Articles and Advice on SCD
  • Help Understanding Clinical Trials

Safety and early hints of benefit seen in phase 1b trial of PF-04447943

Sickle cell disease (SCD) patients were safely treated with a phosphodiesterase 9A (PDE9) inhibitor therapy called PF-04447943 with no serious side effects reported and possible evidence of fewer vaso-occlusion pain crises, a Phase 1b clinical trial reports.

Safety and early signs of possible efficacy — including a drop in biomarkers linked to vaso-occlusion — were seen regardless of whether patients given PF-0444794 3 were also taking hydroxyurea.

The study, “PF-04447943, a phosphodiesterase 9A Inhibitor, in Stable Sickle Cell Disease Patients: A Phase Ib Randomized, Placebo-Controlled Study” was published in the journal, Clinical and Translation Science.

Sickle cell disease, caused by an inherited alteration in a gene that instructs the making of the beta-globin subunit of hemoglobin, the protein responsible for carrying oxygen in the blood, leads to sickle hemoglobin production and sickle-shaped red blood cells.



To improve your experience on this site, we use cookies. This includes cookies essential for the basic functioning of our website, cookies for analytics purposes, and cookies enabling us to personalize site content. By clicking on 'Accept' or any content on this site, you agree that cookies can be placed. You may adjust your browser's cookie settings to suit your preferences. More information

The cookie settings on this website are set to "allow cookies" to give you the best browsing experience possible. If you continue to use this website without changing your cookie settings or you click "Accept" below then you are consenting to this.