• Join Today!

Become a member and connect with:

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

Sickle cell patient with severe anemia rapidly improves with voxelotor, case study shows

Global Blood Therapeutics (GBT) reported a case study in which a sickle cell disease (SCD) patient was granted compassionate access to the company’s ongoing Phase 3 HOPE study of voxelotor (NCT03036813). The patient had severe, transfusion-refractory anemia and experienced rapid improvements with the therapy.

The U.S. Food and Drug Administration (FDA) developed the option of compassionate access, also called “compassionate use,” to enable patient enrollment in an “impossible scenario,” i.e. when a patient is ineligible for ongoing trials or no ongoing trials exist. With this measure, patients may be able to receive the investigational therapy when appropriate.


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.