This is a single-arm, open-label, multi-site, single-dose Phase 1/2 study in up to 12 subjects 18 to 35 years of age with severe sickle cell disease (SCD).
7 United States sites, 1 Belgium site, 1 Canada site, 1 Germany site, 1 Italy site
18 to 35 Years
βS/βS
Phase 1/Phase 2
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and efficacy of SHP655 in participants with baseline health sickle cell disease (SCD) and SCD with acute vaso-occlusive crisis (VOC).
18 to 65 Years
SS
Phase 1/Phase 2
This is a non-randomized, open label, multi-site, single dose, Phase 1/2 study in approximately 50 adults and adolescents with severe SCD.
9 United States sites
12 to 50 Years
βS/βS, βS/β0, βS/β+
Phase 1/Phase 2
November 25, 2019
With Oxbryta, sickle cells are less likely to bind together and form the sickle shape, which can cause low hemoglobin levels due to red blood cell destruction. This therapy provides a new treatment option for patients with this serious and life-threatening condition.
November 19, 2019
Doctors are reporting the first evidence that genetically edited cells could offer a safe way to treat sickle cell disease, a devastating, incurable disorder that afflicts millions of people around the world.
November 15, 2019
Today, the U.S. Food and Drug Administration approved Adakveo (crizanlizumab), a treatment to reduce the frequency of vaso-occlusive crisis – a common and painful complication of sickle cell disease that occurs when blood circulation is obstructed by sickled red blood cells – for patients age 16 years and older.
Sending updates featuring new studies and other relevant information about emerging sickle cell treatment options.
