Study Overview
Bring It Up


Research shows that more than half of patients will participate in a clinical trial if their healthcare provider discusses it with them.1 Learn about and discuss RISE UP with your patients with sickle cell disease.
Rise up Clinical Study Design2
PHASE 2
PHASE 3
BID=twice daily.
Participants will be enrolled in either phase 2 or phase 3 of RISE UP.
Once enrolled, participants cannot reenroll in a different phase.2
Key Inclusion Criteria2
- Age 16 years or older, with a documented diagnosis of sickle cell disease (HbSS, HbSC, HbS/β0 thalassemia, HbS/β+ thalassemia, or other sickle cell syndrome variants)
- At least 2 and no more than 10 sickle cell pain crises in the past 12 months prior to informed consent
- Defined as acute episodes of pain, acute chest syndrome, priapism, hepatic or splenic sequestration
- If taking hydroxyurea, the hydroxyurea dose must be stable for at least 90 days before randomization
Key Exclusion Criteria2
- Pregnant or breastfeeding
- Receiving regularly scheduled transfusions
- Hepatobiliary disorders, significant liver disease, gallbladder disease, or severe kidney disease
- Prior exposure to gene therapy or prior bone marrow or stem cell transplantation
- Currently receiving voxelotor, crizanlizumab, or L-glutamine
- Currently receiving treatment with hematopoietic-stimulating agents
- Taking strong CYP3A4/5 inhibitors or strong inducers of CYP3A4
References:
1. Research!America. National public opinion poll. https://www.researchamerica.org/sites/default/files/uploads/clinicaltrialsminorities.pdf. July 2013. Accessed March 3, 2022.
2. Data on file. Agios Pharmaceuticals, Inc.