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This study is now recruiting.
This Phase 2 clinical research study is evaluating the safety and effectiveness of a once-daily oral investigational medicine that may improve anemia and reduce the need for red blood cell (RBC) transfusions among some patients who are chronically transfused. All participants enrolled in the study will receive the investigational medication.
We are currently enrolling individuals who meet the following criteria:*
- Are 12 to 65 years of age
- Have one of the following diagnoses:
- SCD and receiving regular RBC transfusions to prevent stroke or recurrence of stroke (Cohort A)
- Thalassemia and receiving regular RBC transfusions (Cohort B)
- Thalassemia and not receiving regular RBC transfusions (Cohort C)
There are additional eligibility requirements, which the investigator will explain to you.
Who Do I Contact?
If you are interested in participating in the study or want to learn more please contact our study team at patelj23 [at] chop.edu or 267-426-5602.
If you qualify and decide to participate, your study participation will last for up to 1 year. You will take 2 tablets once daily for 48 weeks. You will be required to report to CHOP for 14 study visits for blood tests, physical exams, ECG, ECHO, DXA and MRI.
If the study medication improves your symptoms and anemia, you may qualify to participate in an extension of the study, in which you will receive the study drug for an additional year.