Beta-Thalassemia Clinical Trial
Official title:
A Phase II, Single Arm, Multicenter, Proof-of-Mechanism Study to Investigate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Bitopertin (RO4917838) in Adults With Non-Transfusion-Dependent Βeta-Thalassemia
This proof-of-mechanism study is being performed to investigate the safety, tolerability,
efficacy, pharmacokinetics, and pharmacodynamics of multiple oral doses of bitopertin in
adults with NTD beta-thalassemia.
This study consists of two parts:
Part 1 - The main study - 16 weeks in total: Participants will undergo a 6-week
dose-escalation period followed by 10 weeks of treatment at the attained target dose.
Part 2 - Open Label Extension (OLE) - up to an additional 12 months. Participants will be
given the option to enroll into the OLE once the 16-week treatment of Part 1 has been
completed. Participants who decide not to enroll in the OLE, at the end of Part 1 will enter
a 6-week follow-up period.
n/a
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