The Efficacy and Safety of Rilzabrutinib in Patients Aged 10 to 65 Years With Sickle-cell Disease (LIBRA)

This is a Phase 3, multicenter, randomized, double-blind, placebo-controlled, parallel-group, group-sequential study (Part A), followed by an open-label LTE period (Part B) to investigate the efficacy, and safety of rilzabrutinib in participants with sickle-cell disease (SCD).Study details include:Study duration: a 52-week double-blind period (Part A), followed by an open-label LTE …

Study to Evaluate the Effect of GBT440 on TCD in Pediatrics With Sickle Cell Disease

This study is a Phase 3, randomized, double-blind, placebo-controlled study of voxelotor in pediatric participants, aged 2 to < 15 years old, with Sickle Cell Disease. The primary objective is to evaluate the effect of voxelotor on the TCD (Transcranial Doppler Ultrasound) measurements in SCD participants in this age range.

The TAPS Trial - Fetoscopic Laser Surgery for Twin Anemia Polycythemia Sequence

This multicenter open-label randomized controlled trial is set up to evaluate the effect of fetoscopic laser surgery on the gestational age at birth for monochorionic twin pregnancies diagnosed with twin anemia-polycythemia sequence. Half op the patients will be treated with fetoscopic laser surgery, while the other half will be managed …

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