L-citrulline Injection in Patients Aged 6-21 Years Old With Sickle Cell Disease Presenting With Vaso-Occlusive Crisis (VOC)

• Status: Recruiting
• Trial end date: 2022-07-01

Summary

The applicant is developing intravenous L-citrulline (Turnobi™) for treatment of sickle cell disease (SCD). The current development program targets treatment of sickle cell-associated vaso-occlusive crisis (VOC) specifically. The aim of Part 1 is to identify the optimum dose regimens for the Part 2 of the trial which is a double-blind, placebo controlled adaptive 'pick-the-winner' design. This study will allow assignment of more subjects to the better treatment arm/s based on emerging data. The study, initially, will evaluate efficacy and tolerability of incremental doses of intravenous (IV) L-citrulline (Turnobi™) in patients with SCD while receiving standard of care therapy for VOC.

• Phase: Phase 1/Phase 2

Details

• Lead Sponsor: Asklepion Pharmaceuticals, LLC