Dose-Finding Study of SC411 in Children With Sickle Cell Disease

Summary

This is a Phase 2, randomized, double-blind, placebo-controlled, parallel-group, dose-finding study of SC411 in children with sickle cell disease (SCD). The primary objective of the study is to evaluate the safety and tolerability of three different doses of SC411 compared to a placebo. All patients will undergo eight weeks of oral study treatment and a four-week safety follow-up period. Patients will be randomized to one of three dose levels of SC411 or placebo.

Timeline

Start

2017-03-02

Primary completion

2017-09-26

Completion

2022-01-02

Publications

• Results Daak AA, Dampier CD, Fuh B, Kanter J, Alvarez OA, Black LV, McNaull MA, Callaghan MU, George A, Neumayr L, Hilliard LM, Sancilio F, Rabinowicz AL, Heeney MM. Double-blind, randomized, multicenter phase 2 study of SC411 in children with sickle cell disease (SCOT trial). Blood Adv. 2018 Aug 14;2(15):1969-1979. doi: 10.1182/bloodadvances.2018021444.

Drugs

Indications