iPSC-based Drug Repurposing for ALS Medicine (iDReAM) Study

Summary

Participation Requirements

Description

The study consists of a 12-week observation period, a 1-week (acceptable window: 5-9 days) transitional period, a 12-week study treatment period, and a 4-week follow-up period. Subjects who have been receiving riluzole since before the enrollment are allowed to continuously receive riluzole during t...

The study consists of a 12-week observation period, a 1-week (acceptable window: 5-9 days) transitional period, a 12-week study treatment period, and a 4-week follow-up period. Subjects who have been receiving riluzole since before the enrollment are allowed to continuously receive riluzole during the 12-week observation period (with the dosage remaining unchanged), and stop receiving riluzole from the beginning of the 1-week (acceptable window: 5-9 days) transitional period. After the completion of the transitional period, subjects whose total ALSFRS-R score decreased by 1 to 3 points during the 12-week observation period will receive bosutinib for 12 weeks to evaluate the safety and tolerability of bosutinib in ALS patients. All ALS drugs including riluzole will be prohibited during the bosutinib treatment period. In this study, 3 to 6 ALS patients will be enrolled in each of the 4 bosutinib dose lelvels [100 mg/day (dose level 1), 200 mg/day (dose level 2), 300 mg/day (dose level 3), or 400mg/day (dose level 4)] to evaluate the safety and tolerability of the investigational drug (bosutinib) under a 3+3 dose escalation study design. The dose will be escalated by 1 dose level at a time; no skipping will be allowed. Dose escalation and MTD will be determined by the safety assessment committee comprising oncologist, hematologist, ALS Expert based on the incidence of DLT in 4 weeks of treatment among 3 subjects enrolled (6 subjects if additionaly enrolled) in each dose level. RP2D will be determined by the safety assessment committee upon completion of 12-week study treatment in all subjects in all dose levels.

Tracking Information