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

Summary

This study consists of a phase 1 part and a phase 2 part. Phase 1 part: This is a phase 1, open-label, multicenter, dose escalation study to evaluate the safety and tolerability of bosutinib to determine the maximum tolerated dose(MTD) and a recommended phase 2 dose (RP2D) of bosutinib for treatment of ALS patients. Also, efficacy will be evaluated exploratory. Phase 2 part: This is an open label, multicenter, phase 2 part whose purpose is to evaluate the efficacy exploratorily and the long-term (for 24 weeks) safety of bosutinib for the treatment of ALS patients.

Timeline

Start

2019-03-19

Primary completion

2024-03-31

Completion

2024-03-31

Publications

• Imamura K, Izumi Y, Egawa N, Ayaki T, Nagai M, Nishiyama K, Watanabe Y, Murakami T, Hanajima R, Kataoka H, Kiriyama T, Nanaura H, Sugie K, Hirayama T, Kano O, Nakamori M, Maruyama H, Haji S, Fujita K, Atsuta N, Tatebe H, Tokuda T, Takahashi N, Morinaga A, Tabuchi R, Oe M, Kobayashi M, Lobello K, Morita S, Sobue G, Takahashi R, Inoue H. Protocol for a phase 2 study of bosutinib for amyotrophic lateral sclerosis using real-world data: induced pluripotent stem cell-based drug repurposing for amyotrophic lateral sclerosis medicine (iDReAM) study. BMJ Open. 2024 Oct 26;14(10):e082142. doi: 10.1136/bmjopen-2023-082142.
• Imamura K, Izumi Y, Nagai M, Nishiyama K, Watanabe Y, Hanajima R, Egawa N, Ayaki T, Oki R, Fujita K, Uozumi R, Morinaga A, Hirohashi T, Fujii Y, Yamamoto T, Tatebe H, Tokuda T, Takahashi N, Morita S, Takahashi R, Inoue H. Safety and tolerability of bosutinib in patients with amyotrophic lateral sclerosis (iDReAM study): A multicentre, open-label, dose-escalation phase 1 trial. EClinicalMedicine. 2022 Oct 25;53:101707. doi: 10.1016/j.eclinm.2022.101707. eCollection 2022 Nov.

Drugs

Indications