iPSC-based Drug Repurposing for ALS Medicine (iDReAM) Study (NCT04744532) | Clinical Trial Compass
UnknownPhase 1/2
iPSC-based Drug Repurposing for ALS Medicine (iDReAM) Study
Japan49 participantsStarted 2019-03-19
Plain-language 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.
Who can participate
Age range
20 Years – 75 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Evidence of a personally signed and dated informed consent document indicating that the patient has been informed of all pertinent aspects of the study. To be additionaly signed by a delegate signer if the subject is unable to handwrite.
. Patients aged ≥20 years and \<80 years at the time of informed consent
. Patients with positive already-reported SOD1 gene mutation and progressive muscle weakness; sporadic ALS patients who are categorized as either "Definite ALS" or "Probable ALS" or "Probable-laboratory supported ALS" in the Updated Awaji Criteria for the diagnosis of ALS
. Patients at Grade 1 or 2 in the Japan ALS Severity Scale of the grant-in-aid program for chronic diseases from the Japanese Ministry of Health, Labour and Welfare; patients with positive SOD1 mutation of Grade 1, 2 or 3
. Patients with ALS that occurred within 2 years at the time of the first registration; patients with positive SOD1 mutation within 5 years after disease onset
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Phase 1 part: Dose-limiting toxicity (DLT)
Timeframe: During the first 4 weeks of treatment with bosutinib
2
Phase 1 part: Dose-limiting toxicity (DLT)
Timeframe: Up to 12 weeks of treatment with bosutinib
3
Phase 2 part: Change in ALSFRS-R from baseline to week 24 in each 200mg and 300mg group compared with the external published data of placebo group
Timeframe: Up to 24 weeks of treatment with bosutinib
4
Phase 2 part: Adverse events
Timeframe: Up to 24 weeks of treatment with bosutinib
5
Phase 2 part: Incidence of abnormal laboratory test results
Timeframe: Up to 24 weeks of treatment with bosutinib
6
Phase 2 part: Incidence of abnormal vital signs
Timeframe: Up to 24 weeks of treatment with bosutinib
7
Phase 2 part: Incidence of abnormal ECG recordings
Timeframe: Up to 24 weeks of treatment with bosutinib
. Patients who can visit hospital regularly as outpatients
. Patients with change in total ALSFRS-R score during the observation period are -1 to -3 points
. Urine pregnancy test (for females of childbearing potential) negative at screening
Exclusion criteria
. Patients with tracheostomy
. Patients who have used non-invasive ventilation due to ALS symptoms
. Patients whose %FVCs are less than 70% at the time of first and second registrations
. Patients who have nerve conduction study findings of demyelination such as conduction block
. Patients who are taking edaravone; patients who started riluzole or edaravone after start of the observation period; patients who changed the dosage of riluzole after start of the observation period
. Patients with bulbar type ALS with dysphagia and dysarthria
. Patients with cognitive impairment
. Pregnant female patients; breastfeeding female patients; fertile male and female patients of childbearing potential who are unwilling or unable to use 1 highly effective methods of contraception as outlined in this protocol for the duration of the study and for at least 28 days after the last dose of investigational product
8
Phase 2 part: Incidence of abnormal X-ray findings
Timeframe: Up to 24 weeks of treatment with bosutinib