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 range20 Years – 75 Years
SexALL
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
✓. 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
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 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
Phase 2 part: Incidence of abnormal X-ray findings
Timeframe: Up to 24 weeks of treatment with bosutinib