Phase 1/2 Study of Bosutinib in Patients with Amyotrophic Lateral Sclerosis (ALS)

JRCT ID: jRCT2051220002

Registered date:08/04/2022

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: Amyotrophic Lateral Sclerosis (ALS)
• Date of first enrollment: 22/05/2022
• Target sample size: 25
• Study type: Interventional
• Intervention(s): Patients will receive bosutinib once daily, orally, for 24 weeks

Outcome(s)

Primary Outcome

Change from baseline (Visit 5: before the start of study drug administration) in ALSFRS-R at week 24 will be compared with the external published data of placebo group in edaravone study (MCI186-19)
Safety during 24 weeks of treatment

Key inclusion & exclusion criteria

• Age minimum: >= 20age old
• Age maximum: <= 75age old
• Gender: Both

Include criteria

1. ALS patients who are categorized as either Definite ALS or Probable ALS in the El Escorial and revised Airlie House criteria for the diagnosis of ALS
2. 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
3. Patients with ALS within 2 years of symptom onset at the time of the first registration
4. Patients with change in total ALSFRS-R score during the observation period from -1 to -4 points
5. Patients with score of at least 2 on all items of ALSFRS-R; 4.Writing, 5.Feeding behavior 1 must have at least 2 points on each side.

Exclude criteria

1. Patients with tracheostomy
2. Patients who had decreased respiratory function and complained of dyspnea at the time of enrollment. One of the three items on the ALSFRS-R related to respiratory dyspnea, orthopnea, or respiratory failure is less than 3 points.
3. Patients whose percent FVCs are at least 80 percent at the time of first and second registrations
4. Patients who have nerve conduction study findings of demyelination such as conduction block

Related Information

• Primary Sponsor: Inoue Haruhisa
• Source(s) of Monetary Support: Japan Agency for Medical Research and Development