Safety of EPI-589 re-administration for ALS

JRCT ID: jRCTs051230064

Registered date:06/07/2023

Basic Information

• Recruitment status: Complete
• Health condition(s) or Problem(s) studied: Amyotrophic lateral sclerosis
• Date of first enrollment: 09/08/2023
• Target sample size: 3
• Study type: Interventional
• Intervention(s): Administer EPI-589 500 mg orally three times daily before meals. The administration period will be from the day after registration of the treatment period to the end of all examinations and observations specified at the 22nd week of the treatment period.

Outcome(s)

• Primary Outcome: Adverse events

Secondary Outcome

Amount and rate of change in ALSFRS-R score
Period from start of treatment period to occurrence of event (all-day use of noninvasive respiratory support device, use of invasive respiratory support device, or death)
Amount of change in %SVC
Clinical examination
Vital signs
Body weight

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

1) Patients who have given written informed consent to participate in this clinical study (or patients for whom a signature was obtained by a proxy, in case patients who have the ability to consent but are unable to sign the consent form in their own handwriting due to physical disabilities).
2) Patients who have entered the investigator-initiated clinical trial (DA350103) and have received EPI-589 for about 24 weeks, and have had no concerns with safety or tolerability.
3) Patients who can go to outpatient clinic

Exclude criteria

1) Patients who have clinically significant complications or medical history other than ALS, and who are judged to be inappropriate for clinical study by the investigator.
2) Patients with psychiatric disorders, cognitive impairment, or Parkinson's disease.
3) Patients undergoing tracheostomy.
4) Patients wearing a non-invasive respiratory support device all day (wearing for 22 hours or more).
5) Patients with a history of or complication of drug allergy or severe allergic disease (anaphylactic shock, etc.).
6) Patients who have a malignant tumor or have a medical history within 5 years before obtaining informed consent.
7) Patients whose AST and ALT levels were 3 times or more higher than the upper limit of normal at the time of screening.
8) Patients with CK =>2.5 times the upper limit of normal at screening.
9) Patients with estimated glomerular filtration rate (eGFR) less than 45 mL/min/1.73m2 at screening.
10) Pregnant women, lactating women, or patients who may be pregnant.
11) Patients or their partners wish to become pregnant, or patients who cannot agree to practice contraception during the period from the time of obtaining informed consent to 30 days after the last dose of the study drug.
12) Patients who participated in a clinical trial other than the investigator-initiated trial (DA350103) before consent was obtained, and 30 days had not passed since the administration of the test drug in the same clinical trial at the time of consent (half-life of the test drug in the same clinical trial If the number of days multiplied by 5 is longer than 30 days, the number of days 5 times the half-life must have passed), or patients participating or plan to participate in a clinical trial other than the investigator-initiated trial (DA350103) at the time of obtaining consent.
13) Patients who received cell therapy or gene therapy before obtaining informed consent.
14) Patients who are judged inappropriate for participation in this clinical study by the investigator.

Related Information

• Primary Sponsor: Nagano Seiichi