NIPH Clinical Trials Search

JAPANESE
国立保健医療科学院
JRCT ID: jRCT2061210031

Registered date:27/08/2021

EPI-589 Early Phase 2 Investigator-initiated Clinical trial for ALS

Basic Information

Recruitment status Recruiting
Health condition(s) or Problem(s) studiedAmyotrophic Lateral Sclerosis
Date of first enrollment15/09/2021
Target sample size10
Countries of recruitmentJapan
Study typeInterventional
Intervention(s)EPI-589 500 mg (2 tablets of EPI-589 250 mg) is orally administered three times a day before meals (at least one hour before breakfast, lunch, and dinner). The treatment period will be from the day after the registration of the treatment period to the end of the examination at 24 weeks of the treatment period.

Outcome(s)

Primary Outcome1) Efficacy the change and change ratio of ALSFRS-R; duration between the start date of treatment and events (intiation of whole-day NPPV, inititation of invasive positive pressure ventilation, or death); the change in %SVC; the change in MMT total score; the change in grip strength (left, right); the change in modified Norris scale total score; the change in the Amyotrophic Lateral Sclerosis Assessment Questionaire-40 (ALSAQ-40) total score 2) Safety Adverse events; laboratory test; vital sign; 12-lead ECG; C-SSRS 3) Biomarkers the change in CSF biomarkers (3-NT, 8-OHdG, ornithine, pNFH, and NfL); the change in plasma biomarkers (3-NT, 8-OHdG, pNFH, NfL, creatinine and homocysteine); the change in MRI mesurements (NAA measured by 1H-MRS and adjusted by creatine, choline, or creatine+choline, Fractional anisotropy by Diffusion Tensor Imaging, Apparent diffusion coefficient, Whole brain 3D T1WI).
Secondary Outcome

Key inclusion & exclusion criteria

Age minimum>= 18age old
Age maximum<= 79age old
GenderBoth
Include criteria(1) subjects providing written informed consent (Subject and representative providing written informed conset are needed, if the subject is under 20 years old.) (2) aged between 18 and 79 years at the time of informed consent (3) meet the revised El Escorial criteria for clinically definite, clinically probable laboratory-supported or clinically probable (4) within 18 months from symptom onset at screening (5) ALSFRS-R total score progression of <-0.3 (>0.3 decline)/month between the onset and screening (If ALSFRS-R at the time of symptom onset is not determined, please set as 48 points) *progression rate is calculated as: (ALSFRS-R at the time of screening - ALSFRS-R at the time of onset)/duration from onset to screening (month). (6) scores of >=2 points on all 12 items of ALSFRS-R (7) scores of 4 points on three items of respiratory subscore of ALSFRS-R (8) scores of >=3 points in ALSFRS-R swallowing item (9) slow vital capacity (SVC) >= 80% (10) on a stable dose of riluzole for at least 30 day prior to registration for pre-observation period (11) ambulant (12) agreement for genetic research
Exclude criteria(1) with a complication or history of significant other medical conditions based on the investigator's judgement (2) complications of psychological disease, cognitive impairment, or Parkinson's disease (3) underwent tracheostomy (4) with experience of using invasive or noninvasive positive pressure ventilation (5) complications or history of drug allergy or severe allergic disease such as anaphylactic shock (6) malignancy within past five years at the time of informed consent (7) contraindications for lumbar puncture (8) contraindications for MRI including Magnetic Resonance Spectroscopy and Difusion Tensor Imaging (9) AST and ALT CK greater than 3 times upper limit of normal at screening (10) CK greater than 2.5 times upper limit of normal at screening (11) eGFR lower than 45 ml/min/1.73m^2 at screening (12) pregnant, nursing, or plan to get pregnant during the course of this clinical trial at screening (13) childbearing potential, or unwilling to use accepted methods of contraception (14) use of edaravone within 30 days from the start of pre-observation period (15) change the dosage or discontinuation of riluzole after informed consent (16) history of administration of EPI-589 (17) participation in a clinical trial before informed consent and within 30 days from investigational drug administration (If 5 times of the investigational drug half life is longer than 30 days, it should takes longer times than 5 times of the investigational drug half life) or willingness to participate for other clinical trial at the time of informed consent (18) history of cell therapy or gene therapy before infromed consent (19) ineligible for the clinical trial based on the investigator's judgement

Related Information

Contact

Public contact
Name Shotaro Haji
Address 2-50-1 Kuramoto-cho, Tokushima-shi, Tokushima, Japan Tokushima Japan 770-8503
Telephone +81-88-633-9658
E-mail chousei_EPI-589@umin.ac.jp
Affiliation Tokushima University Hospital
Scientific contact
Name Koji Fujita
Address 2-50-1 Kuramoto-cho, Tokushima-shi, Tokushima, Japan Tokushima Japan 770-8503
Telephone +81-88-633-9658
E-mail chousei_EPI-589@umin.ac.jp
Affiliation Tokushima University Hospital