Amyotrophic Lateral Sclerosis (ALS)
Conditions
Interventions
24 weeks repeated intrathecal administration of KP-100IT at 2 mg (once per 2 weeks, double blind period)
24 weeks repeated intrathecal administration of placebo (once per 2 weeks, double blind period)
Sponsors
Tohoku University
Kringle Pharma, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: [Interim Registration] Subjects who are - Diagnosed as "Clinically Definite ALS", "Clinically Probable ALS" or "Clinically Possible-Laboratory-supported ALS" according to the El Escorial revised Criteria, and within 30 months after onset of the disease (at the time of obtaining informed consent (IC)) - Classified as class 1 or 2 in ALS severity classification - Aged 20 or older and 70 or younger at the time of obtaining informed consent (IC) - With 2 points or more in every question of ALSFRS-R (in writing and eating action (1), both sides should be 2 points or more) - %FVC is not less than 70 - Two points or more in every question of bulbar symptom scale in the modified Norris Scale - Agreed by written IC for entry in the trial [Registration] Subjects who - meet the following criteria in addition to the inclusion criteria for the interim registration - Change in ALSFRS-R score during 12 weeks-preobsevation period is within the range between -1 and -3 - Can come into hospital by the day before the day of NP022 implantation and can stay hospital until the next day of the first administration
Exclusion criteria
Exclusion criteria: Subjects who - Have serious hepatic disorder, renal disorder, cardiovascular disease, pulmonary disorder, hematological disorder or metabolism disorder, and are judged by the investigators to be improper for entry in the trial - With HbA1c (NGSP scale) of 6.5% or higher at the check of screening period - Participated in clinical trials of INDs or clinical studies receiving treatment within a month before interim registration (30 days including interim registration date) - With the following diseases or symptoms which might affect safety: malignant tumor, intrathecal infection, intrathecal tumor, proliferative retinopathy, depressed respiratory function (less than 70 of %FVC) - Have a medical history of cancer - Have a drug allergy - Newly started taking of Riluzole after obtaining IC - Participated in the Phase I Trial (protocol KP-100-ND001) - With allergy to antibiotics or intolerance for devices implantation - With allergy to the material of NP022, and including suspected above - Have an anatomical difficulty in intrathecal catheterization - Have an abnormality other than ALS in the spinal cord, the spine, the subarachnoidal cavity, or cerebrospinal fluid circulation, and are judged to be inappropriate for this trial - With infectious diseases requiring a systemic therapy, meningitis, sepsis, bacteremia, peritonitis, or cutaneous infection, and including suspected above - With bleeding diathesis - With impaired coagulation-fibrinolysis, or taking anticoagulant or antiplatelet drugs - Diagnosed as dementia - Mentally impaired or have psychiatric disorders, and are judged to be inappropriate for the trial - Pregnant (including suspected), nursing, wishing to become pregnant, or unable to prevent conception during the trial period using contraceptive methods - Judged by the investigators to be inappropriate for any reason
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Difference in the change of ALSFRS-R score (delta ALSFRS-R) during the 24 weeks-double blind period, starting from the time point before initial administration (implantation period) to the point of 24 weeks observation between Placebo and KP-100IT groups | — |
Secondary
| Measure | Time frame |
|---|---|
| Comparison of changing rate in ALSFRS-R score per 30 days between before and after the treatment Difference in the following items between at the time before initial administration (implantation period) and at the point of 24 weeks in administration/observation period ALSFRS-R sub score of each domain (bulbar function, extremities function, respiratory function), %FVC, MMT Score (extremities, neck muscles), Modified Norris Scale (extremities symptom score, bulbar symptom score, total scores), weight, grip strength, ALSAQ40 score, ALS severity classification Time to death or to a defined event (death, inability of independent ambulation, loss of unilateral upper limb function, tracheostomy, ventilated, tube feeding, loss of vocal conversation) Time reaching to the state of %FVC<50% (time length until %FVC decreases to <50%) Time length until ALSFRS-R score decreases by 6 points or more [score change (delta ALSFRS-R) from initial administration (implantation period) is -6 or less] Discontinuance rate after initial administration Safety of repeated administration of KP-100IT for a long period Safety and defects of NP022 when KP-100IT is intrathecally administered repeatedly for a long period | — |
Countries
Japan
Contacts
Public ContactHitoshi Warita, MD, PhD
Tohoku University Hospital Department of Neurology
Outcome results
None listed