Multicenter cooperative and investigator initiated clinical trial using valproic acid in childhood onset spinal muscular atrophy : Confirmatory Trial

JMACCT ID: JMA-IIA00231

Registered date:01/12/2015

Basic Information

• Recruitment status: Completed
• Health condition(s) or Problem(s) studied: spinal muscular atrophy
• Date of first enrollment: 29/01/2016
• Target sample size: 28
• Countries of recruitment: Japan
• Study type: INTERVENTIONAL
• Intervention(s): Intervention type:DRUG. Intervention1:SMART, Dose form:GRANULES, Route of administration:ORAL, intended dose regimen:VPA 12.5mg/kg or 25mg/kg is to be taken once a day after supper.. Control intervention1:Placebo, Dose form:GRANULES, Route of administration:ORAL, Intended dose regimen:Placebo is to be taken once a day after supper..

Outcome(s)

• Primary Outcome: HFMSE score. Timepoint:32week.
• Secondary Outcome: HFMS score, Motor function, WHO Motor milestones. Timepoint: 4week, 8week, 12week, 16week, 20week, 24week, 28week, 32week.

Key inclusion & exclusion criteria

• Age minimum: >=1 YEARS
• Age maximum: <7 YEARS
• Gender: BOTH

Include criteria

(1) Patients with SMN 1 gene mutation proven by gene testing
(2) Patients of SMA types 1 and 2.
(3) Patients with stable head and neck
(4) HFMSE score > 1point
(5) Patients who can visit the hospital as scheduled in clinical trials.
(6) Patients with a guardian under conditions below
1) A fixed guardian can comprehend the status of the patient and observe the patient's daily activities as required in the clinical trial.
2) Management of medicine is definitely possible.

Exclude criteria

(1)Patients who have had an operation for spinal scoliosis.
(2)Patients on mechanical ventilator with tracheotomy or who are judged to require mechanical ventilation with tracheotomy by the investigator (or sub-investigator).
(3)Patient with severe liver dysfunction, renal dysfunction, and circulatory diseases. (judged referring to grade 3 of "Severity Criteria for Drug Adverse Reaction" Yaku-Ann No 80, issued by the chief of Registration Division the Pharmaceutical Affairs Bureau, Ministry of Welfare)
(4)Patients given Riluzole, creatine, butyric acid compound, steroid hormone and sodium valproate within 8 weeks before the agreement was obtained.
(5)Patients with malignant tumors
(6)Patients with severe medical history of drug allergy such as anaphylactic shock, or serious side effect of drug.
(7)Patients participating in other clinical trials and administered an investigational new drug within 12 weeks before obtaining the agreement for this clinical trial.
(8) Patients scheduled to take a carbapenem antibiotic within the period from agreement to the end of observation.
(9) Patients who have started physical therapy for less than four weeks before obtaining the agreement for this clinical trial. Starting the respiratory physical therapy is accepted.
(10) Patients with urea cycle abnormalities.
(11) Other patients judged to be unsuitable to join this clinical trial by the investigator (or sub-investigator).

Related Information

• Primary Sponsor: Institute of Medical Genetics, Tokyo Women's Medical University
• Source(s) of Monetary Support: Japan Agency for Medical Research and Development