A Placebo-Controlled, Double-Blind, Randomized, Group-Controlled Trial Evaluating the Efficacy and Safety of AJA030 in Spinocerebellar Degeneration.

Onodera Osamu0 sites40 target enrollmentSeptember 30, 2020

Overview

Brief Summary

No summary available.

Registry

who.int

Start Date

September 30, 2020

End Date

TBD

Last Updated

2 years ago

Study Type

Interventional

Sex

All

Investigators

Sponsor

Onodera Osamu

Eligibility Criteria

Inclusion Criteria

•1\) Patients with genetically diagnosed autosomal dominant hereditary spinocerebellar degeneration (SCA6\). Note 1\)
•2\) Patients aged 20 years or older at the time of informed consent.
•3\) Patients who scored 1 or more In SARA(Scale for the Assessment and Rating of Ataxia) gait scores in the pre\-enrollment test and had a SARA sum score of 10 or more.
•4\) Patients who are able to walk 10m or more with or without assistive devices in the pre\-enrollment test.
•5\) Patients for whom written informed consent is obtained from the individual for participation in the clinical trial.
•Note 1\) Genetic diagnosis of the patient is not indispensable, when the disease type is confirmed by the genetic diagnosis in the family.

Exclusion Criteria

•1\) Patients with gait or balance disorders due to medical conditions other than spinocerebellar degeneration (stroke, brain tumor, head injury, multiple sclerosis, hypothyroidism, alcoholic ataxia, joint disease, others)
•2\) Patients with arginase deficiency (which exacerbates argininemia), patients with lysinuric protein intolerance who have a greater degree of inhibition of arginine absorption (which causes diarrhea with the use of arginine preparations)
•3\) Patients with dementia (MMSE\<\=23\), mental illness (psychosis, manic\-depressive illness, depression without treatment \[Beck Depression Inventory score\>\=21]), or a history of suicidal attempt.
•4\) Patients with complications that are considered inappropriate for participation in the study, such as serious heart disease, liver disease, kidney disease, or hematologic disease.
•5\) Pregnant or possibly pregnant patients and breastfeeding patients.
•6\) Patients who are unable to consent to use effective contraceptive methods during the study participation period.
•7\) Patients who took oral preparations that were judged by the principal investigator or the subinvestigator to be supplements containing algin U or L\-arginine as the main ingredient within 30 days prior to the date of informed consent.
•8\) Patients who participated in another clinical trial within 6 months before the date of obtaining informed consent.
•9\) Patients who are considered inappropriate to participate in the study by the principal investigator or the subinvestigator.