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

JRCT ID: jRCT2031200135

Registered date:30/09/2020

Basic Information

• Recruitment status: Complete
• Health condition(s) or Problem(s) studied: spinocerebellar degeneration (SCD)
• Date of first enrollment: 29/10/2020
• Target sample size: 40
• Study type: Interventional
• Intervention(s): Randomized, Double-Blind, Placebo-Controlled Study

Outcome(s)

• Primary Outcome: Change from baseline in SARA "Total" scores at Week 48

Secondary Outcome

1) SARA total scores after 4, 8, 16, 24, 32 ,40 and 48 weeks
2) SARA Walking, Standing Scores
3) SARA scores (other than walking and standing scores)
4) Inspection of center of gravity oscillation (total locus length for 30 seconds, rectangular area)
5) Timed Up and Go Test (TUG) (shortest time)
6) Beck Depression Inventory-II (BDI-II)
7) Clinical Global Impression (CGI)
8) Patient Global Impression of Improvement (PGI-I)
9) Short-Form 8 (SF-8)

Key inclusion & exclusion criteria

• Age minimum: >= 20age old
• Age maximum: Not applicable
• Gender: Both

Include 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.

Exclude 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.

Related Information

• Primary Sponsor: Onodera Osamu
• Source(s) of Monetary Support: Japan Agency for Medical Research and Development