A Randomized Parallel-Group Study to Evaluate the Efficacy and Safety of Immunotherapy for Idiopathic Cerebellar Ataxia

JRCT ID: jRCTs031200250

Registered date:16/12/2020

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: idiopathic cerebellar ataxia
• Date of first enrollment: 11/08/2021
• Target sample size: 20
• Study type: Interventional

Intervention(s)

early treatment group: steroid pulse therapy (methylprednisolone 1000 mg/day) at week 1 and week 3.

delayed treatment group: steroid pulse therapy (methylprednisolone 1000 mg/dose) at weeks 5 and 7.

Outcome(s)

• Primary Outcome: The difference in SARA scores between the baseline and week 4.

Secondary Outcome

The difference in 9-hole peg test time between the baseline and week 4.
The difference in SARA scores between baseline and the best-response.
The difference between the change in SARA score during the pre-treatment period and the best change after the initiation of the treatment in the delayed treatment group
The difference between the best 9 HPT before and after the start of treatment in each group
The difference of SARA score before and after the initiation of treatment (4 and 8 weeks) in each group
The difference in 9 HPT between the pre-treatment and post-treatment (4 weeks and 8 weeks) in each group
Pre-treatment serum anti-cerebellar antibody titers and post-treatment (4 weeks and 8 weeks) serum anti-cerebellar antibody titers

Key inclusion & exclusion criteria

• Age minimum: >= 30age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

-30 years-old and over (at the time of obtaining consent)
- idiopathic cerebellar ataxia according to the diagnostic criteria of the Ataxia Research Group of the Ministry of Health, Labour, and Welfare. (Note that "bilateral cerebellar atrophy on head CT and MRI" need not be met in this study.)
- Solitary case (the definition of the solitary case follows the criteria for idiopathic cerebellar ataxia)
- A slowly progressive course of events (a history of cerebellar ataxia of at least one year from onset to visit)
- seropositivity for the anti-cerebellar antibodies (confirmed by immunohistochemistry of rat brain section)
- Positive immunohistological serum anti-cerebellar antibodies
- Able to walk with a cane or by oneself at the time of obtaining consent
- SARA score of 5 or more at the time of consent

Exclude criteria

- already included in another trial which includes any intervention study
- Those with a confirmed genetic diagnosis of hereditary spinocerebellar degeneration or those with a strong suspicion of hereditary origin
- Those with a history of alcoholism or drug abuse
- Use of medications that are likely to cause lightheadedness or cerebellar ataxia at the time of consent (e.g., antiepileptic drugs, benzodiazepines)
- Those who are mentally disabled at the time of obtaining consent
- Previous or currently treated stomach ulcers
- Untreated or poorly controlled diabetes at the time of consent
- Persons with severe hepatic or renal impairment at the time of obtaining consent
- If you are pregnant or hoping to become pregnant at the time of obtaining consent
- Dementia at the time of consent
- At the time of obtaining consent, the patient is taking steroids or immunosuppressive drugs
- Those taking antiplatelet or anticoagulant drugs that cannot be stopped at the time of obtaining consent
- At the time of obtaining consent, the patient has an infectious disease that may be aggravated by steroids
- Other items deemed inappropriate by the principal investigator/associate

Related Information

• Primary Sponsor: Yoshikura Nobuaki
• Source(s) of Monetary Support: Pfizer Inc.