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Effects of Acetyl-Leucine on Spinocerebellar ataxia

Phase 1
Conditions
Spinocerebellar Ataxia.
Hereditary ataxia
Registration Number
IRCT20210413050958N7
Lead Sponsor
Mashhad University of Medical Sciences
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
Pending
Sex
All
Target Recruitment
4
Inclusion Criteria

Age above 6 years
Patients with a definitive diagnosis of Spinocerebellar ataxia
Having clinical signs
If the patient is taking any medication, he/she should maintain a constant dose/not change his/her treatment during the study period.
Have not taken any forbidden drugs including any variant of N-acetyl-DL-leucine, aminopyridines, Riluzole, gabapentin, Varenicline, Chlorzoxazone, sulfasalazine, Rosuvastatin at least 4 weeks before visit 1 and throughout the duration of the study
Signed informed consent form by the subjects or their parents after explaining the study objectives by the research team
Patient satisfaction

Exclusion Criteria

Having chronic diarrhea, visual loss, malignancies or insulin-dependent diabetes mellitus
History of hypersensitivity to the N-Acetyl-Leucine
Having severe vision or hearing impairment
Having arthritis or other musculoskeletal disorders

Study & Design

Study Type
interventional
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Scale for Assessment and Rating of Ataxia (SARA) score. Timepoint: Before the intervention and 4 weeks after taking supplement or placebo in every study stage. Method of measurement: Scale for Assessment and Rating of Ataxia (SARA) Questionnaire.;Spinocerebellar Ataxia Functional Index (SCAFI). Timepoint: Before the intervention and 4 weeks after taking supplement or placebo in every study stage. Method of measurement: Spinocerebellar Ataxia Functional 8-m walking time (SCAFI-8MWT) and Spinocerebellar Ataxia Functional 9-hole peg test (SCAFI-9HPT).
Secondary Outcome Measures
NameTimeMethod
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