Spinocerebellar Ataxia. Hereditary ataxia
Conditions
Interventions
Intervention 1: Intervention group: Subjects in the intervention group receive N-Acetyl-L-Leucine sachet (daily intake of 2-4 gr depending on the subjects’ weight) for 4 weeks (n=2) and then after a 4
Sponsors
Mashhad University of Medical Sciences
Eligibility
Sex/Gender
All
Age
6 Years to No maximum
Inclusion criteria
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
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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
| Measure | Time frame |
|---|---|
| The quality of life. Timepoint: Before the intervention and 4 weeks after taking supplement or placebo in every study stage. Method of measurement: Pediatric Quality of Life (PedsQL) questionnaire.;Cell blood counts. Timepoint: Before the intervention and 4 weeks after taking supplement or placebo in every study stage. Method of measurement: Sysmex autoanalyser.;Aspartate amino transferase. Timepoint: Before the intervention and 4 weeks after taking supplement or placebo in every study stage. Method of measurement: Autoanalyser.;Alanin amino transferase. Timepoint: Before the intervention and 4 weeks after taking supplement or placebo in every study stage. Method of measurement: Autoanalyser.;Creatinine. Timepoint: Before the intervention and 4 weeks after taking supplement or placebo in every study stage. Method of measurement: Autoanalyser.;Urea. Timepoint: Before the intervention and 4 weeks after taking supplement or placebo in every study stage. Method of measurement: Autoanalyser.;Bilirubin. Timepoint: Before the intervention and 4 weeks after taking supplement or placebo in every study stage. Method of measurement: Autoanalyser. | — |
Countries
Iran (Islamic Republic of)
Contacts
Public ContactMaryam Saberi-Karimian
Mashhad University of Medical Sciences
Outcome results
None listed