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The effects of N-Acetyl-L-Leucine on the improvement of symptoms in patients with Spinocerebellar ataxia

Protocol summary

Study aim
Effects of Acetyl Leucine on symptoms of Spinocerebellar ataxia
Design
The current study is a randomized, triple-blind, crossover clinical trial with parallel groups. A total of 4 subjects will be enrolled. The table of random numbers was used for randomization.
Settings and conduct
Patients with Spinocerebellar ataxia referring to Neurology Clinic of the Ghaem Hospital are enrolled in the study. All volunteers, care providers and statistician are blinded after assignment to intervention. The supplements containers were coded as A and B by a non-researcher person and remained confidential until data analysis. Placebo powders are similar to supplements regarding the weight and color.
Participants/Inclusion and exclusion criteria
Inclusion criteria:Age>6 years, Having a definitive diagnosis of Spinocerebellar ataxia with clinical signs, having a stable treatment, and not taken forbidden drugs. Exclusion criteria:Having diarrhea, visual loss, malignancies, diabetes mellitus, history of hypersensitivity to the N-Acetyl-Leucine, Having severe vision or hearing impairment, Having arthritis.
Intervention groups
Intervention group: The intervention group receive N-Acetyl-L-Leucine sachet (daily intake of 2-4 grams depending on their weight) for 4 weeks and then after a 4-weeks wash-out period, crossed over to the alternate regimen. The participants take one sachet every day, which was contained in an unlabeled bottle. Supplements are from Hubei (Shenzhen, China). Control group: Receiving a placebo sachet (daily consumption between 2-4 grams depending on the subject's weight) for 4 weeks and then after a 4-weeks wash-out period, crossed over to the alternate regimen. Participants take a placebo daily orally in an unlabeled bottle. The placebo is prepared by from faculty of pharmacy (Mashhad, Iran).
Main outcome variables
Scale for Assessment and Rating of Ataxia score

General information

Reason for update
Acronym
IRCT registration information
IRCT registration number: IRCT20210413050958N7
Registration date: 2023-05-26, 1402/03/05
Registration timing: prospective

Last update: 2023-05-26, 1402/03/05
Update count: 0
Registration date
2023-05-26, 1402/03/05
Registrant information
Name
Maryam Saberi-Karimian
Name of organization / entity
Country
Iran (Islamic Republic of)
Phone
+98 51 3764 3808
Email address
saberikm@mums.ac.ir
Recruitment status
Recruitment complete
Funding source
Expected recruitment start date
2023-06-22, 1402/04/01
Expected recruitment end date
2023-11-22, 1402/09/01
Actual recruitment start date
empty
Actual recruitment end date
empty
Trial completion date
empty
Scientific title
The effects of N-Acetyl-L-Leucine on the improvement of symptoms in patients with Spinocerebellar ataxia
Public title
Effects of Acetyl-Leucine on Spinocerebellar ataxia
Purpose
Treatment
Inclusion/Exclusion 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:
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
Age
From 6 years old
Gender
Both
Phase
1-2
Groups that have been masked
  • Participant
  • Care provider
  • Investigator
  • Data analyser
Sample size
Target sample size: 4
Randomization (investigator's opinion)
Randomized
Randomization description
Subjects are divided into one of two intervention/control groups using the random numbers prepared by a methodologist using www.sealedenvelope.com, so that who enrolled the trial can select a sealed envelope with random allocation sequence to the intervention or control group. An expert outside the research team blinds the drugs. The executive team register the subjects and assign them to the intervention. All volunteers, the executive team, and the statistical analyst will be blinded by the interventions.
Blinding (investigator's opinion)
Triple blinded
Blinding description
All volunteers, the executive team, and the statistical analyst will be blinded by the interventions. So that, the supplements containers were coded as A and B by a non-researcher person and remained confidential until data analysis. The placebos powders are similar to the supplements regarding the weight and color.
Placebo
Used
Assignment
Crossover
Other design features

Secondary Ids

empty

Ethics committees

1

Ethics committee
Name of ethics committee
Ethics Committee of Mashhad University of Medical Sciences
Street address
Research Council, Ghoreishi bildings, Daneshgah Ave.
City
Mashhad
Province
Razavi Khorasan
Postal code
99199-91766
Approval date
2023-02-28, 1401/12/09
Ethics committee reference number
IR.MUMS.MEDICAL.REC.1402.038

Health conditions studied

1

Description of health condition studied
Spinocerebellar Ataxia
ICD-10 code
G11
ICD-10 code description
Hereditary ataxia

Primary outcomes

1

Description
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

2

Description
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 outcomes

1

Description
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

2

Description
Cell blood counts
Timepoint
Before the intervention and 4 weeks after taking supplement or placebo in every study stage
Method of measurement
Sysmex autoanalyser

3

Description
Aspartate amino transferase
Timepoint
Before the intervention and 4 weeks after taking supplement or placebo in every study stage
Method of measurement
Autoanalyser

4

Description
Alanin amino transferase
Timepoint
Before the intervention and 4 weeks after taking supplement or placebo in every study stage
Method of measurement
Autoanalyser

5

Description
Creatinine
Timepoint
Before the intervention and 4 weeks after taking supplement or placebo in every study stage
Method of measurement
Autoanalyser

6

Description
Urea
Timepoint
Before the intervention and 4 weeks after taking supplement or placebo in every study stage
Method of measurement
Autoanalyser

7

Description
Bilirubin
Timepoint
Before the intervention and 4 weeks after taking supplement or placebo in every study stage
Method of measurement
Autoanalyser

Intervention groups

1

Description
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-weeks wash-out period, they were crossed over to the alternate regimen. The participants take one sachet every day, which was contained in an unlabeled bottle. Supplements are from Hubei ipure Biotech co., ltd (Shenzhen, China).
Category
Treatment - Drugs

2

Description
Control group: The control group received a placebo sachet (daily consumption between 2 to 4 grams depending on the subject's weight) for 4 weeks (n=2) and then after a 4-weeks wash-out period, they were crossed over to the alternate regimen. Participants take a placebo every day orally in an unlabeled bottle. The placebo is prepared by from faculty of pharmacy (Mashhad, Iran) company.
Category
Placebo

Recruitment centers

1

Recruitment center
Name of recruitment center
Ghaem hospital
Full name of responsible person
Maryam Saberi-Karimian
Street address
Ahmadabad Street, Ghaem Hospital
City
Mashhad
Province
Razavi Khorasan
Postal code
99199-91766
Phone
+98 51 3840 0001
Email
saberikm@mums.ac.ir

Sponsors / Funding sources

1

Sponsor
Name of organization / entity
Mashhad University of Medical Sciences
Full name of responsible person
Majid Ghayour-Mobarhan
Street address
Research Council, Ghoreishi bildings, Daneshgah Ave.
City
Mashhad
Province
Razavi Khorasan
Postal code
99199-91766
Phone
+98 51 3845 2472
Email
ghayourm@mums.ac.ir
Grant name
Grant code / Reference number
Is the source of funding the same sponsor organization/entity?
Yes
Title of funding source
Mashhad University of Medical Sciences
Proportion provided by this source
100
Public or private sector
Public
Domestic or foreign origin
Domestic
Category of foreign source of funding
empty
Country of origin
Type of organization providing the funding
Academic

Person responsible for general inquiries

Contact
Name of organization / entity
Mashhad University of Medical Sciences
Full name of responsible person
Maryam Saberi-Karimian
Position
Assistant professor
Latest degree
Ph.D.
Other areas of specialty/work
Biochemistry
Street address
Research Council, Ghoreishi bildings, Daneshgah Ave.
City
Mashhad
Province
Razavi Khorasan
Postal code
99199-91766
Phone
+98 51 3882 3260
Email
saberikm@mums.ac.ir

Person responsible for scientific inquiries

Contact
Name of organization / entity
Mashhad University of Medical Sciences
Full name of responsible person
Maryam Saberi-Karimian
Position
Assistant Professor
Latest degree
Ph.D.
Other areas of specialty/work
Biochemistry
Street address
Research Council, Ghoreishi bildings, Daneshgah Ave.
City
Mashhad
Province
Razavi Khorasan
Postal code
99199-91766
Phone
+98 915 528 2874
Email
saberikm@mums.ac.ir

Person responsible for updating data

Contact
Name of organization / entity
Mashhad University of Medical Sciences
Full name of responsible person
Maryam Saberi-Karimian
Position
Assistant Professor
Latest degree
Ph.D.
Other areas of specialty/work
Biochemistry
Street address
Research Council, Ghoreishi bildings, Daneshgah Ave.
City
Mashhad
Province
Razavi Khorasan
Postal code
99199-91766
Phone
+98 51 3882 3260
Email
saberikm@mums.ac.ir

Sharing plan

Deidentified Individual Participant Data Set (IPD)
No - There is not a plan to make this available
Justification/reason for indecision/not sharing IPD
Raw data will be shared upon a reasonable request from the corresponding author.
Study Protocol
Yes - There is a plan to make this available
Statistical Analysis Plan
No - There is not a plan to make this available
Informed Consent Form
Undecided - It is not yet known if there will be a plan to make this available
Clinical Study Report
Yes - There is a plan to make this available
Analytic Code
No - There is not a plan to make this available
Data Dictionary
No - There is not a plan to make this available
Title and more details about the data/document
There is no plan to share the data.
When the data will become available and for how long
After publication of paper(s) upon a reasonable request
To whom data/document is available
Study PI and executive team
Under which criteria data/document could be used
For reasonable research or clinical purpose
From where data/document is obtainable
Dr. Maryam Saberi-Karimian
What processes are involved for a request to access data/document
Direct e-mail
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