<?xml version="1.0" encoding="utf-8"?>
<!DOCTYPE trials [
<!ELEMENT trials (trial+)>

<!ELEMENT trial (main,contacts,countries,criteria,health_condition_code,health_condition_keyword,intervention_code,
          intervention_keyword,primary_outcome,secondary_outcome,secondary_sponsor,secondary_ids,source_support,ethics_reviews)>

<!ELEMENT main (trial_id,utrn?,reg_name,date_registration,primary_sponsor,public_title,acronym?,scientific_title,scientific_acronym?,
          date_enrolment,type_enrolment,target_size,recruitment_status,url?,study_type,study_design,phase,hc_freetext?,i_freetext?,results_actual_enrolment,results_date_completed,results_url_link,results_summary,           results_date_posted,results_date_first_publication,results_baseline_char,results_participant_flow,results_adverse_events,results_outcome_measures,results_url_protocol,results_IPD_plan, results_IPD_description)>
<!ELEMENT trial_id (#PCDATA)>
<!ELEMENT utrn (#PCDATA)>
<!ELEMENT reg_name (#PCDATA)>
<!ELEMENT date_registration (#PCDATA)><!-- dd/mm/yyyy -->
<!ELEMENT primary_sponsor (#PCDATA)>
<!ELEMENT public_title (#PCDATA)>
<!ELEMENT acronym (#PCDATA)>
<!ELEMENT scientific_title (#PCDATA)>
<!ELEMENT scientific_acronym (#PCDATA)>
<!ELEMENT date_enrolment (#PCDATA)><!-- dd/mm/yyyy -->
<!ELEMENT type_enrolment (#PCDATA)>
<!ELEMENT target_size (#PCDATA)>
<!ELEMENT recruitment_status (#PCDATA)><!-- Pending,Recruiting,Suspended,Complete,Other -->
<!ELEMENT url (#PCDATA)>
<!ELEMENT study_type (#PCDATA)><!-- interventional,observational -->
<!ELEMENT study_design (#PCDATA)>
<!ELEMENT phase (#PCDATA)>
<!ELEMENT hc_freetext (#PCDATA)>
<!ELEMENT i_freetext (#PCDATA)>
<!ELEMENT results_actual_enrolment (#PCDATA)>
<!ELEMENT results_date_completed (#PCDATA)><!-- dd/mm/yyyy -->
<!ELEMENT results_url_link (#PCDATA)>
<!ELEMENT results_summary (#PCDATA)>
<!ELEMENT results_date_posted (#PCDATA)><!-- dd/mm/yyyy -->
<!ELEMENT results_date_first_publication (#PCDATA)><!-- dd/mm/yyyy -->
<!ELEMENT results_baseline_char (#PCDATA)>
<!ELEMENT results_participant_flow (#PCDATA)>
<!ELEMENT results_adverse_events (#PCDATA)>
<!ELEMENT results_outcome_measures (#PCDATA)>
<!ELEMENT results_url_protocol (#PCDATA)>
<!ELEMENT results_IPD_plan (#PCDATA)>
<!ELEMENT results_IPD_description (#PCDATA)>


<!ELEMENT contacts (contact+)>
<!ELEMENT contact (type,firstname,middlename,lastname,address,city,country1,zip,telephone,email,affiliation)>
<!ELEMENT type (#PCDATA)><!-- Public,Scientific -->
<!ELEMENT firstname (#PCDATA)>
<!ELEMENT middlename (#PCDATA)>
<!ELEMENT lastname (#PCDATA)>
<!ELEMENT address (#PCDATA)>
<!ELEMENT city (#PCDATA)>
<!ELEMENT country1 (#PCDATA)>
<!ELEMENT zip (#PCDATA)>
<!ELEMENT telephone (#PCDATA)>
<!ELEMENT email (#PCDATA)>
<!ELEMENT affiliation (#PCDATA)>

<!ELEMENT countries (country2+)>
<!ELEMENT country2 (#PCDATA)>

<!ELEMENT criteria (inclusion_criteria,agemin,agemax,gender,exclusion_criteria)>
<!ELEMENT inclusion_criteria (#PCDATA)>
<!ELEMENT agemin (#PCDATA)>
<!ELEMENT agemax (#PCDATA)>
<!ELEMENT gender (#PCDATA)>
<!ELEMENT exclusion_criteria (#PCDATA)>

<!ELEMENT health_condition_code (hc_code+)>
<!ELEMENT hc_code (#PCDATA)>

<!ELEMENT health_condition_keyword (hc_keyword+)>
<!ELEMENT hc_keyword (#PCDATA)>

<!ELEMENT intervention_code (i_code+)>
<!ELEMENT i_code (#PCDATA)>

<!ELEMENT intervention_keyword (i_keyword+)>
<!ELEMENT i_keyword (#PCDATA)>

<!ELEMENT primary_outcome (prim_outcome+)>
<!ELEMENT prim_outcome (#PCDATA)>

<!ELEMENT secondary_outcome (sec_outcome+)>
<!ELEMENT sec_outcome (#PCDATA)>

<!ELEMENT secondary_sponsor (sponsor_name+)>
<!ELEMENT sponsor_name (#PCDATA)>

<!ELEMENT secondary_ids (secondary_id+)>
<!ELEMENT secondary_id (sec_id,issuing_authority)>
<!ELEMENT sec_id (#PCDATA)>
<!ELEMENT issuing_authority (#PCDATA)>

<!ELEMENT source_support (source_name+)>
<!ELEMENT source_name (#PCDATA)>

<!ELEMENT ethics_reviews (ethics_review+)>
<!ELEMENT ethics_review (status,approval_date,contact_name,contact_address,contact_phone,contact_email)>
<!ELEMENT status (#PCDATA)><!-- Not approved,Approved,NA -->
<!ELEMENT approval_date (#PCDATA)><!-- dd/mm/yyyy -->
<!ELEMENT contact_name (#PCDATA)>
<!ELEMENT contact_address (#PCDATA)>
<!ELEMENT contact_phone (#PCDATA)>
<!ELEMENT contact_email (#PCDATA)>
]>
<trials>
  <trial>
    <main>
      <trial_id>IRCT20210413050958N7</trial_id>
      <utrn></utrn>
      <reg_name>IRCT</reg_name>
      <date_registration>2023-05-26</date_registration>
      <primary_sponsor>Mashhad University of Medical Sciences</primary_sponsor>
      <public_title>Effects of Acetyl-Leucine on Spinocerebellar ataxia</public_title>
      <acronym></acronym>
      <scientific_title>The effects of N-Acetyl-L-Leucine on the improvement of symptoms in patients with Spinocerebellar ataxia</scientific_title>
      <scientific_acronym></scientific_acronym>
      <date_enrolment>2023-06-22</date_enrolment>
      <type_enrolment>anticipated</type_enrolment>
      <target_size>4</target_size>
      <recruitment_status>Complete</recruitment_status>
      <url>https://irct.ir/trial/70303</url>
      <study_type>interventional</study_type>
      <study_design>Randomization: Randomized, Blinding: Triple blinded, Placebo: Used, Assignment: Crossover, Purpose: Treatment, 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 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.</study_design>
      <phase>1-2</phase>
      <hc_freetext>Spinocerebellar Ataxia.</hc_freetext>
      <i_freetext>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-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). Intervention 2: 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.</i_freetext>
      <results_actual_enrolment></results_actual_enrolment>
      <results_date_completed></results_date_completed>
      <results_url_link></results_url_link>
      <results_summary></results_summary>
      <results_date_posted></results_date_posted>
      <results_date_first_publication></results_date_first_publication>
      <results_baseline_char></results_baseline_char>
      <results_participant_flow></results_participant_flow>
      <results_adverse_events></results_adverse_events>
      <results_outcome_measures></results_outcome_measures>
      <results_url_protocol></results_url_protocol>
      <results_IPD_plan>No - There is not a plan to make this available</results_IPD_plan>
      <results_IPD_description>Justification or reason for not sharing IPD is Raw data will be shared upon a reasonable request from the corresponding author.</results_IPD_description>
    </main>
    <contacts>
      <contact>
        <type>public</type>
        <firstname>Maryam Saberi-Karimian</firstname>
        <middlename></middlename>
        <lastname></lastname>
        <address>Research Council, Ghoreishi bildings, Daneshgah Ave.</address>
        <city>Mashhad</city>
        <country1>Iran (Islamic Republic of)</country1>
        <zip>99199-91766</zip>
        <telephone>+98 51 3882 3260</telephone>
        <email>saberikm@mums.ac.ir</email>
        <affiliation>Mashhad University of Medical Sciences</affiliation>
      </contact>
      <contact>
        <type>scientific</type>
        <firstname>Maryam Saberi-Karimian</firstname>
        <middlename></middlename>
        <lastname></lastname>
        <address>Research Council, Ghoreishi bildings, Daneshgah Ave.</address>
        <city>Mashhad</city>
        <country1>Iran (Islamic Republic of)</country1>
        <zip>99199-91766</zip>
        <telephone>+98 915 528 2874</telephone>
        <email>saberikm@mums.ac.ir</email>
        <affiliation>Mashhad University of Medical Sciences</affiliation>
      </contact>
    </contacts>
    <countries>
      <country2>Iran (Islamic Republic of)</country2>
    </countries>
    <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</inclusion_criteria>
      <agemin>6 years</agemin>
      <agemax>no limit</agemax>
      <gender>Both</gender>
      <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</exclusion_criteria>
    </criteria>
    <health_condition_code>
      <hc_code>G11</hc_code>
    </health_condition_code>
    <health_condition_keyword>
      <hc_keyword>Hereditary ataxia</hc_keyword>
    </health_condition_keyword>
    <intervention_code>
      <i_code>Treatment - Drugs</i_code>
      <i_code>Placebo</i_code>
    </intervention_code>
    <intervention_keyword>
      <i_keyword>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).</i_keyword>
      <i_keyword>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.</i_keyword>
    </intervention_keyword>
    <primary_outcome>
      <prim_outcome>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.</prim_outcome>
      <prim_outcome>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).</prim_outcome>
    </primary_outcome>
    <secondary_outcome>
      <sec_outcome>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.</sec_outcome>
      <sec_outcome>Cell blood counts. Timepoint: Before the intervention and 4 weeks after taking supplement or placebo in every study stage. Method of measurement: Sysmex autoanalyser.</sec_outcome>
      <sec_outcome>Aspartate amino transferase. Timepoint: Before the intervention and 4 weeks after taking supplement or placebo in every study stage. Method of measurement: Autoanalyser.</sec_outcome>
      <sec_outcome>Alanin amino transferase. Timepoint: Before the intervention and 4 weeks after taking supplement or placebo in every study stage. Method of measurement: Autoanalyser.</sec_outcome>
      <sec_outcome>Creatinine. Timepoint: Before the intervention and 4 weeks after taking supplement or placebo in every study stage. Method of measurement: Autoanalyser.</sec_outcome>
      <sec_outcome>Urea. Timepoint: Before the intervention and 4 weeks after taking supplement or placebo in every study stage. Method of measurement: Autoanalyser.</sec_outcome>
      <sec_outcome>Bilirubin. Timepoint: Before the intervention and 4 weeks after taking supplement or placebo in every study stage. Method of measurement: Autoanalyser.</sec_outcome>
    </secondary_outcome>
    <secondary_sponsor>
      <sponsor_name></sponsor_name>
    </secondary_sponsor>
    <secondary_ids>
      <secondary_id>
        <sec_id></sec_id>
        <issuing_authority></issuing_authority>
      </secondary_id>
    </secondary_ids>
    <source_support>
      <source_name>Mashhad University of Medical Sciences</source_name>
    </source_support>
    <ethics_reviews>
      <ethics_review>
        <status>Approved</status>
        <approval_date>2023-02-28</approval_date>
        <contact_name>Ethics Committee of Mashhad University of Medical Sciences</contact_name>
        <contact_address>Research Council, Ghoreishi bildings, Daneshgah Ave. Mashhad Razavi Khorasan Iran (Islamic Republic of)</contact_address>
        <contact_phone></contact_phone>
        <contact_email></contact_email>
      </ethics_review>
    </ethics_reviews>
  </trial>
</trials>
