<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT04426149</url>
  </required_header>
  <id_info>
    <org_study_id>UKM PPI/111/8/JEP-2017-826</org_study_id>
    <nct_id>NCT04426149</nct_id>
  </id_info>
  <brief_title>Clinical Effects of Oral Trehalose In Patients With Spinocerebellar Ataxia 3</brief_title>
  <official_title>Clinical Effects of Oral Trehalose In Patients With Spinocerebellar Ataxia 3: A Pilot Study</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>National University of Malaysia</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>National University of Malaysia</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
    <is_us_export>No</is_us_export>
  </oversight_info>
  <brief_summary>
    <textblock>
      There are no clinically established treatments which have been proven to delay the disease&#xD;
      progression in spinocerebellar ataxia (SCA) 3. Most available treatments are only for symptom&#xD;
      alleviation, and thus the majority of patients will eventually progress to needing and wheel&#xD;
      chair and eventually bedridden.&#xD;
&#xD;
      As trehalose appear to be potentially promising treatment in SCA, the investigators aim to&#xD;
      conduct this study using oral trehalose in our genetically confirmed SCA 3 patients.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This prospective single arm interventional study involved 13 genetically confirmed&#xD;
      spinocerebellar ataxia (SCA) 3 patients with no concomitant diabetes, over 6 months.&#xD;
      Following baseline assessment, patients were instructed to ingest 100g of oral trehalose&#xD;
      diluted in 500ml of water or other beverages daily. Assessments were performed at baseline,&#xD;
      2, 4 and 6 months using ataxia rating scales (SARA, SCAFI and INAS) and EQ-5D-3L scale for&#xD;
      quality of life assessment.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">March 7, 2018</start_date>
  <completion_date type="Actual">September 7, 2018</completion_date>
  <primary_completion_date type="Actual">September 7, 2018</primary_completion_date>
  <phase>N/A</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>scale of rating of ataxia (SARA) score months,</measure>
    <time_frame>2 monthly intervals for 6 months</time_frame>
    <description>Assessment of SARA scores by a single assessor</description>
  </primary_outcome>
  <primary_outcome>
    <measure>SCA Functional Index Scores</measure>
    <time_frame>2 monthly intervals for 6 months</time_frame>
    <description>Assessment of SCAFI by a single assessor</description>
  </primary_outcome>
  <primary_outcome>
    <measure>EQ5D3L - quality of life scores</measure>
    <time_frame>2 monthly intervals for 6 months</time_frame>
    <description>Assessment of quality of life scores</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Side effects Profile</measure>
    <time_frame>2 monthly intervals for 6 months</time_frame>
    <description>Adverse Effects</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Blood investigation</measure>
    <time_frame>At baseline and at 6 months</time_frame>
    <description>Measurement of renal profile, fasting blood glucose, full blood count and liver profile</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">13</enrollment>
  <condition>Spinocerebellar Ataxia 3</condition>
  <arm_group>
    <arm_group_label>interventional</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>supplement: trehalose</description>
  </arm_group>
  <intervention>
    <intervention_type>Dietary Supplement</intervention_type>
    <intervention_name>trehalose</intervention_name>
    <description>patients were instructed to ingest 100g of oral trehalose diluted in 500ml of water or other beverages daily</description>
    <arm_group_label>interventional</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. DNA diagnosis of SCA 3 in the study subject of his/ her affected family member(s)&#xD;
&#xD;
          2. Consent to participate in the study&#xD;
&#xD;
          3. The age of 18 years and older&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Unconfirmed SCA 3&#xD;
&#xD;
          2. Concomitant disorder(s) that affect SARA and other ataxia measures used in this study&#xD;
&#xD;
          3. Diabetes&#xD;
&#xD;
          4. Malabsorption of trehalose underlies intolerance to mushrooms, since the lack of&#xD;
             absorption results in diarrhoea and intestinal distress.&#xD;
&#xD;
          5. Less than 18 years old&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>Accepts Healthy Volunteers</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>NORLINAH MOHAMED IBRAHIM, MBBCH</last_name>
    <role>Principal Investigator</role>
    <affiliation>norlinah@ppukm.ukm.edu.my</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Pusat Perubatan Universiti Kebangsaan Malaysia</name>
      <address>
        <city>Kuala Lumpur</city>
        <zip>56000</zip>
        <country>Malaysia</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Malaysia</country>
  </location_countries>
  <verification_date>June 2020</verification_date>
  <study_first_submitted>April 18, 2019</study_first_submitted>
  <study_first_submitted_qc>June 8, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">June 11, 2020</study_first_posted>
  <last_update_submitted>June 8, 2020</last_update_submitted>
  <last_update_submitted_qc>June 8, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">June 11, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>National University of Malaysia</investigator_affiliation>
    <investigator_full_name>Dr Norlinah Mohamed Ibrahim</investigator_full_name>
    <investigator_title>Professor</investigator_title>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Ataxia</mesh_term>
    <mesh_term>Cerebellar Ataxia</mesh_term>
    <mesh_term>Spinocerebellar Ataxias</mesh_term>
    <mesh_term>Spinocerebellar Degenerations</mesh_term>
    <mesh_term>Machado-Joseph Disease</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT04426149
org study id: UKM PPI/111/8/JEP-2017-826
nct id: NCT04426149
lead sponsor: 
has dmc: Yes
is fda regulated drug: No
is fda regulated device: No
is us export: No
There are no clinically established treatments which have been proven to delay the disease
      progression in spinocerebellar ataxia (SCA) 3. Most available treatments are only for symptom
      alleviation, and thus the majority of patients will eventually progress to needing and wheel
      chair and eventually bedridden.

      As trehalose appear to be potentially promising treatment in SCA, the investigators aim to
      conduct this study using oral trehalose in our genetically confirmed SCA 3 patients.
This prospective single arm interventional study involved 13 genetically confirmed
      spinocerebellar ataxia (SCA) 3 patients with no concomitant diabetes, over 6 months.
      Following baseline assessment, patients were instructed to ingest 100g of oral trehalose
      diluted in 500ml of water or other beverages daily. Assessments were performed at baseline,
      2, 4 and 6 months using ataxia rating scales (SARA, SCAFI and INAS) and EQ-5D-3L scale for
      quality of life assessment.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: scale of rating of ataxia (SARA) score months,
time frame: 2 monthly intervals for 6 months
description: Assessment of SARA scores by a single assessor
measure: SCA Functional Index Scores
time frame: 2 monthly intervals for 6 months
description: Assessment of SCAFI by a single assessor
measure: EQ5D3L - quality of life scores
time frame: 2 monthly intervals for 6 months
description: Assessment of quality of life scores
measure: Side effects Profile
time frame: 2 monthly intervals for 6 months
description: Adverse Effects
measure: Blood investigation
time frame: At baseline and at 6 months
description: Measurement of renal profile, fasting blood glucose, full blood count and liver profile
arm group label: interventional
arm group type: Experimental
description: supplement: trehalose
intervention type: Dietary Supplement
intervention name: trehalose
description: patients were instructed to ingest 100g of oral trehalose diluted in 500ml of water or other beverages daily
arm group label: interventional
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: Accepts Healthy Volunteers
last name: NORLINAH MOHAMED IBRAHIM, MBBCH
role: Principal Investigator
affiliation: norlinah@ppukm.ukm.edu.my
facility: 
country: Malaysia
responsible party type: Principal Investigator
investigator affiliation: National University of Malaysia
investigator full name: Dr Norlinah Mohamed Ibrahim
investigator title: Professor
mesh term: Ataxia
mesh term: Cerebellar Ataxia
mesh term: Spinocerebellar Ataxias
mesh term: Spinocerebellar Degenerations
mesh term: Machado-Joseph Disease
sharing ipd: No