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Clinical Effects of Oral Trehalose In Patients With Spinocerebellar Ataxia 3

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ClinicalTrials.gov Identifier: NCT04426149
Recruitment Status : Completed
First Posted : June 11, 2020
Last Update Posted : June 11, 2020
Sponsor:
Information provided by (Responsible Party):
Dr Norlinah Mohamed Ibrahim, National University of Malaysia

Brief Summary:

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.


Condition or disease Intervention/treatment Phase
Spinocerebellar Ataxia 3 Dietary Supplement: trehalose Not Applicable

Detailed Description:
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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 13 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Clinical Effects of Oral Trehalose In Patients With Spinocerebellar Ataxia 3: A Pilot Study
Actual Study Start Date : March 7, 2018
Actual Primary Completion Date : September 7, 2018
Actual Study Completion Date : September 7, 2018


Arm Intervention/treatment
Experimental: interventional
supplement: trehalose
Dietary Supplement: trehalose
patients were instructed to ingest 100g of oral trehalose diluted in 500ml of water or other beverages daily




Primary Outcome Measures :
  1. scale of rating of ataxia (SARA) score months, [ Time Frame: 2 monthly intervals for 6 months ]
    Assessment of SARA scores by a single assessor

  2. SCA Functional Index Scores [ Time Frame: 2 monthly intervals for 6 months ]
    Assessment of SCAFI by a single assessor

  3. EQ5D3L - quality of life scores [ Time Frame: 2 monthly intervals for 6 months ]
    Assessment of quality of life scores


Secondary Outcome Measures :
  1. Side effects Profile [ Time Frame: 2 monthly intervals for 6 months ]
    Adverse Effects

  2. Blood investigation [ Time Frame: At baseline and at 6 months ]
    Measurement of renal profile, fasting blood glucose, full blood count and liver profile



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

Inclusion Criteria:

  1. DNA diagnosis of SCA 3 in the study subject of his/ her affected family member(s)
  2. Consent to participate in the study
  3. The age of 18 years and older

Exclusion Criteria:

  1. Unconfirmed SCA 3
  2. Concomitant disorder(s) that affect SARA and other ataxia measures used in this study
  3. Diabetes
  4. Malabsorption of trehalose underlies intolerance to mushrooms, since the lack of absorption results in diarrhoea and intestinal distress.
  5. Less than 18 years old

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04426149


Locations
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Malaysia
Pusat Perubatan Universiti Kebangsaan Malaysia
Kuala Lumpur, Malaysia, 56000
Sponsors and Collaborators
National University of Malaysia
Investigators
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Principal Investigator: NORLINAH MOHAMED IBRAHIM, MBBCH norlinah@ppukm.ukm.edu.my
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Responsible Party: Dr Norlinah Mohamed Ibrahim, Professor, National University of Malaysia
ClinicalTrials.gov Identifier: NCT04426149    
Other Study ID Numbers: UKM PPI/111/8/JEP-2017-826
First Posted: June 11, 2020    Key Record Dates
Last Update Posted: June 11, 2020
Last Verified: June 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
Additional relevant MeSH terms:
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Ataxia
Cerebellar Ataxia
Spinocerebellar Ataxias
Spinocerebellar Degenerations
Machado-Joseph Disease
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn