Efficacy Of Oral Trehalose In Spinocerebellar Ataxia 3

NCT04399265 | Unknown

• 1 other identifier: DIP-2019-007
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

This study evaluates the effectiveness of oral trehalose in alleviating the neuropathological and motor behaviour deficits among patients with SCA3. A total of 40 participants with SCA3 will be recruited, with 20 participants to be administered with trehalose while another 20 participants to be administered with a maltose placebo.

Conditions

Spinocerebellar Ataxia Type 3

Keywords

Spinocerebellar ataxia Type 3; Cerebellar Cognitive and Affective Syndrome; Trehalose; Neuropsychology; Magnetic resonance imaging

Outcome Measures

Primary Outcomes (1)

• Changes from Baseline Scale for Ataxia Rating Assessment (SARA) at 3 months, 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.

  An 8-item scale to quantify the severity of ataxia with a scoring of 0 (no ataxia) to 40 (most severe ataxia). Total time taken for test administration is estimated at 10 minutes.

  Baseline, 3 months, and 6 months, as well as 3, 6, 9, and 12 months post-treatment follow-up.

Secondary Outcomes (8)

• Changes from Baseline Spinocerebellar Ataxia Functional Index (SCAFI) at 3 months and 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.

  Baseline, 3 months, and 6 months, as well as 3, 6, 9, and 12 months post-treatment follow-up.

• Changes from Baseline Inventory of Non-Ataxia Symptoms (INAS) at 3 months and 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.

  Baseline, 3 months, and 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.

• Changes from Baseline Cerebellar Cognitive & Affective Syndrome (CCAS) Scale at 3 months and 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.

  Baseline, 3 months, and 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.

• Changes from Baseline Wechsler Adult Intelligence Scale (WAIS - 4) at 3 months and 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.

  Baseline, 3 months, and 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.

• Changes from Baseline Delis-Kaplan Executive Function System (D - KEFS) at 3 months and 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.

  Baseline, 3 months, and 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.

Other Outcomes (4)

• Changes from Baseline Structural / T1 MRI at 3 months.

  Baseline and 3 months

• Changes from Baseline Situational Motivation Scale (SIMS) at 3 months and 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.

  Baseline, 3 months, and 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.

• Changes from Baseline EuroQol-5D 3L (EQ-5D-3L) at 3 months and 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.

  Baseline, 3 months, and 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.

Study Arms (2)

Trehalose

EXPERIMENTAL

Trehalose powder form to be dissolved in water, to be consumed by mouth, every day for 3 months.

Dietary Supplement: Trehalose

Maltose placebo

PLACEBO COMPARATOR

Isocaloric maltose powder form to be dissolved in water, to be consumed by mouth, every day for 3 months.

Dietary Supplement: Maltose

Interventions

Trehalose DIETARY_SUPPLEMENT

A disaccharide sugar usually used as food preservative, but for this study it is given at a higher dose

Trehalose

Maltose DIETARY_SUPPLEMENT

Maltose power

Maltose placebo

Eligibility Criteria

• Age: 18 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age between 18 and 85 years old.
• Genetically confirmed SCA 3 diagnosis.
• Presence of progressive cerebellar syndrome (i.e. symptomatic) and genetic confirmation of SCA 3 in one of the immediate family members with similar clinical syndrome.
• Able to read, speak, and understand English or Malay.

You may not qualify if:

• Diagnosis of Diabetes Mellitus Type 1 or Type 2
• Presence of any concomitant neurological condition that might interfere with clinical measures used in this study.
• Presence of contraindication or hypersensitivity to trehalose.
• Use of stimulant / medication, caffeine, and tobacco within less than 24 hours washout period prior to assessment and / or scan.
• Presence of severe visual and / or auditory perceptual deficits.
• MRI contra-indications: claustrophobia, pregnancy, electronic implants (e.g. pacemaker) in the body, aneurysm clip, and current or past employment as machinists, welders or metal workers.

Sponsors & Collaborators

• National University of Malaysia: lead
• Radboud University Medical Center: collaborator

Study Sites (1)

UKM Medical Centre

Cheras, Kuala Lumpur, 56000, Malaysia

RECRUITING

MeSH Terms

Conditions

Machado-Joseph Disease; Cerebellar Cognitive Affective Syndrome

Interventions

Trehalose; Maltose

Condition Hierarchy (Ancestors)

Spinocerebellar Ataxias; Cerebellar Ataxia; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinocerebellar Degenerations; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Ataxia; Dyskinesias; Neurologic Manifestations; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Glucans; Polysaccharides; Carbohydrates; Disaccharides; Oligosaccharides; Sugars

Study Officials

• Norlinah Mohamed Ibrahim, MRCP

  National University of Malaysia

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Kah Hui Yap, Master

CONTACT

+60165536721; kahhui0411@gmail.com

Norlinah Mohamed Ibrahim, MRCP

CONTACT

+60122145306; norlinah@ppukm.ukm.edu.my

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, OUTCOMES ASSESSOR
• Masking Details: Patients with SCA 3 will be randomised into the active treatment group and placebo group to which they are blinded from their group allocation. Likewise, the outcome assessor will be blinded from the patient's group status. A partial unblinding will be made with Group A vs. Group B (rather than the actual grouping) for preliminary analysis on Visit 1 and Visit 2.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Model Details: This study will employ a double-blind randomised controlled trial (RCT) with two arms. Patients with SCA3 will be randomly allocated to oral trehalose group and a maltose placebo group. A total of 20 participants will be allocated to each group. There is a possibility of including other types of SCA at later stage of the study.

Study Record Dates

• First Submitted: March 11, 2020
• First Posted: May 22, 2020
• Study Start: February 24, 2020
• Primary Completion: May 30, 2023
• Study Completion: June 30, 2023
• Last Updated: August 16, 2022

Record last verified: 2022-08

Data Sharing

• IPD Sharing: Will not share

Locations

MY (1)