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NCT Number: NCT04426149

Clinical Effects of Oral Trehalose In Patients With Spinocerebellar Ataxia 3

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.

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Key information

Conditions

Spinocerebellar Ataxia 3 Ataxia Brain Diseases Central Nervous System Diseases Cerebellar Ataxia Cerebellar Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dyskinesias Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Machado-Joseph Disease Nervous System Diseases Neurodegenerative Diseases Neurologic Manifestations Spinal Cord Diseases Spinocerebellar Ataxias Spinocerebellar Degenerations

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Pusat Perubatan Universiti Kebangsaan Malaysia

Kuala Lumpur, 56000, Malaysia

About this study

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.

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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

Exclusion criteria

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

Treatment and study plan

Trehalose

Dietary Supplement

patients were instructed to ingest 100g of oral trehalose diluted in 500ml of water or other beverages daily

Primary outcomes

  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 outcomes

  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

Sponsors and collaborators

Lead sponsor

National University of Malaysia

Other

Registry information

Official study title

Clinical Effects of Oral Trehalose In Patients With Spinocerebellar Ataxia 3: A Pilot Study

Important dates

Study start
2018
Primary completion
2018
Study completion
2018
First posted
Jun 11, 2020
Registry last updated
Jun 11, 2020

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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