Clinical Effects of Oral Trehalose In Patients With Spinocerebellar Ataxia 3

• Sponsor: National University of Malaysia (Other)
• Overall Status: Completed
• CT.gov ID: NCT04426149
• Collaborator: (none)
• Enrollment: 13
• Location: 1
• Arm: 1
• Actual Duration (Months): 6
• Patients Per Site Per Month: 2.2

Study Details

Study Description

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	N/A

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.

Study Design

• Study Type: Interventional
• 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: Mar 7, 2018
• Actual Primary Completion Date: Sep 7, 2018
• Actual Study Completion Date: Sep 7, 2018

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures

1. scale of rating of ataxia (SARA) score months, [2 monthly intervals for 6 months]

   Assessment of SARA scores by a single assessor

2. SCA Functional Index Scores [2 monthly intervals for 6 months]

   Assessment of SCAFI by a single assessor

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

   Assessment of quality of life scores

Secondary Outcome Measures

1. Side effects Profile [2 monthly intervals for 6 months]

   Adverse Effects

2. Blood investigation [At baseline and at 6 months]

   Measurement of renal profile, fasting blood glucose, full blood count and liver profile

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: Yes

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Pusat Perubatan Universiti Kebangsaan Malaysia	Kuala Lumpur		Malaysia	56000

Sponsors and Collaborators

• National University of Malaysia

Investigators

• Principal Investigator: NORLINAH MOHAMED IBRAHIM, MBBCH, [e-mail]

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• 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: Jun 11, 2020
• Last Update Posted: Jun 11, 2020
• Last Verified: Jun 1, 2020

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• 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: Ataxia; Cerebellar Ataxia; Spinocerebellar Ataxias; Spinocerebellar Degenerations; Machado-Joseph Disease

Study Results

No Results Posted as of Jun 11, 2020