The Efficacy and Safety of Vitamin E Mixed Tocotrienols In Patients With Amyotrophic Lateral Sclerosis (ALS)

Phase 2

• Conditions: Amyotrophic Lateral Sclerosis
• Interventions: Dietary Supplement: Tocotrienols; Dietary Supplement: Placebo

• Registration Number: NCT04140136
• Lead Sponsor: University of Malaya

Brief Summary

There is currently no effective treatment in ALS. Oxidative stress, probably interacting with other neurodegenerative processes, is hypothesized to play a leading role in pathogenesis. These include mechanisms that promote glutamate excitotoxicity, mitochondrial dysfunction and axonal dysfunction.

In a transgenic mouse model of fALS that develops a disease with a clinical phenotype similar to ALS, dietary vitamin E supplementation delayed disease onset and slowed progression, although it did not prolong survival. When used as an experimental therapy in human trials, vitamin E did not affect survival significantly, but possibly slowed ALS progression. Two large, prospective epidemiologic studies suggest that longterm use of vitamin E supplements could be inversely associated with risk of ALS or ALS death. In another study, higher baseline serum α-tocopherol was associated with lower subsequent risk of ALS. A modest, non-significant protective effect from supplementation was seen in subjects with baseline serum α-tocopherol levels below median levels. In the current study, we aim to investigate the effects of tocotrienols in patients with ALS, particularly in delaying disease progression as well as assessing its safety profile in this group of patients.

Detailed Description

Not available

Study Timeline

• Study Start: 2019-06-17
• Status Verified: 2019-10
• Study First Submitted: 2019-10-21
• Study First Submitted QC: 2019-10-23
• Last Update Submitted: 2019-10-23
• Study First Posted: 2019-10-25
• Last Update Posted: 2019-10-25
• Primary Completion: 2020-04-01
• Study Completion: 2020-09-01

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

• Patients who have a decrease of 1 to 4 points on the ALSFRS-R score during the 12-weeks observation period prior to screening and enrollment
• Patients of less than 2 years after the diagnosis of ALS.
• Patients without respiratory symptoms (orthopnea, dyspnea)
• Capable of giving signed informed consent

Exclusion Criteria

• Patients who have developed respiratory failure necessitating ventilation
• Patients who have developed unsafe swallowing necessitating enteral feeding tube insertion
• Patients with other neurodegenerative disease such as Parkinson's disease and significant mental health illness
• Patients with certain concomitant diseases which may affect the assessment of safety/efficacy i.e. malignancy within the last 5 years, congestive heart disease, liver disease, kidney failure, bleeding disorders and other autoimmune diseases etc.
• Pregnant, lactating, and probably pregnant patients.
• Patients taking vitamin E tocopherol or tocotrienols supplements within 1 month from screening and randomisation.
• Patients who have participated in other trials within 12 weeks before consent, or who are participating in other clinical trials at present.
• Women of child bearing potential or nursing mother, unless they are willing to practice effective contraceptive measures.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Treatment group	Tocotrienols	This is a pilot randomized, double-blind, placebo-controlled, parallel-group study to evaluate the effects of 24-weeks tocotrienols (Tocovid Suprabio) supplementation in ALS patients.The investigational product is to be administered twice daily, at a dose of 400mg per day.
Placebo group	Placebo	This is a pilot randomized, double-blind, placebo-controlled, parallel-group study to evaluate the effects of 24-weeks tocotrienols (Tocovid Suprabio) supplementation in ALS patients. The placebo is similar in appearance but does not contain tocotrienols and consist of palm oil.

Primary Outcome Measures

Name	Time	Method
Mean change of revised ALS Functional Rating Scale (ALSFRS-R) at baseline and 6 months between treatment group difference.	6 months	Efficacy of oral mixed Tocotrienols in patients with ALS in delaying disease progression as assess based on the cumulative score of ALSFRS-R between treatment group difference at specific time point.

Secondary Outcome Measures

Name	Time	Method
Number of participants with treatment-related adverse events,haematological,renal and liver profile monitored at every visit	6 months	Safety of oral mixed Tocotrienols in ALS patients

Trial Locations

Locations (1)

Clinical Investigation Centre (CIC); 🇲🇾 Kuala Lumpur, Malaysia