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

NCT04140136 | Unknown Phase 2 DMC

• 1 other identifier: 2018613-6392
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

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.

Conditions

Amyotrophic Lateral Sclerosis

Keywords

ALS

Outcome Measures

Primary Outcomes (1)

• Mean change of revised ALS Functional Rating Scale (ALSFRS-R) at baseline and 6 months between treatment group difference.

  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.

  6 months

Secondary Outcomes (1)

• Number of participants with treatment-related adverse events,haematological,renal and liver profile monitored at every visit

  6 months

Study Arms (2)

Treatment group

ACTIVE COMPARATOR

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.

Dietary Supplement: Tocotrienols

Placebo group

PLACEBO COMPARATOR

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.

Dietary Supplement: Placebo

Interventions

Tocotrienols DIETARY_SUPPLEMENT

Tocotrienols as a potent antioxidant to modulate oxidative stress and inflammatory response

Treatment group

Placebo DIETARY_SUPPLEMENT

The placebo is similar in appearance, but does not contain tocotrienols and consist of palm oil

Placebo group

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• University of Malaya: lead

Study Sites (1)

Clinical Investigation Centre (CIC)

Kuala Lumpur, 59100, Malaysia

RECRUITING

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

Tocotrienols

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Vitamin E; Benzopyrans; Pyrans; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Central Study Contacts

Piek Lian Wang

CONTACT

+603-79492622; wangpl@ummc.edu.my

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: October 21, 2019
• First Posted: October 25, 2019
• Study Start: June 17, 2019
• Primary Completion: April 1, 2020
• Study Completion: September 1, 2020
• Last Updated: October 25, 2019

Record last verified: 2019-10

Locations

MY (1)