Acetylcholine Receptors From Human Muscles as Pharmacological Target for ALS

NCT02645461 | Completed

• 1 other identifier: 3314
• Study Type: interventional
• Target: 50
• Locations: 0 countries
• Sites: N/A

Brief Summary

Amyotrophic lateral sclerosis (ALS) is a fatal disease leading to motor neuron degeneration and progressive paralysis. Other studies have revealed defects in skeletal muscle even in absence of motor neuron anomalies, focusing on acetylcholine receptors (AChRs) and supporting the so-called "dying-back" hypothesis. Outcome of this study will be to understand if the endocannabinoid palmitoylethanolamide (PEA) can reduce the rundown of AChRs currents in ALS muscle, and if it can modify ALS patients' clinical and electrophysiological parameters.

Conditions

Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (1)

• Changes from baseline in pulmonary capacity of ALS patients at 6 months.

  Changes of the percentage of predicted forced vital capacity (FVC %) will be measured

  six months

Secondary Outcomes (3)

• Changes in acetylcholine receptors (AChR) currents and Analysis of the composition of AChRs subunits in ALS muscles.

  six months

• Changes from baseline in muscle strength of ALS patients at 6 months.

  six months

• Changes from baseline in electrophysiological parameters of ALS patients at 6 months

  six months

Study Arms (2)

Riluzole

ACTIVE COMPARATOR

Riluzole 50 mg twice daily in ALS patients

Drug: Riluzole

PEA plus Riluzole

EXPERIMENTAL

Riluzole 50 mg twice daily plus Endocannabinoid palmitoylethanolamide (PEA) (ultramicronized) 600 mg twice daily in ALS patients

Drug: endocannabinoid palmitoylethanolamide (PEA); Drug: Riluzole

Interventions

endocannabinoid palmitoylethanolamide (PEA) DRUG

Endocannabinoid palmitoylethanolamide (PEA) (ultramicronized) 600 mg twice daily

Also known as: PEA

PEA plus Riluzole

Riluzole DRUG

Riluzole 50 mg twice daily

PEA plus Riluzole; Riluzole

Eligibility Criteria

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

You may qualify if:

• Diagnosis of ALS according to the El-Escorial criteria;
• Age\> 18 years;
• ALS Functional Rating Scale-Revised (ALSFRS- r) score\> 20;
• Forced Vital Capacity (FVC)\> 30%;
• Treatment with Riluzole.

You may not qualify if:

• Other diseases motor neurons;
• Experimental treatments in the previous three months;
• Pregnant or breast-feeding;
• Contraindications to the use of riluzole;
• Patients undergoing tracheostomy, enteral or parenteral supply;
• Severe psychiatric disorders.

Sponsors & Collaborators

• University of Roma La Sapienza: lead

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

Riluzole

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)

Thiazoles; Sulfur Compounds; Organic Chemicals; Benzothiazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: BASIC SCIENCE
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associated Professor of Neurology

Study Record Dates

• First Submitted: December 22, 2015
• First Posted: January 1, 2016
• Study Start: January 1, 2014
• Primary Completion: June 1, 2015
• Study Completion: December 1, 2015
• Last Updated: January 5, 2016

Record last verified: 2016-01