Acetylcholine Receptors From Human Muscles as Pharmacological Target for ALS

Acetylcholine Receptors From Human Muscles as Pharmacological Target for ALS

Sponsors

Lead Sponsor: University of Roma La Sapienza

Source University of Roma La Sapienza
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.

Detailed Description

Outcome: Monitoring the efficacy and safety of PEA in the treatment of patients with ALS. Analysis of AChR currents and description of the composition of AChRs subunits in ALS muscles Design of the Study: A randomized controlled blinded study. Patients with sporadic ALS will receive riluzole alone or riluzole+PEA in order to investigate the clinical and electrophysiological effects of treatment. The expected number of enrolled patients will be 50. All patients satisfying the selection criteria will be randomized into two groups: a first group will be treated only with riluzole, the second group with riluzole associated with PEA (Normast 600 mg microgranular, 2 sachets/day). The randomization will be done stratifying patietns according to type of clinical onset (bulbar vs. spinal). The patients will be enrolled in the Department of Neurology and Psychiatry, University of Rome "Sapienza". The visits will be performed at 0 (randomization), 3 and 6 months. At each visit the ALS Functional Rating Scale-Revised (ALSFRS-R), the percentage of predicted forced vital capacity (FVC%), the Medical Research Council (MRC) score for muscle strength limited to the right upper limbs, and the compound muscle action potentials (CMAP) from right ulnar and phrenic nerves will be assessed. A muscle biopsy will be done at the end of the study. The obtained results will be compared with those observed in muscle samples from denervated (non-ALS) control patients.

Overall Status Completed
Start Date 2014-01-01
Completion Date 2015-12-01
Primary Completion Date 2015-06-01
Phase N/A
Study Type Interventional
Primary Outcome
Measure Time Frame
Changes from baseline in pulmonary capacity of ALS patients at 6 months. six months
Secondary Outcome
Measure Time Frame
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
Enrollment 50
Condition
Intervention

Intervention Type: Drug

Intervention Name: endocannabinoid palmitoylethanolamide (PEA)

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

Arm Group Label: PEA plus Riluzole

Other Name: PEA

Intervention Type: Drug

Intervention Name: Riluzole

Description: Riluzole 50 mg twice daily

Eligibility

Criteria:

Inclusion Criteria: - 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. Exclusion Criteria: - 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.

Gender:

All

Minimum Age:

18 Years

Maximum Age:

N/A

Healthy Volunteers:

No

Verification Date

2016-01-01

Responsible Party

Type: Principal Investigator

Investigator Affiliation: University of Roma La Sapienza

Investigator Full Name: Maurizio Inghilleri

Investigator Title: Associated Professor of Neurology

Has Expanded Access No
Condition Browse
Number Of Arms 2
Arm Group

Label: Riluzole

Type: Active Comparator

Description: Riluzole 50 mg twice daily in ALS patients

Label: PEA plus Riluzole

Type: Experimental

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

Acronym AchALS
Study Design Info

Allocation: Randomized

Intervention Model: Parallel Assignment

Primary Purpose: Basic Science

Masking: Double (Participant, Investigator)

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