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Acetylcholine Receptors From Human Muscles as Pharmacological Target for ALS (AchALS)

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ClinicalTrials.gov Identifier: NCT02645461
Recruitment Status : Completed
First Posted : January 1, 2016
Last Update Posted : January 5, 2016
Sponsor:
Information provided by (Responsible Party):
Maurizio Inghilleri, 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.

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Drug: endocannabinoid palmitoylethanolamide (PEA) Drug: Riluzole Not Applicable

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 50 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Basic Science
Official Title: Acetylcholine Receptors From Human Muscles as Pharmacological Target for ALS
Study Start Date : January 2014
Actual Primary Completion Date : June 2015
Actual Study Completion Date : December 2015


Arm Intervention/treatment
Active Comparator: Riluzole
Riluzole 50 mg twice daily in ALS patients
Drug: Riluzole
Riluzole 50 mg twice daily

Experimental: PEA plus Riluzole
Riluzole 50 mg twice daily plus Endocannabinoid palmitoylethanolamide (PEA) (ultramicronized) 600 mg twice daily in ALS patients
Drug: endocannabinoid palmitoylethanolamide (PEA)
Endocannabinoid palmitoylethanolamide (PEA) (ultramicronized) 600 mg twice daily
Other Name: PEA

Drug: Riluzole
Riluzole 50 mg twice daily




Primary Outcome Measures :
  1. Changes from baseline in pulmonary capacity of ALS patients at 6 months. [ Time Frame: six months ]
    Changes of the percentage of predicted forced vital capacity (FVC %) will be measured


Secondary Outcome Measures :
  1. Changes in acetylcholine receptors (AChR) currents and Analysis of the composition of AChRs subunits in ALS muscles. [ Time Frame: six months ]
    Utilization of voltage-clamp intracellular recordings in oocytes transplanted with membranes from ALS muscles.

  2. Changes from baseline in muscle strength of ALS patients at 6 months. [ Time Frame: six months ]
    Changes of the Medical Research Council (MRC) scale score will be measured

  3. Changes from baseline in electrophysiological parameters of ALS patients at 6 months [ Time Frame: six months ]
    Changes of the compound muscle action potential (CMAP) amplitude of ulnar and phrenic nerves will be measured



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

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Responsible Party: Maurizio Inghilleri, Associated Professor of Neurology, University of Roma La Sapienza
ClinicalTrials.gov Identifier: NCT02645461    
Other Study ID Numbers: 3314
First Posted: January 1, 2016    Key Record Dates
Last Update Posted: January 5, 2016
Last Verified: January 2016
Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases
Palmidrol
Acetylcholine
Riluzole
Anticonvulsants
Excitatory Amino Acid Antagonists
Excitatory Amino Acid Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Physiological Effects of Drugs
Neuroprotective Agents
Protective Agents
Vasodilator Agents
Cholinergic Agonists
Cholinergic Agents
Analgesics
Sensory System Agents
Peripheral Nervous System Agents
Anti-Inflammatory Agents, Non-Steroidal
Analgesics, Non-Narcotic
Anti-Inflammatory Agents