Amyotrophic Lateral SclerosisAchALSNCT02645461
Acetylcholine Receptors From Human Muscles as Pharmacological Target for ALS
A study testing endocannabinoid palmitoylethanolamide (PEA) and Riluzole in people with Amyotrophic Lateral Sclerosis, run by University of Roma La Sapienza.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- endocannabinoid palmitoylethanolamide (PEA) and Riluzole
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- No sites listed yet
- Study dates
- Jan 2014to Dec 2015
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
About this study
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.
Read the 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.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- endocannabinoid palmitoylethanolamide (PEA), Riluzole
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
endocannabinoid palmitoylethanolamide (PEA)Drug
Also called PEA
Endocannabinoid palmitoylethanolamide (PEA) (ultramicronized) 600 mg twice daily
RiluzoleDrug
Riluzole 50 mg twice daily
| Study group | Role | What they receive |
|---|---|---|
| Riluzole | Active comparator | Riluzole 50 mg twice daily in ALS patients Receives: Drug: Riluzole |
| PEA plus Riluzole | Experimental | Riluzole 50 mg twice daily plus Endocannabinoid palmitoylethanolamide (PEA) (ultramicronized) 600 mg twice daily in ALS patients Receives: Drug: endocannabinoid palmitoylethanolamide (PEA); Drug: Riluzole |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: six months
Secondary outcomes (3)
Changes in acetylcholine receptors (AChR) currents and Analysis of the composition of AChRs subunits in ALS muscles.
Utilization of voltage-clamp intracellular recordings in oocytes transplanted with membranes from ALS muscles.
Measured: six months
Changes from baseline in muscle strength of ALS patients at 6 months.
Changes of the Medical Research Council (MRC) scale score will be measured
Measured: six months
Changes from baseline in electrophysiological parameters of ALS patients at 6 months
Changes of the compound muscle action potential (CMAP) amplitude of ulnar and phrenic nerves will be measured
Measured: six months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jan 2016 (estimated)
- Study startJan 2014
- Primary completionJun 2015
- Study completionDec 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- More trials run by University of Roma La SapienzaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 50 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Roma La Sapienza
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Maurizio Inghilleri, University of Roma La Sapienza
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02645461
- Sponsor's protocol ID
- 3314
- Acronym
- AchALS
- Status last verified
- Jan 2016
- First submitted
- 22 Dec 2015
- Last updated
- 5 Jan 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Jan 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Acetylcholine Receptors From Human Muscles as Pharmacological Target for ALS
- ClinicalTrials.gov ID
- NCT02645461
- Acronym / sponsor ID
- AchALS / 3314
- Conditions
- Amyotrophic Lateral Sclerosis
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 50 participants (actual)
- Lead sponsor
- University of Roma La SapienzaUniversity, hospital or other
- Study start
- Jan 2014
- Primary completion
- Jun 2015 (actual)
- Study completion
- Dec 2015 (actual)
- First posted
- 1 Jan 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Riluzole | Active comparator | Drug: RiluzoleRiluzole 50 mg twice daily in ALS patients |
| PEA plus Riluzole | Experimental | Drug: endocannabinoid palmitoylethanolamide (PEA), Drug: RiluzoleRiluzole 50 mg twice daily plus Endocannabinoid palmitoylethanolamide (PEA) (ultramicronized) 600 mg twice daily in ALS patients |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (6 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| endocannabinoid palmitoylethanolamide (PEA)Also: PEAEndocannabinoid palmitoylethanolamide (PEA) (ultramicronized) 600 mg twice daily | Drug | PEA plus Riluzole |
| RiluzoleRiluzole 50 mg twice daily | Drug | PEA plus Riluzole, Riluzole |
Primary outcome
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
3 secondary outcomes
Changes in acetylcholine receptors (AChR) currents and Analysis of the composition of AChRs subunits in ALS muscles.
Time frame: six months
Changes from baseline in muscle strength of ALS patients at 6 months.
Time frame: six months
Changes from baseline in electrophysiological parameters of ALS patients at 6 months
Time frame: six months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Jan 2016.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A study testing endocannabinoid palmitoylethanolamide (PEA) and Riluzole in people with Amyotrophic Lateral Sclerosis, run by University of Roma La Sapienza.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 5 Jan 2016. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Changes from baseline in pulmonary capacity of ALS patients at 6 months. (time frame: six months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Jan 2016.