<p>The National ALS Clinic and Research Group based at Beaumont Hospital Dublin provides a multidisciplinary service to the entire Irish ALS population.</p>
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Professor of Neurology

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Professor Hardiman trained in Neurology in Boston prior to her appointment as [&hellip;]

Orla Hardiman - TRICALS

Associate Professor and Consultant Neurologist

Professor Bede is a consultant neurologist specialised in neuromuscular disorders and neurodegenerative [&hellip;]

Peter Bede - TRICALS

Research Fellow

Dr. Deirdre Murray MISCP (PhD) is a Research Fellow in Trinity College [&hellip;]

Deirdre Murray - TRICALS

Ussher Assistant Professor

Russell McLaughlin graduated from University of Bristol in 2005 with a BSc [&hellip;]

Russell McLaughlin - TRICALS

Our Specialists

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<h2>Dazucorilant</h2>
Unbalanced levels of cortisol (a stress hormone) have been reported in people with ALS. Long exposure to higher levels of cortisol may increase inflammation in the nervous system. Dazucorilant is a small molecule that modulates cortisol activity which may reduce the neurotoxic effects of prolonged cortisol exposure. Therefore, it may slow functional loss and disease progression.
<h2>Study design</h2>
DAZALS is a phase 2 double-blind, randomized, placebo-controlled study. This means that the efficacy of dazucorilant is compared to a placebo. The placebo looks exactly like dazucorilant but contains no active drug. Participants will be randomly assigned to either the placebo or dazucorilant. You will have a two-thirds (2/3) chance of receiving dazucorilant. Neither you nor your study doctor or the study staff will know which treatment you are receiving.

During the study there will be 10 research visits over a period of 28 weeks. 7 visits will take place at the research centre and 3 visits will be by telephone. At the last visit in week 24, you can choose to continue with dazucorilant as part of an ‘open-label’ study period. You will then continue visits both at the research centre and by telephone for up to 140 weeks. If you do not wish to participate in the open-label extension or are not eligible, you will be asked to continue study visits over the phone for up to 132 weeks.

The study drug (refers to both dazucorilant and placebo) is a softgel capsule packaged in a blister card. Study drug (4 capsules in total) should be taken by mouth once a day with food and 240 ml water, at approximately the same time each day.
<h2>Can I participate in the study?</h2>
The main criteria to participate in this study are:
<ul>
 	<li>You are diagnosed with ALS.</li>
 	<li>You are able to swallow capsules.</li>
 	<li>If you are taking an approved treatment for ALS, you must be on a stable dose of that treatment prior to entering this study.</li>
</ul>
<h2>Registration and more information</h2>
If you are interested in this study and fit the above criteria, please contact one of the participating TRICALS centres below (contact details can be found by clicking on ‘read more’). They can provide you with more information and discuss any questions you may have. If you experience difficulty contacting a centre, you may reach out directly to the TRICALS operational team at info@tricals.org for support in this process.

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DAZALS trial

Industry trial

<h3>ION363</h3>
The study drug (ION363) is designed to target the mutated FUS gene, which makes FUS protein. When FUS protein builds up in motor neurons, it may contribute to ALS. The study drug is designed to stop the FUS gene from making this protein, which may improve symptoms of ALS.
<h3>Study design</h3>
The FUSION trial is a phase 1-3 double-blind, randomized, placebo-controlled study. This means that the efficacy of ION363 is compared to a placebo. A placebo is a drug without an active ingredient. In Part 1 of the study, particpants will be randomly assigned to either the placebo or ION363. You will have a 2/3 chance of receiving ION363 in the first part of the study. Neither you nor your study doctor or the study staff will know which treatment you are receiving during that time. All participants will then have the opportunity to receive ION363 in Part 2 of the study.

You will receive the study drug or placebo as an injection through the spinal canal. The injection will take 1 to 3 minutes. You may receive local anesthesia to make the injection more comfortable.

The study consists of two parts. The total study duration is 3 years and 11 months. Part 1 of the study involves up to 2 weeks of a screening period, approximately 14 months for treatment (ION363 or placebo) and 3 months of post-treatment follow-up. During the treatment period, if your condition worsens significantly, you may be eligible to change early into Part 2 of the study where everyone receives ION363.

After completion of Part 1, you can continue to Part 2 which includes up to 20 months of treatment with ION363 followed by 9 months of post-treatment follow-up. After Part 2 of the study, you may continue to receive injections of ION363 for up to 3 additional years if the drug is not commercially available.

During the study there will be approximately 20 visits (9 visits for Part 1 and 11 visits for Part 2). The length of your visits will vary. One visit will require overnight stay and visits when the study drug is being administered will require at least 6 hours at the study site. You might have to stay overnight at the study site if your study doctor feels that it is best to observe you overnight for your safety.
<h3>Can I participate in the study?</h3>
The main criteria to participate in this study are:
<ul>
 	<li>You are at least 12 years old</li>
 	<li>You have ALS with a mutation in the FUS gene</li>
</ul>
<h3>Registration and more information</h3>
If you are interested in participating in the FUSION trial and fit the above profile, please contact one of the participating tricals centres below (contact details can be found by clicking on ‘read more’). They can provide you with more information and answer any questions you may have.

FUSION trial

<h2><strong>AMX0035</strong></h2>
AMX0035 consists of a combination of two molecules: tauroursodeoxycholic acid (TUDCA) and sodium phenylbutyrate. Both were previously shown to be safe and well tolerated. The combination of the two drugs is believed to decrease nerve cell damage in people living with ALS.
<h2><strong>Study overview</strong></h2>
This phase 3 study is a double-blind, randomized, placebo-controlled study. This means that we compare the effect of AMX0035 against the effect of a placebo. A placebo is a drug without an active ingredient, a 'fake drug'. The study drug and placebo are supplied in a sachet and must be dissolved in 1 cup of water. Either treatment should be taken orally (ingested) daily for 48 weeks. Participants will be randomly assigned to receive the placebo or AMX0035 treatment. The participant and the research team do not know which treatment the participant is receiving.

During a period of 1 year, participants will visit the research centre approximately 6 times for examinations. They will furthermore be contacted approximately 9 times by telephone or video calling.
<h2><strong>Key Eligibility Criteria</strong></h2>
The main criteria to participate in this study are:
<ul>
 	<li>You are diagnosed with ALS</li>
 	<li>The duration of illness from the first symptoms is no longer than 24 months</li>
 	<li>Your lung function is at least 55%, without using a ventilator</li>
 	<li>You must not be pregnant or breast-feeding for the entire duration of the study</li>
 	<li>If you have or are considering having diaphragm pacing during the study, you cannot participate</li>
 	<li>When using Riluzole: a stable dose of ≥30 days prior to participation</li>
</ul>
Participants can continue their treatment with Riluzole for the entire duration of the study.

Amylyx is looking to enrol 600 participants worldwide. Currently, the study is active in the following European countries: the United Kingdom, Ireland, the Netherlands, Spain, Sweden, Italy, France, Poland, Belgium, Germany and Portugal.
<h2><strong>More information</strong></h2>
More information about the PHOENIX trial can be found on the website of <a href="https://www.amylyxalstrial.com" target="_blank" rel="noopener">Amylyx</a> and <a href="https://clinicaltrials.gov/ct2/show/NCT05021536" target="_blank" rel="noopener noreferrer">here</a>.

Inclusion for this trial is closed.

PHOENIX trial

<h2><strong>FNP122</strong></h2>
Oxidative stress is thought to contribute to nerve cell death in ALS. FNP122 is an oral formulation of edaravone, a compound that can help prevent oxidative stress. Therefore, by decreasing oxidative stress, edaravone could potentially slow ALS progression.
<h2><strong>Study design</strong></h2>
The ADORE trial is <a href="https://www.ferrer.com/en/node/3250">sponsored</a> by the pharmaceutical company Ferrer. It is a randomised, double-blind, placebo-controlled trial. This means that the effect of FNP122 is compared against a placebo treatment. A placebo is something that looks exactly the same as the study medicine but does not have any active medicine in it. Participants will be randomly assigned to receive FNP122 or placebo. Neither the participant nor the study team will be told which group they have been placed into until after the study has finished.

Both placebo and FNP122 need to be dissolved in water and ingested daily. The study will run for up to 48 weeks. Participants will need to visit the clinic at various occasions:
<ul>
 	<li>For a screening appointment</li>
 	<li>For a baseline visit</li>
 	<li>At week 4</li>
 	<li>At week 12</li>
 	<li>Every 12 weeks thereafter</li>
</ul>
Monthly telephone appointments will take place in between the visits to the clinic until Week 48.
<h2><strong>Inclusion criteria</strong></h2>
Key eligibility criteria for people with ALS to participate, include:
<ul>
 	<li>Age between 18 – 80 years</li>
 	<li>Diagnosis of ALS</li>
 	<li>Disease onset within the prior 24 months</li>
 	<li>SVC equal to or more than 70% at screening visit</li>
 	<li>Change in ALSFRS-R score between 0.35 points and 1.5 points per month (both inclusive) in the period from onset of first symptoms to the Screening visit;</li>
 	<li>Patients on riluzole should be on stable doses ≥30 days prior to the baseline visit and this dose should be maintained during the entire trial.</li>
 	<li>Female participants should not be (able to become) pregnant or breast-feeding</li>
</ul>
Participants can continue their treatment with Riluzole for the entire duration of the study.

The study aims to enrol 300 patients across European countries. Several TRICALS Centres will be <a href="https://www.tricals.org/en/news/adore-trial-enrolls-first-patient/">participating</a> in this trial. Currently, the study is active in the United Kingdom, Ireland, the Netherlands, Belgium, Poland, Italy, Germany, Spain, France and Sweden.

Inclusion for this trial is closed.

ADORE trial

TUDCA (tauroursodeoxycholic acid) is a small molecule that is being explored for its potential as a treatment for amyotrophic lateral sclerosis (ALS). TUDCA may decrease nerve cell death through its ability to act as an antioxidant that prevents toxic reactive oxygen from accumulating inside cells. There may also be an effect on immune function.
<h3>Study setup</h3>
TUDCA-ALS is a randomized, double-blind, placebo-controlled study. This means the effect of TUDCA is tested by comparing a group of patients that receive TUDCA to a group of patients receiving a placebo-drug. Patients will be randomized to one of two treatment arms. During the study, both the patient as the research team do not know who is receiving placebo or TUDCA. This will help researchers to assess potential side- and treatment effects of TUDCA.

TUDCA and the placebo capsules are ingested twice daily, 10-15 minutes after a meal. Patients will also be taking riluzole. The study duration is 18 months and clinical assessments during the trial phase will be performed every three months. This will allow measuring if the drug improves disease progression rate.
<h3><b>Would you like to participate?</b></h3>
Key eligibility criteria for people with ALS to participate, include:
<ul>
 	<li>Between 18 and 80 years old</li>
 	<li>Diagnosed with ALS</li>
 	<li>Disease duration ≤ 18 months</li>
 	<li>No swallowing difficulty</li>
 	<li>Forced vital capacity ≥70% of predicted values</li>
 	<li>On a stable dose of riluzole for at least 3 months</li>
</ul>

TUDCA-ALS

Investigator initiated trial

Current trials

Orla Hardiman

National ALS Clinic and Research Group, Beaumont Hospital Dublin/Academic Unit of Neurology

Trinity College Dublin

The National ALS Clinical and Research Group undertakes research in epidemiology, cognition, [&hellip;]

Trinity College Dublin - TRICALS

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