<p>Being the referral center in the Valencian region, the MND Unit of the Hospital Universitari i Polit&egrave;cnic La Fe follows 150 patients per year.</p>



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Juan Francisco V&aacute;zquez Costa obtained his degree in Medicine from the University [&hellip;]

Juan Francisco Vázquez Costa - 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

<h2>Utreloxastat</h2>
Nerve cells that control muscle movement, called motor neurons, are lost in ALS, leading to symptoms including muscle weakness. One mechanism involved with motor neuron loss is ferroptosis. Ferroptosis is a form of cell-death due to accumulation of toxic levels of iron that results from a key enzymatic pathway that becomes dysfunctional in ALS. Utreloxastat (also known as PTC857) is an oral small molecule with a mechanism of action that targets and inhibits activity of that key enzyme and is believed to decrease nerve cell damage in people living with ALS.
<h2>Study design</h2>
<p style="font-weight: 400;">If you qualify and agree to be in the study, you will be in the study for about 7 months or a little longer than a year if you decide to take part in an extension study. Participants are randomly, double-blind placed into 1 of 2 study groups:</p>

<ul>
 	<li>Group 1 takes the study drug (utreloxastat)</li>
 	<li>Group 2 takes the placebo</li>
</ul>
<p style="font-weight: 400;">Both the study drug and the placebo are liquid that is swallowed 2 times a day. The placebo looks like the study drug, but it contains no active ingredient. Two-thirds of participants will take the study drug, and one-third will take the placebo.</p>
<p style="font-weight: 400;">There are 4 parts to this study:</p>

<ul>
 	<li>Screening: Lasts about 8 weeks with at least 1 visit to the study clinic and 1 telephone call visit. You will receive tests and exams to see if you qualify to take part in the study.</li>
 	<li>Treatment Part A: Lasts 24 weeks, with around 3 visits occurring at the study clinic and 5 over the telephone. A home health provider will come to your home up to 5 times to take vital signs and draw blood. You will take the assigned study drug or the placebo during this part.</li>
 	<li>Treatment Part B (optional): Continues for 28 weeks with 3 visits occurring at the study clinic and 3 over the telephone. In-home vital signs and blood draws will occur 3 times. All participants who agree to continue in the study will take the study drug.</li>
 	<li>Follow-up: All participants will have a final follow-up visit by telephone 4 weeks after they stop taking the study drug or placebo.</li>
</ul>
<h2>Can I participate in the study?</h2>
<p style="font-weight: 400;">The main criteria to participate in this study are:</p>

<ul>
 	<li>You are 18-80 years old;</li>
 	<li>Began having symptom(s) less than 2 years ago that led to your ALS diagnosis;</li>
 	<li>Are able to swallow the liquid study drug;</li>
</ul>
<p style="font-weight: 400;"><em>Note: This list is not exhaustive, but these are the main criteria.</em></p>

<h2>Registration and more information</h2>
<p style="font-weight: 400;">If you are interested in participating in the CARDINALS study 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. If you experience difficulty contacting a centre, you may reach out directly to the TRICALS operational team at <a href="mailto:info@tricals.org">info@tricals.org</a> for support in this process. More information about the study can be found at https://classic.clinicaltrials.gov/ct2/show/NCT05349721?term=NCT05349721&amp;rank=1</p>

CARDINALS

<h3>Investigational drug</h3>
Approximately 2% of people living with ALS have a mutation in the <em>SOD1</em> gene. Mutations in the <em>SOD1</em> gene can lead to the production of abnormal SOD1 protein that is likely to be toxic to cells and could possibly lead to nerve cell death observed in people with ALS. The ATLAS study is evaluating whether an investigational drug (tofersen) may delay the onset of signs or symptoms of ALS and/or slow the decline in function once clinical signs or symptoms appear (compared to the initiation of tofersen at the time of, or after, emergence of ALS)

Tofersen (BIIB067) is an antisense oligonucleotide (ASO), which is designed to reduce levels of SOD1 protein in people with ALS associated with a <em>SOD1</em> gene variant.
<h3>Study overview</h3>
The study is comprised of 4 parts as described below.

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<h3>Part A</h3>
If you test positive for one of the <em>SOD1</em> gene variants being studied and pass other screening tests, you will be eligible to enter Part A of the study. During this part, you will not receive any study treatment, but your health status and neurofilament (NF) level will be closely monitored (at home or at the study center). The study team will evaluate your blood samples monthly for an NF level that is above the threshold. You and the study doctor will know when the NF level is above the threshold to consider eligibility for the next part of the study (Part B). The duration of Part A will vary.
<h3>Part B</h3>
If your NF level increases above the pre-determined threshold during your participation in Part A, the study doctor will evaluate the cause of the increase in NF. If the study doctor determines that the NF increase is not attributable to an alternative cause unrelated to ALS disease activity, you may choose to be screened for eligibility for potential participation in Part B. During this part of the study, you will be assigned to receive either the investigational drug or a placebo. You will be assigned randomly by a computer, and you will have an equal chance of receiving the investigational drug or placebo. Neither you nor the study team will know whether you received the placebo or the investigational drug; this is called a double-blind period.

The investigational drug or placebo is delivered intrathecally. Intrathecally means that the study drug is given to you by a procedure called a lumbar puncture.
<h3>Part C</h3>
If you show signs or symptoms of ALS during Part B of the study and your study doctor and a group of independent reviewers confirm that these signs or symptoms definitely indicate onset of ALS, you may choose to be screened for eligibility for potential participation in Part C of the study. During this part, everyone will receive the investigational drug; this is called an open-label period. The total length of participation in Parts B and C of the study is up to approximately two years.
<h3>Part D</h3>
If you start to show signs or symptoms of ALS during your participation in Part A or during screening for Part B, and your study doctor and a group of independent reviewers confirm that these signs or symptoms definitely indicate onset of ALS, you may choose to be screened for eligibility for potential participation in Part D of the study. Part D is another open-label period during which everyone will receive the investigational drug. The total length of participation in Part D is up to approximately two years.
<h3>Key Eligibility Criteria</h3>
To be able to enroll in the ATLAS study (Part A), participants need to:
<ul>
 	<li>Not have any signs or symptoms of ALS (that definitely indicate onset of ALS)</li>
 	<li>Have a certain <em>SOD1</em> mutation (that is associated with high/complete penetrance and rapid disease progression)</li>
 	<li>Be 18 years of age or older</li>
 	<li>Have a plasma NF level below the pre-defined threshold</li>
</ul>
If you are eligible and choose to participate, you will receive the following at no cost:
<ul>
 	<li>Comprehensive study-related health evaluations and assessments, including genetic testing</li>
 	<li>Investigational drug or placebo</li>
 	<li>All study-related visits and care</li>
</ul>
<h3>Travel Support</h3>
Assistance with travel and accommodations, and reimbursement for study-related expenses may also be available.

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ATLAS 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

Current trials

Juan Francisco Vázquez Costa

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MND Unit, Neurology Department

Hospital Universitari i Politècnic La Fe

The MND Unit of the Hospital Universitari i Polit&egrave;cnic La Fe is [&hellip;]

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