<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

<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.

<img class="alignnone wp-image-23821" src="https://backend.tricals.org/wp-content/uploads/2023/02/26802_Biogen_ATLAS-Study_Editable_Schematic_092722_EN.jpg" alt="" width="950" height="441" />
<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.

&nbsp;

ATLAS trial

Industry 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