<p>The Leuven ALS center combines the diagnosis and multidisciplinary care for ALS and ALS/FTD (frontotemporal dementia) patients with clinical and basic research activities.</p>



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Philip van Damme studied neurology at KU Leuven medical school. During his [&hellip;]

Philip van Damme - TRICALS

Our Specialists

<h2>AP101</h2>
<p style="font-weight: 400;">ALS is a nervous system disease that weakens muscles and impacts physical function. There are two types of ALS: sporadic ALS is the most common type and means it happens sometimes without a clear cause. Familial ALS runs in families and it is caused by changes to a certain gene, e.g. superoxide dismutase 1 or SOD1. AL-S Pharma is developing a new study drug, AP101, to treat ALS by reducing affected SOD1.</p>

<h2>Study design</h2>
<p style="font-weight: 400;">This study includes 2 parts: a double-blind phase (where neither you nor your doctor know that you receive medication or placebo) and an open label extension phase (OLE, where you receive medication). The purpose of the double-blind phase is to compare the effects of the research medicine, AP-101, with placebo. A placebo looks like the research medicine but does not contain any active medicine. Researchers use a placebo to see if a research medicine works better or is safer than not taking anything at all.</p>
<p style="font-weight: 400;">If you agree to take part in the double-blind phase of the study, you will be assigned randomly (by chance, like flipping a coin) to receive either the research medicine, AP-101, or placebo. You will have a 66% (2 in 3) chance of receiving AP-101 and a 33% (1 in 3) chance of receiving placebo. Both AP-101 and placebo will be given to you as intravenous injections (also called “infusions”; this is a slow injection into a vein in your arm).</p>
<p style="font-weight: 400;">If you are assigned to AP-101 you will receive a first dose of 500 mg on day 1, and on day 2 a dose of 2500 mg of AP-101. After day 2 you will receive a 2500 mg dose of AP-101 every 3 weeks over a treatment period of 6 months in total. If you are assigned to placebo you will receive the placebo infusions instead of AP-101 infusions. Each infusion will last between 1 and 2 hours. You will be asked to stay at the research site for up to 6 hours after the infusion is complete to monitor your health.</p>
<p style="font-weight: 400;">In total, your participation in the trial will last for about 11 months for the double-blind phase. You will need to visit the research site about 15 times during the study. You will also have a telephone study visit.</p>
<p style="font-weight: 400;">If you are currently receiving medication for ALS (riluzole or edaravone) you can continue taking it in the same dose throughout the study. However, starting</p>
<p style="font-weight: 400;">or modifying doses of riluzole or edaravone are not allowed during the study.</p>
<p style="font-weight: 400;">If you complete the treatment period for this double-blind phase you may be able to continue receiving the study medicine by taking part in the separate OLE phase in which all participants will receive AP-101 (there will be no placebo group).</p>

<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 have familial ALS caused by a SOD1 gene mutation</li>
 	<li>Your symptoms were initially detected within the last two years</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 AP101-02 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 <a href="https://clinicaltrials.gov/ct2/show/NCT05039099">clinicaltrials.gov</a></p>

<h2 style="font-weight: 400;"><strong>Participating TRICALS centres</strong></h2>
<p style="font-weight: 400;">Please note that it is also possible to participate in some of these centres if you are resident in another country. Travel and accommodation costs may be reimbursed.</p>

AP101-02

Gesponsorde 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

Philip van Damme

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UZLeuven

ALS Center Leuven

UZ Leuven

Our clinical research activities include genetic, imaging (such as PET/MRI), electrophysiological and [&hellip;]

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