<p>The ALS Unit at the Hospital 12 de Octubre in Madrid is one of the 4 ALS Units in the capital district of Spain. It is the only one in the whole country which includes a research laboratory.</p>



Principal investigator

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Our Specialists

<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>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 Netherlands, Spain, Sweden, Italy and France.
<h2><strong>More information</strong></h2>
More information about the PHOENIX trial can be found <a href="https://clinicaltrials.gov/ct2/show/NCT05021536" target="_blank" rel="noopener noreferrer">here</a>.

PHOENIX trial

Industry 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 72 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:
<ol>
 	<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>
</ol>
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 Netherlands, Belgium, Poland, Italy, Germany, Spain and Ireland.

ADORE Trial

<h3><strong>Reldesemtiv in people with ALS</strong></h3>
Reldesemtiv was developed by Cytokinetics in order to counteract the decline in muscle function. In a phase 2 study, the drug was tested in people with ALS for a 12 week period. The current study is testing its effectiveness in the longer term and in a larger group of people.
<h3><strong>Study design</strong></h3>
This phase 3 study is a double-blind, randomised, placebo-controlled study. This means we are comparing the effects of reldesemtiv against the effects of a placebo. A placebo is a drug without an active ingredient, a ‘fake drug’.

Both reldesemtiv and the placebo should be taken as a daily capsule. Participants will be randomly allocated either the placebo or reldesemtiv. Neither the participant nor the research team will know whether the participant is receiving the placebo or reldesemtiv.

The entire study will last 48 weeks. Participants will be examined in hospital 8 times during this period. Participants will receive either reldesemtiv or a placebo during the first 24 weeks.  All participants will receive reldesemtiv during the second 24 weeks.
<h3><strong>Entry criteria</strong></h3>
The main entry criteria for participating in this study are as follows:
<ul>
 	<li>Participants must be between 18 and 80 years old</li>
 	<li>Disease onset within the last 24 months</li>
 	<li>The participant's lung function is at least 65%, without the use of a ventilator</li>
 	<li>When using Riluzole: a stable dose of ≥30 days prior to participation</li>
 	<li>Participants are not pregnant or breastfeeding</li>
 	<li>Participants haven’t participated in another clinical trial in the past 30 days</li>
</ul>
<em>Note: This list is not exhaustive, but these are the main criteria.</em>

Participants can continue their treatment with Riluzole for the entire study duration. Participants can also participate in the study if they don’t take Riluzole.

The study is currently being conducted in the following countries: The Netherlands, Ireland and Spain.

COURAGE-ALS Trial

<h3>Pegcetacoplan</h3>
Pegcetacoplan (also known as APL-2) has not previously been tested in people with ALS, but has been tested in previous trials in healthy volunteers and people with blood, kidney and eye diseases. Researchers believe that the immune system is changed in people with ALS and that this contributes to nerve cell loss. Pegcetacoplan is designed to shut down the functioning of part of your immune system. Therefore, it is now being investigated whether pegcetacoplan can possibly prevent the loss of nerve cells.
<h3>Study design</h3>
This phase 2 study was commissioned by Apellis Pharmaceuticals and is a double-blind, randomised, placebo-controlled study. This means we’re comparing the effects of pegcetacoplan with the effects of a placebo. A placebo is a drug without an active ingredient, a ‘fake drug’.

Both pegcetacoplan and the placebo are to be administered by injection twice a week. Participants are randomly assigned either the placebo treatment or the pegcetacoplan treatment. Neither the participant nor the research team will know which treatment the participant receives.

The entire study will last 116 weeks, which is approximately 2 years. During this period, the participant will visit the hospital 11 times for examinations. Participants will receive treatment with pegcetacoplan or the placebo for 52 weeks. After this phase, all participants will enter the 'open label' phase, during which everyone will receive pegcetacoplan.
<h3>Entry criteria</h3>
The main criteria for participation in this study are as follows:
<ul>
 	<li>The participant is at least 18 years old</li>
 	<li>The duration of their illness is no more than 72 weeks from when symptoms first appeared</li>
 	<li>The participant's lung function is at least 60%, without the use of a ventilator</li>
 	<li>Participants must not get pregnant or be breastfeeding throughout the duration of the study</li>
 	<li>Participants have been vaccinated within the last 5 years, or are willing to be vaccinated, against pneumococcus (<em>Streptococcus pneumoniae</em>), meningococcus (<em>Neisseria meningitidis</em>; types A, C, W, Y and B), and HiB (<em>Haemophilus influenzae</em> type B).</li>
 	<li>If the participant has a diaphragm pacing or considers undertaking the procedure during the study, participation is not possible</li>
 	<li>When using Riluzole: a stable dose of ≥30 days prior to participation</li>
 	<li>When using Edaravone: a stable dose of ≥60 days prior to participation</li>
</ul>
This study requires 228 participants from different countries. Several TRICALS centres are participating in this study. The study is currently ongoing in the following European countries: The Netherlands, Belgium, France, Ireland, Italy, and Spain.

APL2-ALS-206 Trial

<h2>RT001 in people with ALS</h2>
RT001 is made from slightly modified linoleic acid. Linoleic acid is typically part of a normal human diet and is found in most oils. RT001 can protect cells from lipid peroxidation – a process that is believed to cause damage in several neurodegenerative diseases, including ALS. RT001 has previously been shown to be safe when administered to humans. However, RT001 can currently only be prescribed in research settings.
<h2>Study overview</h2>
This phase 2 trial is sponsored by Retrotope Inc and is a randomised, double-blind, placebo-controlled study. This means that the safety and treatment effects are studied by comparing a group of ALS patients that receive RT001 against a group of patients receiving a placebo drug. A placebo drug looks exactly the same as the RT001, but does not contain any active medicine (a mock treatment). Both treatments need to be ingested orally during breakfast, lunch and dinner. Participants will be randomly assigned to receive RT001 or placebo. Both the participant and the research team do not know who receives a placebo treatment or RT001.

The entire study duration is 28 weeks. During this period you are expected to visit the hospital at least 4 times for examinations. Each visit typically takes around 3 to 4 hours.
<h2>Can I participate in this study?</h2>
<strong>*** Please note that enrolment for this study has now been completed ***</strong>

The study intends to recruit 40 participants living with ALS from different countries.

Key eligibility criteria for people with ALS to participate, include:
<ul>
 	<li>Male or female aged between 20 years to 75 years</li>
 	<li>The onset of ALS is less than 3 years ago</li>
 	<li>Respiratory function (SVC) is at least 70%, without ventilatory support</li>
 	<li>Concurrent treatment with other experimental therapies is not allowed</li>
 	<li>If participants have a feeding tube or if the need for a feeding tube is anticipated within the first 24 weeks after starting the study, they cannot participate</li>
</ul>
<h3><strong>More information</strong></h3>
Additional information about the trial can be found on <a href="http://www.clinicaltrialsregister.eu">www.clinicaltrialsregister.eu</a> and <a href="https://clinicaltrials.gov/ct2/show/NCT04762589?term=RT001&amp;draw=2&amp;rank=1">www.clinicaltrials.gov</a>.

RT001 in Amyotrophic Lateral Sclerosis

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>

University Hospital 12 de Octubre

Contact

Our Specialists

Jesús Esteban Pérez

Current trials

PHOENIX trial

ADORE Trial

COURAGE-ALS Trial

APL2-ALS-206 Trial

RT001 in Amyotrophic Lateral Sclerosis

TUDCA-ALS