<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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Jesús Esteban Pérez - TRICALS

Our Specialists

<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

Industry trial

<h3>Study design</h3>
The ALXN1210-ALS-308 trial is sponsored by Alexion Pharmaceuticals and is a randomised, double-blind, placebo-controlled trial. This means that the effect of ravulizumab is tested by comparing a group of patients that receive ravulizumab to a group of patients receiving placebo. Participants will be randomly assigned to receive ravulizumab or placebo. A placebo is something that looks exactly the same as the study medicine but does not have any active medicine in it. Participants are twice as likely to be assigned to ravulizumab treatment versus placebo.The study has a randomized controlled period of 50 weeks and during this time, the participant and the research team do not know who is receiving placebo or ravulizumab.  After the randomized controlled period, participants can enter the Open-label Extension Period of the study and receive ravulizumab.

Ravulizumab and placebo will be administered via an injection into the bloodstream (intravenously).
<h3><strong>Key Inclusion Criteria</strong></h3>
<h4><strong>*** Enrolment for this study has been completed ***</strong></h4>
The study intends to recruit 354 adults with either <a href="https://alsnewstoday.com/forms-of-als/sporadic-als/">sporadic</a> or <a href="https://alsnewstoday.com/forms-of-als/familial-als/">familial ALS</a>. Key eligibility criteria for people with ALS to participate, include:
<ul>
 	<li>Disease onset within the prior 36 months</li>
 	<li>Documented meningococcal vaccination not more than 3 years prior to, or at the time of, initiating study treatment</li>
 	<li>Slow vital capacity (SVC), a measure of respiratory function, of at least 65% predicted at screening</li>
 	<li>If the participant is taking riluzole, he/she must be on a stable dose for 30 days</li>
 	<li>If the participant is taking edaravone, he/she must have been on a stable dose for 60 days</li>
 	<li>Body weight at least 40 kilograms at screening.</li>
 	<li>Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies</li>
</ul>
The ALXN1210-ALS-308 trial runs in ALS centres around the globe, including several TRICALS centres. For further information, please refer to:  <a href="https://clinicaltrials.gov/ct2/show/NCT04248465" target="_blank" rel="noopener noreferrer">https://clinicaltrials.gov/ct2/show/NCT04248465</a>.

ALXN1210-ALS-308 trial

<h3><b>Tofersen</b></h3>
Approximately 2% of  people living with ALS have a mutation in the superoxide dismutase 1 (SOD1) gene. The mutation of the SOD1 gene leads to the production of an abnormal SOD1 protein that is likely to be toxic to cells and could possibly lead to the nerve cell death seen in people with ALS. The VALOR study is evaluating a potential new treatment for people living with ALS caused by a SOD1 gene mutation (SOD1-ALS). Tofersen (BIIB067) is an antisense oligonucleotide (ASO), which is designed to reduce levels of SOD1 protein in people with SOD1-ALS.
<h3>Study overview</h3>
You will be assigned to a study drug group at random (by chance) to receive either the investigational drug or placebo. A placebo is a substance that looks like the investigational drug but contains no actual active drug. They help us to make sure that any changes seen during the study are due to the investigational drug alone and not another reason. Study participants will have a 2-in-3 chance of being assigned to the investigational drug group, and a 1-in-3 chance of being assigned to the placebo group. Neither the participant nor the study team will be told which group they have been placed into until after the study has finished.

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. All participants will receive 8 lumbar punctions throughout the study duration. The study duration is 32-36 weeks, including:
<ul>
 	<li>4-week screening</li>
 	<li>24-week study treatment period</li>
 	<li>4 to 8-week follow-up period</li>
</ul>
<h4><strong>Key eligibility criteria</strong></h4>
<strong><span class="underline-word">The study is now active and is no longer enrolling new participants.</span></strong>

To enrol in the VALOR study, participants need to be:
<ul>
 	<li>18 years of age or older</li>
 	<li>Have weakness caused by ALS</li>
 	<li>Agree to genetic testing for the SOD1 mutation</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><strong>Travel Support</strong></h3>
Assistance with travel and accommodations, and reimbursement for study-related expenses may be available.
<h3><strong>Additional information</strong></h3>
<ul>
 	<li>More information about the VALOR trial can be found <a href="https://clinicaltrials.gov/ct2/show/NCT02623699" target="_blank" rel="noopener noreferrer">here</a>.</li>
 	<li>The patient brochure can be downloaded <a href="https://backend.tricals.org/wp-content/uploads/2020/10/233AS101-United-States-English-United-States-Study-Brochure-Study-Brochure.pdf" target="_blank" rel="noopener noreferrer">here</a>.</li>
</ul>

VALOR study

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

RT001 in Amyotrophic Lateral Sclerosis

ALXN1210-ALS-308 trial

VALOR study

TUDCA-ALS