<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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Professor of Neurology

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Philip van Damme - TRICALS

Specialists

<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>
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

Industry 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

<h3>Triumeq</h3>
Triumeq is a drug that is used to treat patients with the human immunodeficiency virus (HIV) and is shown to be safe. It is thought that ancient viruses (retroviruses) have left their genetic material in our DNA during the human evolution. In some people, this old genetic material may become activated and is believed to be a cause of ALS.  In a small trial, it has been shown that Triumeq could suppress this genetic reactivation and may slow disease progression in patients with ALS. Triumeq was shown to be safe and well tolerated in patients with ALS. With this clinical trial we aim to confirm the efficacy of Triumeq in patients with ALS.

<a href="https://www.ema.europa.eu/en/medicines/human/EPAR/triumeq" target="_blank" rel="noopener noreferrer">Read more about Triumeq</a>
<h3><strong>Study design</strong></h3>
The Lighthouse 2 trial is a randomized, double-blind, placebo-controlled study. This means that participants will be randomly allocated to receive a treatment with either Triumeq or a control medicine (placebo). During the study, both the patient and the research team do not know who is receiving the placebo treatment or Triumeq. This will help researchers to objectively assess the efficacy and side-effects of Triumeq.

The Lighthouse 2 trial

Investigator initiated trial

MAGNET

Platform 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>

UZ Leuven

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