<p>The ALS research group at Ume&aring; University Hospital performs clinical studies, including phase 1&mdash;3 trials as well as molecular pathology research using genetic (DNA), proteomic (proteins) and metabolomic approaches.</p>



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Professor of Neurology and Senior Neurologist

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Professor Andersen obtained his medical degree from Copenhagen University and specialised in [&hellip;]

Peter Andersen - TRICALS

Researcher and Medical Doctor

Anton Tjust obtained his medical and doctorate degree from Ume&aring; University. His [&hellip;]

Anton Tjust - TRICALS

Neurologist

Frida Nordin obtained her medical degree from Ume&aring; University in 2008 and [&hellip;]

Frida Nordin - TRICALS

Resident in Neurology and Researcher

Karins Forsberg obtained her medical from Ume&aring; University. Her doctoral thesis was [&hellip;]

Karin Forsberg - TRICALS

Researchnurse in clinical trials

Erica Almgren Stenberg is a Researchnurse in clinical trials for ALS in [&hellip;]

Erica Almgren Stenberg - 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>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

<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

Current trials

Peter Andersen

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ALS Research Group

Umeå University Hospital

In 1992, the ALS Clinical Center was established at Ume&aring; University Hospital. [&hellip;]