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Study of utreloxastat (PTC857) in adults with Amyotrophic Lateral Sclerosis (ALS) to evaluate its effectiveness in slowing disease progression

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What is this study about?

This study focuses on Amyotrophic Lateral Sclerosis (ALS), a condition that affects nerve cells controlling muscle movement, leading to progressive muscle weakness. The research evaluates a new medication called PTC857 (also known as utreloxastat) which is given as an oral solution. The purpose of the study is to determine if PTC857 can slow down the progression of ALS.

The study compares PTC857 with placebo in people who have ALS. Participants will receive either PTC857 or placebo for 24 weeks. During this time, patients can continue taking their standard ALS medications, including riluzole, edaravone, or sodium phenylbutyrate/taurursodiol, as long as they were already using these medications before joining the study.

The research will measure how the disease progresses by looking at changes in physical function and survival. The study will also check how safe PTC857 is and how well patients tolerate it. Additionally, researchers will examine the medication’s effects by measuring certain substances in the blood and spinal fluid that may indicate how the disease is developing.

1 Initial assessment

You will undergo an evaluation to confirm your diagnosis of amyotrophic lateral sclerosis (ALS) and verify that your symptoms started within the last 24 months

Your breathing capacity will be tested to ensure it is at least 60% of normal function

Your ALS Functional Rating Scale-Revised (ALSFRS-R) score must be at least 34 points to participate

2 Treatment assignment

You will be randomly assigned to receive either PTC857 oral solution or a matching placebo

Neither you nor your doctor will know which treatment you are receiving

If you are currently taking other ALS medications (riluzole, edaravone, or sodium phenylbutyrate/taurursodiol), you can continue using them if the dose remains stable

3 Treatment period

The treatment period will last 24 weeks

You will take the assigned medication orally according to the prescribed schedule

Regular assessments will track changes in your ALS symptoms and overall function

4 Monitoring and assessments

Your ALS symptoms will be evaluated using the ALSFRS-R scale

Regular breathing tests will measure your lung function

Blood samples will be collected to measure medication levels and disease markers

Quality of life assessments will be conducted using a specialized questionnaire

Safety monitoring will include physical examinations, vital signs, and laboratory tests

5 Final evaluation

After 24 weeks, a final assessment will measure the changes in your condition

The effectiveness of the treatment will be evaluated based on your functional abilities and survival

Follow-up safety assessments will be conducted

Who Can Join the Study?

  • Age between 18 and 80 years at screening
  • Diagnosed with ALS within the last 24 months before screening
  • Have an ALSFRS-R score (a scale that measures physical function in ALS) of at least 34
  • Have adequate breathing function with slow vital capacity (a breathing test) of 60% or higher
  • Must be on stable doses of any regular medications for at least 14 days before screening
  • If taking ALS treatments (riluzole, edaravone, or sodium phenylbutyrate/taurursodiol), doses must be stable for at least 30 days before screening
  • Female participants must:
    • Have normal breast cancer screening within 6 months before screening
    • Not donate eggs during the study and for 90 days after
    • Be post-menopausal, surgically sterile, or use effective contraception
  • Male participants must:
    • Use barrier contraception if sexually active with females who could become pregnant
    • Not donate sperm during the study and for 90 days after
  • Must be able to understand and comply with study procedures
  • Must provide written informed consent to participate in the study

Who Cannot Join the Study?

  • Age below 18 or above 80 years old
  • Presence of any significant medical conditions that could interfere with the study, such as:
    • Severe heart disease
    • Uncontrolled high blood pressure
    • Active cancer
    • Severe kidney or liver problems
  • Participation in another clinical trial within the past 30 days
  • Known allergies to PTC857 or similar medications
  • Pregnancy or breastfeeding
  • Unable to provide informed consent
  • History of non-compliance with medical treatments
  • Use of medications that could interact with the study drug
  • Inability to attend regular study visits
  • Significant abnormal laboratory test results
  • Mental health conditions that could affect study participation

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Oslo Universitetssykehus HF Oslo Norway
Medizinische Hochschule Hannover Hanover Germany
Hospital Universitario Y Politecnico La Fe Valencia Spain
University Hospital Of Clermont-Ferrand Clermont Ferrand France
City Clinic Research Sp. z o.o. Warsaw Poland
University Hospital Jena KöR Jena Germany
Centre Hospitalier Universitaire De Bordeaux Bordeaux France
Rostock University Medical Center Rostock Germany
Centre Hospitalier Universitaire De Lille Lille France
Katholieke Universiteit te Leuven Leuven Belgium

Other Sites

Site Name City Country Status
Azienda Ospedaliero Universitaria Di Modena Modena Italy
Region Vaesterbotten Umea Sweden
Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone Palermo Italy
AZ Sint-Lucas & Volkskliniek Gent Belgium
Region Skane Skanes Universitetssjukhus Lund Sweden
Vestre Viken HF Drammen Norway
Fondazione IRCCS Policlinico San Matteo Pavia Italy
Universitair Medisch Centrum Utrecht Utrecht The Netherlands
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Fakultni Nemocnice Brno Brno Czechia
IRCCS Ospedale Policlinico San Martino Genoa Italy
Centre Hospitalier Universitaire De Montpellier Montpellier France
Centre Hospitalier Universitaire De Nice Nice France
Azienda Ospedaliero-Universitaria Maggiore Della Carita Novara Italy
Fondazione Salvatore Maugeri Clinica Del Lavoro E Della Riabilitazione Pavia Italy
Institut fuer Klinische Transfusionsmedizin und Immungenetik Ulm gGmbH Ulm Germany
Centre Hospitalier Lyon Sud Pierre Benite France
University Of Luebeck Luebeck Germany
Neuroprotect Sp. z o.o. Warsaw Poland
Beaumont Hospital Dublin Ireland
Forbeli s.r.o. Prague Czechia
Clxozg Cwaugml Nxio Milan Italy
Craklj Hqwxuhjavdz Ev Uxfjdgwavzvcl Dh Lncarwm Limoges France
Hhbit Bjlrcg Hg Bergen Norway
Akcpdcp Ojengbxigbq Uwckbxjwhjnzi Cffxmkgebgqb Dqaif Svfuab E Djcan Slugfag Da Trobrg Turin Italy
Hxcmkgqt Da Lq Swxcg Cpbu I Syxj Pcq Barcelona Spain
Usxklgjukk Digws Sxkcl Ds Rhwt Ln Snseyafd Rome Italy
Hrvnclyq Vsog dtkxfzyh Barcelona Spain
Iqmgd Oqxgybfq Azrgxfbqcr Spm Lyht Milan Italy

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Belgium Belgium
Not recruiting
14.02.2023
Czechia Czechia
Not recruiting
14.02.2023
France France
Not recruiting
14.02.2023
Germany Germany
Not recruiting
14.02.2023
Ireland Ireland
Not recruiting
14.02.2023
Italy Italy
Not recruiting
14.02.2023
Norway Norway
Not recruiting
14.02.2023
Poland Poland
Not recruiting
14.02.2023
Spain Spain
Not recruiting
14.02.2023
Sweden Sweden
Not recruiting
14.02.2023
The Netherlands The Netherlands
Not recruiting
14.02.2023

Trial locations

Investigated drugs:

PTC857 is an investigational medication being studied for treating amyotrophic lateral sclerosis (ALS). It is being tested to see if it can help slow down the progression of ALS symptoms in adult patients. This is a new drug that is still being researched and is currently known by its development code name since it hasn’t yet been given a standard generic name.

Investigated diseases:

Amyotrophic Lateral Sclerosis – A progressive neurological disease that affects nerve cells responsible for controlling voluntary muscle movement. The condition causes the death of motor neurons, which gradually reduces a person’s ability to move muscles. ALS affects various physical functions, including walking, speaking, swallowing, and breathing. The disease typically begins with muscle twitching, weakness in a limb, or slurred speech. As motor neurons degenerate, the brain loses its ability to control muscle movement.

Trial ID:
2023-510317-26-00
Protocol code:
PTC857-CNS-001-ALS
NCT ID:
NCT05349721
Trial Phase:
Therapeutic exploratory (Phase II)

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