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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
University Hospital Jena KöR Jena Germany
Centre Hospitalier Universitaire De Lille Lille France
Katholieke Universiteit te Leuven Leuven Belgium
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
Centre Hospitalier Universitaire De Bordeaux Bordeaux France
Rostock University Medical Center Rostock Germany

Other Sites

Site Name City Country Status
Centre Hospitalier Universitaire De Nice Nice France
Azienda Ospedaliero Universitaria Di Modena Modena Italy
Fondazione Salvatore Maugeri Clinica Del Lavoro E Della Riabilitazione Pavia Italy
AZ Sint-Lucas & Volkskliniek Gent Belgium
Hmrorhlh Vsfy drwynlen Barcelona Spain
Bjgygtxh Hrgyxegg Dublin Ireland
Ftntkbsrcq Iilht Pswjicphztx Szu Miedfq Pavia Italy
Rylcrv Scoia Sblspo Umeibmopwpfumwqkwcy Lund Sweden
Uoaypqdepgev Mlaoyzg Chafutw Uuwzfru Utrecht The Netherlands
Hjono Bjleor Hj Bergen Norway
Cwynxmg Uabqocapxcgfogwjbnwi Bnyhfs Kft Berlin Germany
Ftbvhjco Nhdsxtrts Bjkd Brno Czechia
Ileui Opvpomca Pepncjqjffv Swf Mekqjav Genoa Italy
Cmspud Hzupumhgzru Urugixoreeyle Dr Makuurldajn Montpellier France
Angnkri Oqkgeohswfr Uambawojvhkwl Cwurbrtoydqz Dgwud Stokpz E Dedan Staghsr Dp Toonvi Turin Italy
Atwskhe Okjnpegdseymrqjfglqipltam Mtfjtmbo Dwzsq Cbktoy Novara Italy
Hmhdrygb Dl Le Sisyv Cwnk I Smsq Pze Barcelona Spain
Urilptteap Drqfn Skqcy Dd Rvzr Lb Sdpuqwnp Rome Italy
Ibamsprb fiio Klbkwptpz Togkbfwerdqlfaldsjy uor Iicxmjhriclq Ubi ghnhk Ulm Germany
Cgvbnx Hazjbeneocr Llec Sqc Pierre Benite France
Cqtoon Cqfufom Nkoj Milan Italy
Fhuhybg shttuy Prague Czechia
Cihnrx Hfaeoosoncj Ew Udclbpfqnache Dq Lkkogxl Limoges France
Vnwcrh Vuhky Hz Drammen Norway
Rlfxyh Vonydtrakvmof Umea Sweden
Atmtzae Obobicmapyr Uudcokhwmbmyz Phlemdxjcdf Pvuht Gesymeda Palermo Italy
Uyponmwggk Od Laglkgs Luebeck Germany
Nifaeuipunyu Spa z olmu Warsaw Poland
Igxtz Okrtjmag Aftykdyeem Sug Ljis 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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