Study on the Safety and Effectiveness of AMX0035 (Phenylbutyrate and Ursodoxicoltaurine) for Adults with Amyotrophic Lateral Sclerosis (ALS)

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What is this study about?

This clinical trial is focused on studying Amyotrophic Lateral Sclerosis (ALS), a progressive disease that affects nerve cells in the brain and spinal cord, leading to muscle weakness and loss of movement. The trial will evaluate the safety and effectiveness of a treatment called AMX0035, which is a combination of two substances: phenylbutyrate and ursodoxicoltaurine. These substances are provided in a powder form and taken orally. The study will compare the effects of AMX0035 to a placebo over a period of 48 weeks.

The purpose of the study is to assess how AMX0035 impacts the progression of ALS in adult patients. Participants will be randomly assigned to receive either AMX0035 or a placebo, and neither the participants nor the researchers will know who is receiving which treatment. This is known as a double-blind study. Throughout the 48 weeks, participants will have regular visits to the clinic to monitor their health and the progression of the disease.

During the trial, researchers will measure changes in the participants’ abilities to perform daily activities and their overall survival. The study aims to provide valuable information on whether AMX0035 can slow down the progression of ALS and improve the quality of life for those affected by this challenging condition.

1 joining the study

Upon joining the study, the participant will be informed about the trial’s purpose, procedures, and requirements. The participant must provide informed consent, confirming understanding and willingness to comply with the trial procedures.

2 baseline assessment

A baseline assessment will be conducted to evaluate the participant’s current health status and ALS condition. This includes measuring the ALS Functional Rating Scale-Revised (ALSFRS-R) score, which assesses the participant’s physical function.

3 treatment phase

The participant will receive the study medication, AMX0035, or a placebo. AMX0035 is an oral powder containing phenylbutyrate and ursodoxicoltaurine, taken by mouth. The treatment will last for 48 weeks.

The participant will take the medication as directed, following the dosage and frequency specified by the study protocol.

4 regular assessments

Throughout the 48-week treatment period, the participant will attend regular clinic visits and remote assessments. These visits will monitor the participant’s health, ALS progression, and any side effects from the medication.

The ALSFRS-R score will be reassessed at various intervals, including at Week 24 and Week 48, to track changes in physical function.

5 end of treatment evaluation

At the end of the 48-week treatment period, a final evaluation will be conducted. This includes a comprehensive assessment of the participant’s ALS condition and overall health.

The primary endpoint is the change in the ALSFRS-R total score from baseline to Week 48, adjusted for mortality.

6 follow-up period

After completing the treatment phase, the participant will enter a follow-up period. This involves continued monitoring of health status and any long-term effects of the treatment.

Participants are required to adhere to birth control measures for 3 months after the last dose of the study drug.

Who Can Join the Study?

Must be a male or female who is at least 18 years old.
Men should not plan to father a child or donate sperm during the trial and for 3 months after the last dose of the study drug.
Must have a diagnosis of Amyotrophic Lateral Sclerosis (ALS), confirmed by a doctor experienced in ALS management.
The first symptom of ALS should have started less than 24 months ago.
If taking medications like riluzole or edaravone, these should have been started and kept stable for at least 14 days for riluzole or a full treatment cycle for edaravone before the trial begins.
Must be able to give informed consent, which means understanding and agreeing to participate in the trial.
Must be capable and willing to follow trial procedures, including attending clinic visits, remote visits, and reporting status as required.
Women who can have children must agree to use effective birth control during the trial and for 3 months after the last dose of the study drug.
Women must not be pregnant or planning to become pregnant during the trial and for 3 months after the last dose of the study drug.
Men must agree to use contraception during the trial and for at least 3 months after the last dose of the study drug.

Who Cannot Join the Study?

Patients who have a medical condition other than Amyotrophic Lateral Sclerosis (ALS) that could interfere with the study.
Patients who are unable to follow the study procedures or attend the required visits.
Patients who are currently participating in another clinical trial.
Patients who have a history of allergic reactions to the study medication or similar drugs.
Patients who have a serious illness or condition that could affect their safety during the study.
Patients who are pregnant or breastfeeding.
Patients who have a history of drug or alcohol abuse that could interfere with the study.
Patients who have had a recent major surgery or are planning to have surgery during the study period.
Patients who have a mental health condition that could affect their ability to participate in the study.
Patients who are unable to provide informed consent, which means they cannot understand and agree to the study’s requirements.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Medizinische Hochschule Hannover	Hanover	Germany
Bellvitge University Hospital	L'hospitalet De Llobregat	Spain
Hospital Universitario Y Politecnico La Fe	Valencia	Spain
University Hospital Of Clermont-Ferrand	Clermont Ferrand	France
Azienda Ospedaliera Universitaria Universita’ Degli Studi Della Campania Luigi Vanvitelli	Naples	Italy
Linden Sp. z o.o. sp.k.	Cracow	Poland
City Clinic Research Sp. z o.o.	Warsaw	Poland
University Hospital Jena KöR	Jena	Germany
Universitaetsklinikum Heidelberg AöR	Heidelberg	Germany
Rostock University Medical Center	Rostock	Germany
Centre Hospitalier Universitaire De Lille	Lille	France
Katholieke Universiteit te Leuven	Leuven	Belgium
Unidade Local De Saúde De Santa Maria, E.P.E.	Lisbon	Portugal

Other Sites

Site Name	City	Country
Azienda Ospedaliero Universitaria Di Modena	Modena	Italy
Region Vaesterbotten	Umea	Sweden
Hospital Del Mar	Barcelona	Spain
Region Stockholm – SLSO	Stockholm	Sweden
Azienda Ospedaliera di Padova	Padua	Italy
Pia Fondazione Di Culto E Religione Card G Panico	Tricase	Italy
Universitair Medisch Centrum Utrecht	Utrecht	The Netherlands
Charite Universitaetsmedizin Berlin KöR	Berlin	Germany
Centre Hospitalier Universitaire De Montpellier	Montpellier	France
Centre Hospitalier Universitaire De Nice	Nice	France
Institut fuer Klinische Transfusionsmedizin und Immungenetik Ulm gGmbH	Ulm	Germany
Centre Hospitalier Lyon Sud	Pierre Benite	France
Hopital Beaujon	Clichy	France
Beaumont Hospital	Dublin	Ireland
Cnhssz Cctibbv Nkzr	Milan	Italy
Chkpsp Hijvszxibtv Er Uywxiadqkhzcr Ds Llqkihn	Limoges	France
Aywzswajxz Pefqymte Hxfneayj Du Mxkzayhiz	Marseille	France
Czfrdy Haautvbmxwx Rxvlatgr Ubxlbxmehotfv Da Tepao	Tours	France
Ankqbhy Oiorcntdioa Ufkpelnnhelfg Czsqvviwzksk Dtkbo Swsozy E Ddrxe Sctjqiz Dh Tyofls	Turin	Italy
Htuzhqbz Uunckdblmuwgd Ddviqkzw	Donostia / San Sebastian	Spain
Ikvac Oouvgnuk Abtrripngp Sdc Lglo	Milan	Italy

Trial status

Country	Status	Recruitment Start
Belgium	Not recruiting	02.12.2021
France	Not recruiting	02.12.2021
Germany	Not recruiting	02.12.2021
Ireland	Not recruiting	02.12.2021
Italy	Not recruiting	02.12.2021
Poland	Not recruiting	02.12.2021
Portugal	Not recruiting	02.12.2021
Spain	Not recruiting	02.12.2021
Sweden	Not recruiting	02.12.2021
The Netherlands	Not recruiting	02.12.2021

Trial locations

Investigated drugs:

AMX0035 is a medication being studied for its potential to slow down the progression of Amyotrophic Lateral Sclerosis (ALS) in adults. ALS is a disease that affects nerve cells in the brain and spinal cord, leading to loss of muscle control. This trial aims to evaluate the safety and effectiveness of AMX0035 over a 48-week period.

Investigated diseases:

Amyotrophic lateral sclerosis

Amyotrophic Lateral Sclerosis (ALS) – ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord. It leads to the gradual degeneration and death of motor neurons, which are responsible for controlling voluntary muscles. As these neurons die, the brain loses the ability to initiate and control muscle movement, leading to muscle weakness and atrophy. Over time, individuals with ALS may experience difficulty speaking, swallowing, and breathing. The progression of the disease varies among individuals, but it typically leads to increasing disability. ALS is considered a rare disease and its exact cause is not fully understood.

Trial ID:

2023-508511-23-00

Protocol code:

A35-004

NCT ID:

NCT05021536

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on the Safety and Effectiveness of AMX0035 (Phenylbutyrate and Ursodoxicoltaurine) for Adults with Amyotrophic Lateral Sclerosis (ALS)

What is this study about?

Who Can Join the Study?

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