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Amyotrophic Lateral SclerosisPhase 2CENTAUR-OLENCT03488524

Open Label Extension Study of AMX0035 in Patients With ALS

A Phase 2 study testing AMX0035 in people with Amyotrophic Lateral Sclerosis and ALS, run by Amylyx Pharmaceuticals Inc. at 24 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This extension study, in which all participants received active treatment (AMX0035), was designed to assess the longer-term safety and therapeutic potential of AMX0035 for participants who have completed the Main Study (AMX3500, also known as CENTAUR). Read more about this study

Who is it for?
Any age
Conditions the study is for
Amyotrophic Lateral Sclerosis, ALS
What is being tested?
AMX0035
Could I receive a placebo?
No, everyone receives an active treatment
How many people
90 participants
Where
24 sitesUnited States
Study dates
29 Mar 2018to 1 Mar 2021
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Any age
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Completion of all visits in the randomized, double blind AMX3500 study. Participants who received tracheostomy or permanent assisted ventilation (PAV) during the course of the main study could enroll in the OLE if they completed all visits in the main study.
  • Must enroll in the OLE within 28 days of the Week 24 visit of the main study.
  • Signed informed consent to enter the OLE phase.

Who cannot join

  • Discontinued study drug prematurely in the double-blind phase of the study for reasons other than tracheostomy or PAV.
  • Exposure to or anticipated requirement for any disallowed medication listed in the protocol.
  • Any ongoing adverse events that in the opinion of the Site Investigator are clear contraindications to the study drug.
  • Unstable cardiac or other life-threatening disease emergent during the randomized, double blind study
  • Any major medical condition that in the opinion of the Site Investigator would interfere with the study and place the subject at increased risk.

Trial locations and contacts

24 sites in 1 country.

24 sites in 1 country

United States24 sites

  • University of Michigan, Michigan Medicine

    Ann Arbor, Michigan, 48109

  • Emory University Hospital

    Atlanta, Georgia, 30322

  • Johns Hopkins Hospital

    Baltimore, Maryland, 21205

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114

  • The Ohio State University, Wexner Medical Center

    Columbus, Ohio, 43221

  • Texas Neurology, PA

    Dallas, Texas, 75214

  • University of Florida College of Medicine

    Gainesville, Florida, 32610

  • University of Iowa, Carver College of Medicine

    Iowa City, Iowa, 52242

  • University of Kentucky Medical Center

    Lexington, Kentucky, 40536

  • Neurology Associates, PC

    Lincoln, Nebraska, 68506

  • Hennepin County Medical Center

    Minneapolis, Minnesota, 55415

  • Ochsner Neuroscience Institute

    New Orleans, Louisiana, 70121

  • University of California, Irvine

    Orange, California, 92868

  • The Penn Comprehensive Neuroscience Center

    Philadelphia, Pennsylvania, 19107

  • Temple University Hospital

    Philadelphia, Pennsylvania, 19140

  • Barrow Neurological Institute-Dignity Health, St Joseph's Hospital and Medical Center

    Phoenix, Arizona, 85013

  • Oregon Health & Science University

    Portland, Oregon, 97239

  • University of Texas Health Science Center, San Antonio

    San Antonio, Texas, 78229

  • California Pacific Medical Center

    San Francisco, California, 94114

  • Swedish Neuroscience Center

    Seattle, Washington, 98122

  • Washington University Medical Center

    St Louis, Missouri, 63110

  • University of South Florida College of Medicine

    Tampa, Florida, 33612

  • Wake Forest School of Medicine

    Winston-Salem, North Carolina, 27157

  • University of Massachusetts Memorial Medical Center

    Worcester, Massachusetts, 01655

Other trials in United States

About this study

Official title: Evaluation of the Safety, Tolerability, Efficacy, and Activity of AMX0035, a Fixed Combination of Phenylbutyrate (PB) and Tauroursodeoxycholic Acid (TUDCA), for Treatment of Amyotrophic Lateral Sclerosis: Open-Label Extension

This extension study, in which all participants received active treatment (AMX0035), was designed to assess the longer-term safety and therapeutic potential of AMX0035 for participants who have completed the Main Study (AMX3500, also known as CENTAUR).

Read the detailed description

The Centaur Open Label Extension Study (CENTAUR-OLE) is an extension study for patients with ALS who participated in the CENTAUR study (Study AMX3500). During the OLE, all participants received active treatment (AMX0035), and the investigators, evaluators, and participants remained blinded to the randomized treatment assigned at the beginning of the double-blind main study. The study was designed to assess the longer term safety and therapeutic potential of AMX0035.

Being tested
AMX0035
Medical subject headings
Amyotrophic Lateral Sclerosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • AMX0035Drug

    Also called Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO, also known as Ursodoxicoltaurine or Tauroursodeoxycholic Acid [TUDCA])

    Combination therapy of PB and TURSO

Study groups in NCT03488524
Study groupRoleWhat they receive
AMX0035ExperimentalAMX0035 twice daily--a combination of Sodium Phenylbutyrate (3g) and Taurursodiol (1g)
Receives: Drug: AMX0035

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of Participants With Treatment-Emergent Adverse Events (TEAEs)

    Number of participants with TEAEs from baseline in the OLE study through the last participant's last visit in the OLE

    Measured: From the Baseline Visit in the OLE study through Week 132 or the Early Discontinuation (Final Safety) Visit for each participant (for up to approximately 132 weeks)

Secondary outcomes (7)

  1. Amyotrophic Lateral Sclerosis Rating Scale Revised Total Score (ALSFRS-R) Change in Slope

    Comparison of the difference in change in slope of Amyotrophic Lateral Sclerosis Rating Scale Revised (ALSFRS-R) total score from the Main Study baseline through Week 48 of the open-label extension (OLE) between the 2 treatment groups: those randomized to AMX0035 in the Main Study and those randomized to Placebo in the main study (all participants in the OLE received AMX0035). The slope is measured as a change in score divided by a change in time. ALSFRS-R consists of 12 items across 4 subdomains of function (bulbar, fine motor, gross motor, and breathing) with each item scored on a scale from 0 (total loss of function) to 4 (no loss of function). Total scores range from 0 to 48, with higher scores indicating better function.

    Measured: From baseline in the Main Study through Week 24 of the OLE (48 weeks overall)

  2. Survival - Time to Death

    Median survival in months

    Measured: From date of first dose in the Main Study (each participant's Baseline Visit) through up to approximately 42 months (approximately 182 weeks)

  3. Composite of Time to Hospitalization, Death or Death Equivalent

    The composite endpoint of time to hospitalization, death or death equivalent was defined as hospitalization, death, tracheostomy or PAV. PAV is defined as more than 22 hours daily of non-invasive mechanical ventilation for more than 1 week (7 days).

    Measured: From date of first dose in the Main Study (each participant's Baseline Visit) through up to approximately 42 months (approximately 182 weeks)

  4. Accurate Testing of Limb Isometric Strength (ATLIS) Change in Slope - Upper Extremities

    Comparison of the difference in change in slope of Accurate Testing of Limb Isometric Strength (ATLIS) upper extremity score from the Main Study baseline through Week 48 of the open-label extension (OLE) between the 2 treatment groups: those randomized to AMX0035 in the Main Study and those randomized to Placebo in the main study (all participants in the OLE received AMX0035). The ATLIS device measures isometric strength in 6 upper extremity muscle groups. Raw values were standardized to percent predicted normal values for strength based on sex, age, weight, and height.

    Measured: From baseline in the Main Study through Week 24 of the OLE (48 weeks overall)

  5. Accurate Testing of Limb Isometric Strength (ATLIS) Change in Slope - Lower Extremities

    Comparison of the difference in change in slope of Accurate Testing of Limb Isometric Strength (ATLIS) lower extremity score from the Main Study baseline through Week 48 of the open-label extension (OLE) between the 2 treatment groups: those randomized to AMX0035 in the Main Study and those randomized to Placebo in the main study (all participants in the OLE received AMX0035). The ATLIS device measures isometric strength in 6 lower extremity muscle groups. Raw values were standardized to percent predicted normal values for strength based on sex, age, weight, and height.

    Measured: From baseline in the Main Study through Week 24 of the OLE (48 weeks overall)

  6. Slow Vital Capacity Change in Slope

    Comparison of the difference in change in slope of Slow Vital Capacity (SVC) from the Main Study baseline through Week 48 of the open-label extension (OLE) between the 2 treatment groups: those randomized to AMX0035 in the Main Study and those randomized to Placebo in the main study (all participants in the OLE received AMX0035). SVC volumes were standardized to predicted percent normalized values for respiratory muscle function based on age, sex, and height.

    Measured: From baseline in the Main Study through Week 24 of the OLE (48 weeks overall)

  7. Accurate Testing of Limb Isometric Strength (ATLIS) Total Score Change in Slope

    Comparison of the difference in change in slope of Accurate Testing of Limb Isometric Strength (ATLIS) total score from the Main Study baseline through Week 48 of the open-label extension (OLE) between the 2 treatment groups: those randomized to AMX0035 in the Main Study and those randomized to Placebo in the main study (all participants in the OLE received AMX0035). The ATLIS device measures isometric strength in 6 upper and 6 lower extremity muscle groups. Raw values were standardized to percent predicted normal values for strength based on sex, age, weight, and height.

    Measured: From Baseline in the Main Study through Week 24 in the OLE (48 weeks overall)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted5 Apr 2018
  2. Study start29 Mar 2018
  3. Primary completion1 Mar 2021
  4. Study completion1 Mar 2021

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Model description
This is an open-label extension study to CENTAUR (AMX3500)
Masking
None (open label)
Enrolment
90 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03488524
Sponsor's protocol ID
AMX-3500-OLE
Acronym
CENTAUR-OLE
Status last verified
Jul 2025
First submitted
23 Mar 2018
Last updated
13 Aug 2025
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Paganoni S, Watkins C, Cawson M, Hendrix S, Dickson SP, Knowlton N, Timmons J, Manuel M, Cudkowicz M. Survival analyses from the CENTAUR trial in amyotrophic lateral sclerosis: Evaluating the impact of treatment crossover on outcomes. Muscle Nerve. 2022 Aug;66(2):136-141. doi: 10.1002/mus.27569. Epub 2022 May 31.

    PubMed 35508892

  • Paganoni S, Hendrix S, Dickson SP, Knowlton N, Berry JD, Elliott MA, Maiser S, Karam C, Caress JB, Owegi MA, Quick A, Wymer J, Goutman SA, Heitzman D, Heiman-Patterson TD, Jackson C, Quinn C, Rothstein JD, Kasarskis EJ, Katz J, Jenkins L, Ladha SS, Miller TM, Scelsa SN, Vu TH, Fournier C, Johnson KM, Swenson A, Goyal N, Pattee GL, Babu S, Chase M, Dagostino D, Hall M, Kittle G, Eydinov M, Ostrow J, Pothier L, Randall R, Shefner JM, Sherman AV, Tustison E, Vigneswaran P, Yu H, Cohen J, Klee J, Tanzi R, Gilbert W, Yeramian P, Cudkowicz M. Effect of sodium phenylbutyrate/taurursodiol on tracheostomy/ventilation-free survival and hospitalisation in amyotrophic lateral sclerosis: long-term results from the CENTAUR trial. J Neurol Neurosurg Psychiatry. 2022 May 16;93(8):871-5. doi: 10.1136/jnnp-2022-329024. Online ahead of print.

    PubMed 35577511

  • Paganoni S, Hendrix S, Dickson SP, Knowlton N, Macklin EA, Berry JD, Elliott MA, Maiser S, Karam C, Caress JB, Owegi MA, Quick A, Wymer J, Goutman SA, Heitzman D, Heiman-Patterson TD, Jackson CE, Quinn C, Rothstein JD, Kasarskis EJ, Katz J, Jenkins L, Ladha S, Miller TM, Scelsa SN, Vu TH, Fournier CN, Glass JD, Johnson KM, Swenson A, Goyal NA, Pattee GL, Andres PL, Babu S, Chase M, Dagostino D, Hall M, Kittle G, Eydinov M, McGovern M, Ostrow J, Pothier L, Randall R, Shefner JM, Sherman AV, St Pierre ME, Tustison E, Vigneswaran P, Walker J, Yu H, Chan J, Wittes J, Yu ZF, Cohen J, Klee J, Leslie K, Tanzi RE, Gilbert W, Yeramian PD, Schoenfeld D, Cudkowicz ME. Long-term survival of participants in the CENTAUR trial of sodium phenylbutyrate-taurursodiol in amyotrophic lateral sclerosis. Muscle Nerve. 2021 Jan;63(1):31-39. doi: 10.1002/mus.27091. Epub 2020 Oct 30.

    PubMed 33063909

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing AMX0035 in people with Amyotrophic Lateral Sclerosis and ALS, run by Amylyx Pharmaceuticals Inc. at 24 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 13 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 24 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 13 Aug 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants With Treatment-Emergent Adverse Events (TEAEs) (time frame: From the Baseline Visit in the OLE study through Week 132 or the Early Discontinuation (Final Safety) Visit for each participant (for up to approximately 132 weeks)). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03488524, which also lists 3 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Aug 2025.

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