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Completed

NCT Number: NCT03488524

Open Label Extension Study of AMX0035 in Patients With ALS

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).

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Key information

Conditions

Amyotrophic Lateral Sclerosis ALS Central Nervous System Diseases Metabolic Diseases Motor Neuron Disease Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Nutritional and Metabolic Diseases Proteostasis Deficiencies Spinal Cord Diseases TDP-43 Proteinopathies

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Barrow Neurological Institute-Dignity Health, St Joseph's Hospital and Medical Center, Phoenix, Arizona, United States

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.

Exclusion criteria

  • 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.

Treatment and study plan

AMX0035

Drug

Combination therapy of PB and TURSO

Other names: Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO, also known as Ursodoxicoltaurine or Tauroursodeoxycholic Acid [TUDCA])

Primary outcomes

  1. 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)

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

Secondary outcomes

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

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

    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.

  2. Survival - Time to Death

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

    Median survival in months

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

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

    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).

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

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

    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.

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

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

    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.

  6. Slow Vital Capacity Change in Slope

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

    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.

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

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

    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.

Sponsors and collaborators

Lead sponsor

Amylyx Pharmaceuticals Inc.

Industry

Collaborators

  • Massachusetts General Hospital

Registry information

Official study 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

Acronym: CENTAUR-OLE

Important dates

Study start
2018
Primary completion
2021
Study completion
2021
First posted
Apr 5, 2018
Registry last updated
Aug 13, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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