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CompletedNCT03488524CENTAUR-OLEUpdated Aug 13, 2025Results posted

Open Label Extension Study of AMX0035 in Patients With ALS

A Phase 2 interventional study of AMX0035 in Amyotrophic Lateral Sclerosis and ALS, sponsored by Amylyx Pharmaceuticals Inc.. Completed at 24 sites in United States. Per ClinicalTrials.gov, last updated 2025-08-13.

Sponsored by Amylyx Pharmaceuticals Inc. · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
90
Allocation
Not applicable
Sex
All
01

Study 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 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.

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Conditions studied

  • Amyotrophic Lateral Sclerosis
  • ALS
03

In context

Amyotrophic Lateral Sclerosis

978 studies on the registry are indexed under Amyotrophic Lateral Sclerosis; 277 are open to participants now.

This study's enrollment of 90 is above the median of 38 across 662 interventional studies indexed under Amyotrophic Lateral Sclerosis.

Browse Amyotrophic Lateral Sclerosis studies →

Lead sponsor

Amylyx Pharmaceuticals Inc. is the lead sponsor of 13 studies on the registry; 2 are open to participants now.

Of its 6 completed or terminated interventional studies of FDA-regulated products, 3 (50%) have results posted.

Counted across the registry records on this site, refreshed daily.

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Who can participate

Ages eligible
Child (0–17), Adult (18–64), Older adult (65+)
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  1. 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.
  2. Must enroll in the OLE within 28 days of the Week 24 visit of the main study.
  3. Signed to enter the OLE phase.

Exclusion criteria

Exclusion Criteria:

  1. Discontinued study drug prematurely in the double-blind phase of the study for reasons other than tracheostomy or PAV.
  2. Exposure to or anticipated requirement for any disallowed medication listed in the protocol.
  3. Any ongoing adverse events that in the opinion of the Site Investigator are clear to the study drug.
  4. Unstable cardiac or other life-threatening disease emergent during the randomized, double blind study
  5. Any major medical condition that in the opinion of the Site Investigator would interfere with the study and place the subject at increased risk.
Terms in these criteria, in plain language(2)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

Contraindication

A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
90 participants (actual)

Study arms

  • Experimental
    AMX0035

    AMX0035 twice daily--a combination of Sodium Phenylbutyrate (3g) and Taurursodiol (1g)

    Drug: AMX0035

Interventions

  • DrugAMX0035

    Combination therapy of PB and TURSO

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

06

What researchers measure

Primary outcomes

  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

    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)

Secondary outcomes

  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.

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

  2. Survival - Time to Death

    Median survival in months

    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)

  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).

    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)

  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.

    Time frame: 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.

    Time frame: 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.

    Time frame: 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.

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

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Results

Posted Aug 13, 2025

Participant flow

Participants who completed the 24-week double-blind main study (Study AMX3500) on treatment were eligible to enter into the OLE.

Participant flow — Overall Study
MilestoneAMX0035
Started90
Completed2
Not completed88
Withdrew: Withdrawal by subject51
Withdrew: Death19
Withdrew: Study sponsor11
Withdrew: Physician decision4
Withdrew: Lost to follow-up3

Outcome measures

PrimaryNumber 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

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)
Reported as:
Count of participants · Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
ParticipantsAMX0035 in OLE
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)81
SecondaryAmyotrophic 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.

Time frame:
From baseline in the Main Study through Week 24 of the OLE (48 weeks overall)
Reported as:
Least squares mean · Change in ALSFRS-R Total Score per Month
Amyotrophic Lateral Sclerosis Rating Scale Revised Total Score (ALSFRS-R) Change in Slope
Change in ALSFRS-R Total Score per MonthRandomized to Placebo in Main Study and Did or Did Not Participate in OLERandomized to AMX0035 in Main Study and Did or Did Not Participate in OLE
Amyotrophic Lateral Sclerosis Rating Scale Revised Total Score (ALSFRS-R) Change in Slope17.38 ± 1.54521.61 ± 1.178
Statistical analysis
  • Randomized to Placebo in Main Study and Did or Did Not Participate in OLE vs Randomized to AMX0035 in Main Study and Did or Did Not Participate in OLE · Mixed Models Analysis · p = 0.0239 (Significance testing was 2-tailed and considered statistically significant if the calculated p-value was ≤0.05.) · Mean difference (final values): 4.23 · 95% CI 0.56 to 7.90Random-slope, shared-baseline, linear mixed model was adjusted for age and prebaseline ALSFRS-R slope.
SecondarySurvival - Time to Death

Median survival in months

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)
Reported as:
Median · Months
Survival - Time to Death
MonthsRandomized to Placebo in Main Study and Did or Did Not Participate in OLERandomized to AMX0035 in Main Study and Did or Did Not Participate in OLE
Survival - Time to Death18.7 (13.5 to 24.4)23.5 (19.5 to 26.2)
Statistical analysis
  • Randomized to Placebo in Main Study and Did or Did Not Participate in OLE vs Randomized to AMX0035 in Main Study and Did or Did Not Participate in OLE · Hazard ratio · p = 0.0475 · Hazard ratio (hr): 0.644 · 95% CI 0.416 to 0.995
SecondaryComposite 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).

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)
Reported as:
Median · Months
Composite of Time to Hospitalization, Death or Death Equivalent
MonthsRandomized to Placebo in Main Study and Did or Did Not Participate in OLERandomized to AMX0035 in Main Study and Did or Did Not Participate in OLE
Composite of Time to Hospitalization, Death or Death Equivalent10.0 (6.1 to 12.7)14.8 (10.7 to 18.2)
Statistical analysis
  • Randomized to Placebo in Main Study and Did or Did Not Participate in OLE vs Randomized to AMX0035 in Main Study and Did or Did Not Participate in OLE · Hazard Ratio · p = 0.0308 · Hazard ratio (hr): 0.621 · 95% CI 0.403 to 0.957
SecondaryAccurate 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.

Time frame:
From baseline in the Main Study through Week 24 of the OLE (48 weeks overall)
Reported as:
Least squares mean · % of Predicted Normal Change per Month
Accurate Testing of Limb Isometric Strength (ATLIS) Change in Slope - Upper Extremities
% of Predicted Normal Change per MonthRandomized to Placebo in Main Study and Did or Did Not Participate in OLERandomized to AMX0035 in Main Study and Did or Did Not Participate in OLE
Accurate Testing of Limb Isometric Strength (ATLIS) Change in Slope - Upper Extremities12.06 ± 3.28319.83 ± 2.591
Statistical analysis
  • Randomized to Placebo in Main Study and Did or Did Not Participate in OLE vs Randomized to AMX0035 in Main Study and Did or Did Not Participate in OLE · Mixed Models Analysis · p = 0.0291 · Mean difference (final values): 7.77 · 95% CI 0.80 to 14.75
SecondaryAccurate 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.

Time frame:
From baseline in the Main Study through Week 24 of the OLE (48 weeks overall)
Reported as:
Least squares mean · % of Predicted Normal Change per Month
Accurate Testing of Limb Isometric Strength (ATLIS) Change in Slope - Lower Extremities
% of Predicted Normal Change per MonthRandomized to Placebo in Main Study and Did or Did Not Participate in OLERandomized to AMX0035 in Main Study and Did or Did Not Participate in OLE
Accurate Testing of Limb Isometric Strength (ATLIS) Change in Slope - Lower Extremities20.48 ± 3.65325.24 ± 2.893
Statistical analysis
  • Randomized to Placebo in Main Study and Did or Did Not Participate in OLE vs Randomized to AMX0035 in Main Study and Did or Did Not Participate in OLE · Mixed Models Analysis · p = 0.2261 · Mean difference (final values): 4.76 · 95% CI -2.95 to 12.47
SecondarySlow 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.

Time frame:
From baseline in the Main Study through Week 24 of the OLE (48 weeks overall)
Reported as:
Least squares mean · % of Predicted Normal Change per Month
Slow Vital Capacity Change in Slope
% of Predicted Normal Change per MonthRandomized to Placebo in Main Study and Did or Did Not Participate in OLERandomized to AMX0035 in Main Study and Did or Did Not Participate in OLE
Slow Vital Capacity Change in Slope37.85 ± 4.42748.52 ± 3.356
Statistical analysis
  • Randomized to Placebo in Main Study and Did or Did Not Participate in OLE vs Randomized to AMX0035 in Main Study and Did or Did Not Participate in OLE · Mixed Models Analysis · p = 0.0372 (Nominal p-value) · Mean difference (final values): 10.66 · 95% CI 0.63 to 20.69
SecondaryAccurate 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.

Time frame:
From Baseline in the Main Study through Week 24 in the OLE (48 weeks overall)
Reported as:
Least squares mean · % of Predicted normal change per month
Accurate Testing of Limb Isometric Strength (ATLIS) Total Score Change in Slope
% of Predicted normal change per monthRandomized to Placebo in Main Study and Did or Did Not Participate in OLERandomized to AMX0035 in Main Study and Did or Did Not Participate in OLE
Accurate Testing of Limb Isometric Strength (ATLIS) Total Score Change in Slope16.65 ± 2.97322.84 ± 2.371
Statistical analysis
  • Randomized to Placebo in Main Study and Did or Did Not Participate in OLE vs Randomized to AMX0035 in Main Study and Did or Did Not Participate in OLE · Mixed Models Analysis · p = 0.0503 (Nominal p-value)

Adverse events

Collected over Adverse event data were collected from baseline in the OLE study through study completion (generally about 2.5 years). Vital status was collected from date of first dose in the Main Study (each participant's Baseline Visit) through up to approximately 42 months (approximately 182 weeks). Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
AMX003519/90 (21.1%)31/90 (34.4%)81/90 (90%)
Most frequent serious events
Showing 10 of 24
Most frequent serious events
EventAMX0035
Respiratory failureRespiratory, thoracic and mediastinal disorders11/90
Pneumonia aspirationRespiratory, thoracic and mediastinal disorders4/90
Disease progressionGeneral disorders2/90
Deep vein thrombosisVascular disorders2/90
Acute respiratory failureRespiratory, thoracic and mediastinal disorders1/90
DyspnoeaRespiratory, thoracic and mediastinal disorders1/90
HypoxiaRespiratory, thoracic and mediastinal disorders1/90
Pleural effusionRespiratory, thoracic and mediastinal disorders1/90
Pulmonary embolismRespiratory, thoracic and mediastinal disorders1/90
Catheter site infectionInfections and infestations1/90
Most frequent other events
Showing 10 of 17
Most frequent other events
EventAMX0035
FallInjury, poisoning and procedural complications20/90
NauseaGastrointestinal disorders16/90
DiarrhoeaGastrointestinal disorders15/90
Respiratory failureRespiratory, thoracic and mediastinal disorders11/90
ConstipationGastrointestinal disorders10/90
DyspnoeaRespiratory, thoracic and mediastinal disorders8/90
Muscular weaknessMusculoskeletal and connective tissue disorders8/90
DizzinessNervous system disorders7/90
AnxietyPsychiatric disorders7/90
Urinary tract infectionInfections and infestations6/90

Baseline characteristics

Participants from the Main Study (AMX3500) who participated in the OLE study. Demographic and baseline characteristics for the Main Study can be found in record NCT03127514.

Age, Continuous
Age, Continuous(years)Placebo in Main Study-AMX0035 in OLEAMX0035 in Main Study-AMX0035 in OLETotal
Mean58.5 ± 7.4157.8 ± 10.2258.0 ± 9.22
Sex: Female, Male
Sex: Female, Male(Participants)Placebo in Main Study-AMX0035 in OLEAMX0035 in Main Study-AMX0035 in OLETotal
Female101323
Male244367
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Placebo in Main Study-AMX0035 in OLEAMX0035 in Main Study-AMX0035 in OLETotal
Hispanic or Latino145
Not Hispanic or Latino335285
Unknown or Not Reported000
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Placebo in Main Study-AMX0035 in OLEAMX0035 in Main Study-AMX0035 in OLETotal
American Indian or Alaska Native000
Asian123
Native Hawaiian or Other Pacific Islander000
Black or African American011
White335285
More than one race000
Unknown or Not Reported011
Body Mass Index
Body Mass Index(kg/m^2)Placebo in Main Study-AMX0035 in OLEAMX0035 in Main Study-AMX0035 in OLETotal
Mean27.1 ± 6.5026.8 ± 4.6726.9 ± 5.40
Del-FS: Rate of disease progression prior to entering the study
Del-FS: Rate of disease progression prior to entering the study(ScaleScore/Months post-symptom onset)Placebo in Main Study-AMX0035 in OLEAMX0035 in Main Study-AMX0035 in OLETotal
Mean1.03 ± 0.730.92 ± 0.460.96 ± 0.57
Time since ALS diagnosis
Time since ALS diagnosis(months)Placebo in Main Study-AMX0035 in OLEAMX0035 in Main Study-AMX0035 in OLETotal
Mean11.9 ± 3.5411.7 ± 3.8011.8 ± 3.69
Time since onset of ALS symptoms
Time since onset of ALS symptoms(months)Placebo in Main Study-AMX0035 in OLEAMX0035 in Main Study-AMX0035 in OLETotal
Mean19.2 ± 3.7219.4 ± 4.2919.3 ± 4.07

6 further baseline measures are reported on the registry.

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Study locations

24 sites
  • Barrow Neurological Institute-Dignity Health, St Joseph's Hospital and Medical Center
    Phoenix, Arizona 85013, United States
  • University of California, Irvine
    Orange, California 92868, United States
  • California Pacific Medical Center
    San Francisco, California 94114, United States
  • University of Florida College of Medicine
    Gainesville, Florida 32610, United States
  • University of South Florida College of Medicine
    Tampa, Florida 33612, United States
  • Emory University Hospital
    Atlanta, Georgia 30322, United States
  • University of Iowa, Carver College of Medicine
    Iowa City, Iowa 52242, United States
  • University of Kentucky Medical Center
    Lexington, Kentucky 40536, United States
  • Ochsner Neuroscience Institute
    New Orleans, Louisiana 70121, United States
  • Johns Hopkins Hospital
    Baltimore, Maryland 21205, United States
  • Massachusetts General Hospital
    Boston, Massachusetts 02114, United States
  • University of Massachusetts Memorial Medical Center
    Worcester, Massachusetts 01655, United States
  • University of Michigan, Michigan Medicine
    Ann Arbor, Michigan 48109, United States
  • Hennepin County Medical Center
    Minneapolis, Minnesota 55415, United States
  • Washington University Medical Center
    St Louis, Missouri 63110, United States
  • Neurology Associates, PC
    Lincoln, Nebraska 68506, United States
  • Wake Forest School of Medicine
    Winston-Salem, North Carolina 27157, United States
  • The Ohio State University, Wexner Medical Center
    Columbus, Ohio 43221, United States
  • Oregon Health & Science University
    Portland, Oregon 97239, United States
  • The Penn Comprehensive Neuroscience Center
    Philadelphia, Pennsylvania 19107, United States
  • Temple University Hospital
    Philadelphia, Pennsylvania 19140, United States
  • Texas Neurology, PA
    Dallas, Texas 75214, United States
  • University of Texas Health Science Center, San Antonio
    San Antonio, Texas 78229, United States
  • Swedish Neuroscience Center
    Seattle, Washington 98122, United States
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References and documents

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 ↗

Study documents

PDFs hosted by the registry, ClinicalTrials.gov.

Individual participant data

Plan to share: No

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Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reachedSimilar studies: half by the dot; the band is the middle halfHalf of similar studies reached it by the dot; the band is the middle half, from a quarter to three quarters

Ran from Mar 2018 to primary completion in Mar 2021, 35 months.

Results were first posted in Aug 2025, 4 years 5 months after primary completion.

Of 36 industry-sponsored phase 2 interventional studies under Amyotrophic Lateral Sclerosis, half reached primary completion within 21 months of starting (the median), counting those still running; a quarter within 15 and three quarters within 35. This study's 35 months is above that.

Counting every sponsor, half of 73 studies reached it within 24 months; a quarter within 16 and three quarters within 35.

How these figures are worked out, and which studies are left out of them.

Registered
Mar 23, 2018
First posted
Apr 5, 2018
StartActual
Mar 29, 2018
Primary completionActual
Mar 1, 2021
CompletionActual
Mar 1, 2021
Results first posted
Aug 13, 2025
Last update
Aug 13, 2025
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Aug 13, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
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Registry details

Key details

Study ID
NCT03488524
Lead sponsor
Amylyx Pharmaceuticals Inc.
Collaborators
Massachusetts General Hospital
Responsible party
Sponsor
First posted
Apr 5, 2018
Start date
Mar 29, 2018
Primary completion
Mar 1, 2021
Completion
Mar 1, 2021
Results posted
Aug 13, 2025
Last update
Aug 13, 2025

Study contacts

Sabrina Paganoni, MD, PhD
principal investigator · Massachusetts General Hospital

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Jul 2025. You cannot join it, but the record below documents what was studied.

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Discussion

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Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Studies of the drug tested here: Sodium phenylbutyrate and taurursodiol

Other studies in this condition

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Other studies from this sponsor

Amylyx Pharmaceuticals Inc.

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