AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS) (CENTAUR)
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ClinicalTrials.gov Identifier: NCT03127514 |
Recruitment Status :
Completed
First Posted : April 25, 2017
Last Update Posted : November 3, 2020
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Condition or disease | Intervention/treatment | Phase |
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Amyotrophic Lateral Sclerosis Motor Neuron Disease Neuromuscular Diseases Neurodegenerative Diseases Spinal Cord Diseases TDP-43 Proteinopathies Nervous System Diseases Central Nervous System Diseases | Drug: AMX0035 Other: Placebo | Phase 2 Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 137 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Primary Purpose: | Treatment |
Official Title: | Evaluation of the Safety, Tolerability, Efficacy and Activity of AMX0035, a Fixed Combination of Phenylbutyrate (PB) and Tauroursodeoxycholic Acid (TUDCA), for the Treatment of ALS |
Actual Study Start Date : | June 22, 2017 |
Actual Primary Completion Date : | September 25, 2019 |
Actual Study Completion Date : | November 24, 2019 |

Arm | Intervention/treatment |
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Placebo Comparator: Placebo
Placebo administered twice daily p.o. for 24 weeks
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Other: Placebo
Matching Placebo Comparator |
Experimental: AMX0035
AMX0035 administered twice daily p.o. for 24 weeks
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Drug: AMX0035
AMX0035
Other Name: Proprietary formulation of taurursodiol and sodium phenylbutyrate |
- ALSFRS-R Slope [ Time Frame: 24 Weeks ]Change in slope of ALS Functional Rating Scale over treatment duration
- Incidence of Adverse Events [ Time Frame: 24 Weeks ]Comparison Between Groups of Incidence of Adverse Events Until Planned Completion
- Proportion of subjects in each group able to remain on study drug until planned discontinuation [ Time Frame: 24 weeks ]A comparison of the proportion of subjects in each group able to remain on study drug until planned discontinuation between groups
- Accurate Testing of Limb Isometric Strength (ATLIS) [ Time Frame: 24 Weeks ]Change in rate of decline of isometric muscle strength over treatment duration
- Blood-based Biomarkers [ Time Frame: 24 Weeks ]Change in levels of plasma-based biomarkers of neuronal death and axonal integrity from baseline to week 24
- Slow Vital Capacity [ Time Frame: 24 Weeks ]Change in slope of slow vital capacity decline over treatment duration
- Survival, tracheostomy and hospitalizations [ Time Frame: 24 Weeks ]Comparison of rate of occurrence between groups
- Imaging Biomarkers [ Time Frame: 24 Weeks ]

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Ages Eligible for Study: | 18 Years to 80 Years (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Key Inclusion Criteria:
- Male or female, aged 18-80 years of age
- Sporadic or familial ALS diagnosed as definite as defined by the World Federation of Neurology revised El Escorial criteria
- Less than or equal to 18 months since ALS symptom onset
- Capable of providing informed consent and following trial procedures
- Slow Vital Capacity (SVC) >60% of predicted value for gender, height, and age at the Screening Visit
- Subjects must either not take riluzole or be on a stable dose of riluzole for at least 30 days prior to the Screening Visit. Riluzole-naïve subjects are permitted in the study.
- Women of child bearing potential (e.g. not post-menopausal for at least one year or surgically sterile) must agree to use adequate birth control for the duration of the study and 3 months after last dose of study drug. Women must not be planning to become pregnant for the duration of the study and 3 months after last dose of study drug
- Men must agree to practice contraception for the duration of the study and 3 months after last dose of study drug. Men must not plan to father a child or provide for sperm donation for the duration of the study and 3 months after last dose of study drug
Key Exclusion Criteria:
- Presence of tracheostomy
- Exposure to PB, Taurursodiol or UDCA within 3 months prior to the Screening Visit or planning to use these medications during the course of the study
- History of known allergy to PB or bile salts
- Abnormal liver function defined as AST and/or ALT > 3 times the upper limit of the normal
- Renal insufficiency as defined by a serum creatinine > 1.5 times the upper limit of normal
- Poorly controlled arterial hypertension (SBP>160mmHg or DBP>100mmHg) at the Screening Visit
- Pregnant women or women currently breastfeeding
- History of cholecystectomy
- Biliary disease which impedes biliary flow including active cholecystitis, primary biliary cirrhosis, sclerosing cholangitis, gallbladder cancer, gallbladder polyps, gangrene of the gallbladder, abscess of the gallbladder.
- History of Class III/IV heart failure (per New York Heart Association - NYHA)
- Severe pancreatic or intestinal disorders that may alter the enterohepatic circulation and absorption of TUDCA including biliary infections, pancreatitis and ileal resection
- The presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that would impair ability of the subject to provide informed consent, according to Site Investigator judgment
- Clinically significant unstable medical condition (other than ALS) that would pose a risk to the subject if they were to participate in the study
- Active participation in an ALS clinical trial evaluating a small molecule within 30 days of the Screening Visit
- Exposure at any time to any biologic under investigation for the treatment of subjects with ALS (off-label use or investigational) including cell therapies, gene therapies, and monoclonal antibodies.
- Implantation of Diaphragm Pacing System (DPS)

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03127514

Study Director: | Patrick Yeramian, MD | Amylyx Pharmaceuticals Inc. | |
Principal Investigator: | Sabrina Paganoni, MD | Massachusetts General Hospital |
Publications:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: | Amylyx Pharmaceuticals Inc. |
ClinicalTrials.gov Identifier: | NCT03127514 |
Other Study ID Numbers: |
AMX-3500 |
First Posted: | April 25, 2017 Key Record Dates |
Last Update Posted: | November 3, 2020 |
Last Verified: | October 2020 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Undecided |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Randomized Double-blind Placebo-controlled |
Amyotrophic Lateral Sclerosis Sodium Phenylbutyrate Tauroursodeoxycholic Acid |
Motor Neuron Disease Amyotrophic Lateral Sclerosis Nervous System Diseases Neuromuscular Diseases Neurodegenerative Diseases Central Nervous System Diseases Spinal Cord Diseases |
TDP-43 Proteinopathies Sclerosis Pathologic Processes Proteostasis Deficiencies Metabolic Diseases 4-phenylbutyric acid Antineoplastic Agents |