AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS)
NCT ID: NCT03127514
Last Updated: 2024-05-16
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2
137 participants
INTERVENTIONAL
2017-06-22
2019-11-24
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Placebo
Placebo administered by mouth or via feeding tube for 24 weeks: once daily for first 3 weeks and then twice daily for remainder of study if participant tolerating
Placebo
Matching Placebo Comparator
AMX0035
AMX0035 administered by mouth or via feeding tube for 24 weeks: once daily for first 3 weeks and then twice daily for remainder of study if participant tolerating
AMX0035
AMX0035
Interventions
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AMX0035
AMX0035
Placebo
Matching Placebo Comparator
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
2. Sporadic or familial ALS diagnosed as definite as defined by the World Federation of Neurology revised El Escorial criteria
3. Less than or equal to 18 months since ALS symptom onset
4. Capable of providing informed consent and following trial procedures
5. Slow Vital Capacity (SVC) \>60% of predicted value for gender, height, and age at the Screening Visit
6. 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.
7. 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
8. 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
Exclusion Criteria
2. 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
3. History of known allergy to PB or bile salts
4. Abnormal liver function defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) \> 3 times the upper limit of the normal
5. Renal insufficiency as defined by a serum creatinine \> 1.5 times the upper limit of normal
6. Poorly controlled arterial hypertension (systolic blood pressure (SBP)\>160mmHg or diastolic blood pressure (DBP)\>100mmHg) at the Screening Visit
7. Pregnant women or women currently breastfeeding
8. History of cholecystectomy
9. 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.
10. History of Class III/IV heart failure (per New York Heart Association - NYHA)
11. Severe pancreatic or intestinal disorders that may alter the enterohepatic circulation and absorption of TUDCA including biliary infections, pancreatitis and ileal resection
12. 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
13. Clinically significant unstable medical condition (other than ALS) that would pose a risk to the subject if they were to participate in the study
14. Active participation in an ALS clinical trial evaluating a small molecule within 30 days of the Screening Visit
15. 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.
16. Implantation of Diaphragm Pacing System (DPS)
18 Years
80 Years
ALL
No
Sponsors
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ALS Finding a Cure
OTHER
ALS Association
OTHER
Northeast ALS Consortium
OTHER
Neurological Clinical Research Institute at Massachusetts General Hospital
OTHER
Leandro P. Rizzuto Foundation
OTHER
Amylyx Pharmaceuticals Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Patrick Yeramian, MD
Role: STUDY_DIRECTOR
Amylyx Pharmaceuticals Inc.
Sabrina Paganoni, MD
Role: PRINCIPAL_INVESTIGATOR
Massachusetts General Hospital
Locations
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Barrow Neurological Institute
Phoenix, Arizona, United States
UC Irvine Medical Center
Orange, California, United States
Forbes Norris MDA/ALS Research Center - California Pacific Medical Center
San Francisco, California, United States
University of Florida Medical Center
Gainesville, Florida, United States
Carol and Frank Morsini Center for Advanced Health Care - University of South Florida
Tampa, Florida, United States
Emory University Hospital
Atlanta, Georgia, United States
University of Iowa Hospitals and Clinics
Iowa City, Iowa, United States
University of Kentucky Medical Center
Lexington, Kentucky, United States
Ochsner Neuroscience Institute
New Orleans, Louisiana, United States
Johns Hopkins Hospital
Baltimore, Maryland, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
University of Massachusetts Memorial Medical Center
Worcester, Massachusetts, United States
University of Michigan Medical Center
Ann Arbor, Michigan, United States
Hennepin County Medical Center
Minneapolis, Minnesota, United States
Washington University Medical Center
St Louis, Missouri, United States
Neurology Associates P.C.
Lincoln, Nebraska, United States
Mount Sinai Beth Israel
New York, New York, United States
Wake Forest Baptist Medical Center
Winston-Salem, North Carolina, United States
The Ohio State University Wexner Medical Center
Columbus, Ohio, United States
Oregon Health & Science University
Portland, Oregon, United States
The Penn Comprehensive ALS Center
Philadelphia, Pennsylvania, United States
Temple University Hospital
Philadelphia, Pennsylvania, United States
Texas Neurology, P.A.
Dallas, Texas, United States
University of Texas Health Science Center at San Antonio
San Antonio, Texas, United States
ALS Center at the Swedish Neuroscience Institute
Seattle, Washington, United States
Countries
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References
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Elia AE, Lalli S, Monsurro MR, Sagnelli A, Taiello AC, Reggiori B, La Bella V, Tedeschi G, Albanese A. Tauroursodeoxycholic acid in the treatment of patients with amyotrophic lateral sclerosis. Eur J Neurol. 2016 Jan;23(1):45-52. doi: 10.1111/ene.12664. Epub 2015 Feb 9.
Cudkowicz ME, Andres PL, Macdonald SA, Bedlack RS, Choudry R, Brown RH Jr, Zhang H, Schoenfeld DA, Shefner J, Matson S, Matson WR, Ferrante RJ; Northeast ALS and National VA ALS Research Consortiums. Phase 2 study of sodium phenylbutyrate in ALS. Amyotroph Lateral Scler. 2009 Apr;10(2):99-106. doi: 10.1080/17482960802320487.
Bowser R, An J, Mehta L, Chen J, Timmons J, Cudkowicz M, Paganoni S. Effect of sodium phenylbutyrate and taurursodiol on plasma concentrations of neuroinflammatory biomarkers in amyotrophic lateral sclerosis: results from the CENTAUR trial. J Neurol Neurosurg Psychiatry. 2024 Jun 17;95(7):605-608. doi: 10.1136/jnnp-2023-332106.
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.
Paganoni S, Macklin EA, Hendrix S, Berry JD, Elliott MA, Maiser S, Karam C, Caress JB, Owegi MA, Quick A, Wymer J, Goutman SA, Heitzman D, Heiman-Patterson T, 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, Dickson SP, Ellison N, Hall M, Hendrix K, Kittle G, McGovern M, Ostrow J, Pothier L, Randall R, Shefner JM, Sherman AV, Tustison E, Vigneswaran P, Walker J, Yu H, Chan J, Wittes J, Cohen J, Klee J, Leslie K, Tanzi RE, Gilbert W, Yeramian PD, Schoenfeld D, Cudkowicz ME. Trial of Sodium Phenylbutyrate-Taurursodiol for Amyotrophic Lateral Sclerosis. N Engl J Med. 2020 Sep 3;383(10):919-930. doi: 10.1056/NEJMoa1916945.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Related Links
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TUDCA in patients with ALS
Other Identifiers
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AMX-3500
Identifier Type: -
Identifier Source: org_study_id
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