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
Results First Posted : August 11, 2021
Last Update Posted : August 11, 2021
|Condition or disease||Intervention/treatment||Phase|
|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|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||137 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|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|
Placebo Comparator: 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
Matching Placebo Comparator
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
Other Name: Proprietary formulation of taurursodiol and sodium phenylbutyrate
- Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Slope Change [ Time Frame: 24 Weeks ]Change in slope of Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) over treatment duration. The 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.
- Number of Participants With Adverse Events [ Time Frame: 24 Weeks ]Comparison Between Groups of Number of Participants With Adverse Events Until Planned Completion
- Number of Participants in Each Group Able to Remain on Study Drug Until Planned Discontinuation [ Time Frame: 24 weeks ]A comparison of the number of participants in each group able to remain on study drug until planned discontinuation between groups
- Accurate Testing of Limb Isometric Strength (ATLIS) Total Score Change [ Time Frame: 24 Weeks ]The ATLIS device assess the isometric muscle strength of six upper-limb and six lower-limb muscle groups. At least two trials are performed for each muscle group to assess change in rate of decline of isometric muscle strength over treatment duration. Values are standardized to the percentage of predicted normal strength based on sex, age, weight, and height. Results are presented as percent of predicted normal.
- Change in Plasma Levels of Phosphorylated Axonal Neurofilament H Subunit (pNF-H) [ Time Frame: 24 Weeks ]Neuronal degeneration releases phosphorylated axonal neurofilament H subunit (pNF-H) into the cerebrospinal fluid and subsequently the blood and is thought to be a potential biomarker of motor neuron degeneration; elevated plasma levels of pNF-H are presumed to correlate with neuronal injury. Change in levels of plasma pNF-H were measured from baseline to week 24
- Rate of Decline in Slow Vital Capacity (SVC) [ Time Frame: 24 Weeks ]Respiratory muscle function was assessed according to slow vital capacity (SVC). SVC was measured in an upright position for at least three trials per assessment. SVC volumes were standardized to the percentage of predicted normal value based on age, sex, and height.
- Death, Tracheostomy, and Hospitalization [ Time Frame: 24 Weeks ]The composite outcome was defined as death, a death-equivalent event (which consisted of only tracheostomy in one participant in this trial), or hospitalization, whichever occurred first; there were no instances of permanent ventilation delivered by noninvasive means in the study.
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|