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| Name | Class |
|---|---|
| Massachusetts General Hospital | OTHER |
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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).
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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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| AMX0035 | Experimental | AMX0035 twice daily--a combination of Sodium Phenylbutyrate (3g) and Taurursodiol (1g) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| AMX0035 | Drug | Combination therapy of PB and TURSO |
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| Measure | Description | Time Frame |
|---|---|---|
| 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 | 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) |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Sabrina Paganoni, MD, PhD | Massachusetts General Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Barrow Neurological Institute-Dignity Health, St Joseph's Hospital and Medical Center | Phoenix | Arizona | 85013 | United States | ||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 35508892 | Result | 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. | |
| 35577511 | Result |
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To maintain the study blind, participants who entered the OLE initiated dosing in the OLE with 2 sachets of AMX0035 daily (ie, participants who switched from placebo to AMX0035 were not titrated using a starting dose of 1 sachet daily for the first 3 weeks, which was the starting dosing in the Main Study AMX3500).
Participants who completed the 24-week double-blind main study (Study AMX3500) on treatment were eligible to enter into the OLE.
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| ID | Title | Description |
|---|---|---|
| FG000 | AMX0035 | AMX0035 twice daily--a combination of Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO) |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Mar 30, 2020 | Jul 22, 2024 |
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This is an open-label extension study to CENTAUR (AMX3500)
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| From baseline in the Main Study through Week 24 of the OLE (48 weeks overall) |
| Survival - Time to Death | Median survival in months | From date of first dose in the Main Study (each participant's Baseline Visit) through up to approximately 42 months (approximately 182 weeks) |
| 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). | From date of first dose in the Main Study (each participant's Baseline Visit) through up to approximately 42 months (approximately 182 weeks) |
| 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. | From baseline in the Main Study through Week 24 of the OLE (48 weeks overall) |
| 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. | From baseline in the Main Study through Week 24 of the OLE (48 weeks overall) |
| 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. | From baseline in the Main Study through Week 24 of the OLE (48 weeks overall) |
| 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. | From Baseline in the Main Study through Week 24 in the OLE (48 weeks overall) |
| 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 |
| 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. |
| 33063909 | Result | 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. |
| COMPLETED |
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| NOT COMPLETED |
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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.
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| ID | Title | Description |
|---|---|---|
| BG000 | Placebo in Main Study-AMX0035 in OLE | AMX0035 twice daily--a combination of Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO) |
| BG001 | AMX0035 in Main Study-AMX0035 in OLE | AMX0035 twice daily--a combination of Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO) |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Body Mass Index | Mean | Standard Deviation | kg/m^2 |
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| Del-FS: Rate of disease progression prior to entering the study | DEL-FS score has been shown to be a strong predictor of functional decline. The score is calculated as the points lost on the ALSFRS-R from a maximum score of 48 divided by the months since symptom onset at baseline. | Population for this measure is the mITT population, which included included all participants who received at least 1 dose of study medication in the main study who had at least 1 post baseline total ALSFRS-R score available. | Mean | Standard Deviation | ScaleScore/Months post-symptom onset |
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| Time since ALS diagnosis | Population for this measure is the mITT population, which included included all participants who received at least 1 dose of study medication in the main study who had at least 1 post baseline total ALSFRS-R score available. | Mean | Standard Deviation | months |
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| Time since onset of ALS symptoms | Population for this measure is the mITT population, which included included all participants who received at least 1 dose of study medication in the main study who had at least 1 post baseline total ALSFRS-R score available. | Mean | Standard Deviation | months |
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| Use of either edaravone or riluzole at or prior to study entry | Population for this measure is the mITT population, which included included all participants who received at least 1 dose of study medication in the main study who had at least 1 post baseline total ALSFRS-R score available. | Count of Participants | Participants |
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| Slow vital capacity (SVC) - % Predicted | SVC volumes were standardized to predicted percent normalized values based on age, sex, and height. | Population for this measure is the mITT population, which included included all participants who received at least 1 dose of study medication in the main study who had at least 1 post baseline total ALSFRS-R score available. Not all participants had baseline data for this measure. | Mean | Standard Deviation | percent of predicted |
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| ALS Functional Rating Scale - Revised (ALSFRS-R) total score | The ALSFRS questionnaire is designed to determine the participant's assessment of their capability and independence in 12 functional activities relevant to ALS. 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. | Population for this measure is the mITT population, which included included all participants who received at least 1 dose of study medication in the main study who had at least 1 post baseline total ALSFRS-R score available. | Mean | Standard Deviation | scores on a scale |
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| Accurate Test of Limb Isometric Strength (ATLIS) upper extremity score | The ATLIS device measures isometric strength in 6 upper extremity muscle groups (right and left grip strength, elbow extension and flexion). Raw values were standardized to percent predicted normal values for strength based on sex, age, weight, and height. | Population for this measure is the mITT population, which included included all participants who received at least 1 dose of study medication in the main study who had at least 1 post baseline total ALSFRS-R score available. Not all participants had baseline data for this measure. | Mean | Standard Deviation | percent of predicted normal values |
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| Accurate Test of Limb Isometric Strength (ATLIS) lower extremity score | The ATLIS device measures isometric strength in 6 lower extremity muscle groups (left knee extension, right knee extension, left knee flexion, right knee flexion, left ankle dorsiflexion, right ankle dorsiflexion). Raw values were standardized to percent predicted normal values for strength based on sex, age, weight, and height. | Population for this measure is the mITT population, which included included all participants who received at least 1 dose of study medication in the main study who had at least 1 post baseline total ALSFRS-R score available. Not all participants had baseline data for this measure. | Mean | Standard Deviation | percent of predicted normal values |
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| Accurate Test of Limb Isometric Strength (ATLIS) total score | The ATLIS device measures isometric strength in 6 lower extremity and 6 upper extremity muscle groups. Raw values were standardized to percent predicted normal values for strength based on sex, age, weight, and height. | Population for this measure is the mITT population, which included included all participants who received at least 1 dose of study medication in the main study who had at least 1 post baseline total ALSFRS-R score available. Not all participants had baseline data for this measure. | Mean | Standard Deviation | percent of predicted normal values |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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 | Safety Population, which included all participants who received at least 1 dose of study medication in the OLE. | Posted | Count of Participants | Participants | 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) |
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| Secondary | 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. | mITT Population. Participants from the Main Study who did not enter the OLE were included. | Posted | Least Squares Mean | Standard Error | Change in ALSFRS-R Total Score per Month | From baseline in the Main Study through Week 24 of the OLE (48 weeks overall) |
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| Secondary | Survival - Time to Death | Median survival in months | ITT Population. Participants from the Main Study who did not enter the OLE were included. | Posted | Median | 95% Confidence Interval | Months | From date of first dose in the Main Study (each participant's Baseline Visit) through up to approximately 42 months (approximately 182 weeks) |
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| Secondary | 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). | ITT population. Participants from the Main Study who did not enter the OLE were included. | Posted | Median | 95% Confidence Interval | Months | From date of first dose in the Main Study (each participant's Baseline Visit) through up to approximately 42 months (approximately 182 weeks) |
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| Secondary | 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. | mITT Population. Participants from the Main Study who did not enter the OLE were included in this analysis. | Posted | Least Squares Mean | Standard Error | % of Predicted Normal Change per Month | From baseline in the Main Study through Week 24 of the OLE (48 weeks overall) |
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| Secondary | 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. | mITT Population. Participants from the Main Study who did not enter the OLE were included in this analysis. | Posted | Least Squares Mean | Standard Error | % of Predicted Normal Change per Month | From baseline in the Main Study through Week 24 of the OLE (48 weeks overall) |
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| Secondary | 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. | mITT Population. Participants from the Main Study who did not enroll in the OLE were included. | Posted | Least Squares Mean | Standard Error | % of Predicted Normal Change per Month | From baseline in the Main Study through Week 24 of the OLE (48 weeks overall) |
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| Secondary | 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. | mITT Population. Participants from the Main Study who did not enter the OLE were included. | Posted | Least Squares Mean | Standard Error | % of Predicted normal change per month | From Baseline in the Main Study through Week 24 in the OLE (48 weeks overall) |
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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)
Symptoms of progression/worsening of ALS, including 'normal' progression, were to be recorded as AEs.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | AMX0035 | AMX0035 twice daily--a combination of Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO) | 19 | 90 | 31 | 90 | 81 | 90 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA v23.1 | Systematic Assessment |
| |
| Pneumonia aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA v23.1 | Systematic Assessment |
| |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA v23.1 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA v23.1 | Systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA v23.1 | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA v23.1 | Systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA v23.1 | Systematic Assessment |
| |
| Catheter site infection | Infections and infestations | MedDRA v23.1 | Systematic Assessment |
| |
| Diverticulits | Infections and infestations | MedDRA v23.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA v23.1 | Systematic Assessment |
| |
| Medical device site infection | Infections and infestations | MedDRA v23.1 | Systematic Assessment |
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| Pneumonia | Infections and infestations | MedDRA v23.1 | Systematic Assessment |
| |
| Pneumonia bacterial | Infections and infestations | MedDRA v23.1 | Systematic Assessment |
| |
| Urosepsis | Infections and infestations | MedDRA v23.1 | Systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA v23.1 | Systematic Assessment |
| |
| Ileus | Gastrointestinal disorders | MedDRA v23.1 | Systematic Assessment |
| |
| Disease progression | General disorders | MedDRA v23.1 | Systematic Assessment |
| |
| Deep vein thrombosis | Vascular disorders | MedDRA v23.1 | Systematic Assessment |
| |
| Cardiac arrest | Cardiac disorders | MedDRA v23.1 | Systematic Assessment |
| |
| Cholecystitis acute | Hepatobiliary disorders | MedDRA v23.1 | Systematic Assessment |
| |
| Facial bones fracture | Injury, poisoning and procedural complications | MedDRA v23.1 | Systematic Assessment |
| |
| Musculoskeletal stiffness | Musculoskeletal and connective tissue disorders | MedDRA v23.1 | Systematic Assessment |
| |
| Amyotrophic lateral sclerosis | Nervous system disorders | MedDRA v23.1 | Systematic Assessment |
| |
| Device malfunction | Product Issues | MedDRA v23.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Nausea | Gastrointestinal disorders | MedDRA v23.1 | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA v23.1 | Systematic Assessment |
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| Constipation | Gastrointestinal disorders | MedDRA v23.1 | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA v23.1 | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA v23.1 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA v23.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA v23.1 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA v23.1 | Systematic Assessment |
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| Pneumonia | Infections and infestations | MedDRA v23.1 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA v23.1 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA v23.1 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA v23.1 | Systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA v23.1 | Systematic Assessment |
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| Weight decreased | Investigations | MedDRA v23.1 | Systematic Assessment |
| |
| Disease progression | General disorders | MedDRA v23.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA v23.1 | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA v23.1 | Systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Chief Medical Officer | Amylyx Pharmaceuticals, Inc. | (877) 374-1208 | medinfo@amylyx.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan: Primary SAP | Oct 31, 2019 | Jul 22, 2024 | SAP_001.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan: Integrated Survival Analysis SAP | Mar 27, 2020 | Jul 22, 2024 | SAP_002.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan: Addendum to Integrated Survival Analysis SAP | Aug 26, 2020 | Jul 22, 2024 | SAP_003.pdf |
Not provided
| ID | Term |
|---|---|
| D000690 | Amyotrophic Lateral Sclerosis |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D016472 | Motor Neuron Disease |
| D019636 | Neurodegenerative Diseases |
| D057177 | TDP-43 Proteinopathies |
| D009468 | Neuromuscular Diseases |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C000723627 | sodium phenylbutyrate and taurursodiol |
| C075773 | 4-phenylbutyric acid |
| C031655 | ursodoxicoltaurine |
Not provided
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