Trial Outcomes & Findings for Open Label Extension Study of AMX0035 in Patients With ALS (NCT NCT03488524)
NCT ID: NCT03488524
Last Updated: 2025-08-13
Results Overview
Number of participants with TEAEs from baseline in the OLE study through the last participant's last visit in the OLE
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
90 participants
Primary outcome timeframe
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)
Results posted on
2025-08-13
Participant Flow
Participants who completed the 24-week double-blind main study (Study AMX3500) on treatment were eligible to enter into the OLE.
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).
Participant milestones
| Measure |
AMX0035
AMX0035 twice daily--a combination of Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO)
|
|---|---|
|
Overall Study
STARTED
|
90
|
|
Overall Study
COMPLETED
|
2
|
|
Overall Study
NOT COMPLETED
|
88
|
Reasons for withdrawal
| Measure |
AMX0035
AMX0035 twice daily--a combination of Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO)
|
|---|---|
|
Overall Study
Withdrawal by Subject
|
51
|
|
Overall Study
Death
|
19
|
|
Overall Study
Study Sponsor
|
11
|
|
Overall Study
Physician Decision
|
4
|
|
Overall Study
Lost to Follow-up
|
3
|
Baseline Characteristics
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.
Baseline characteristics by cohort
| Measure |
Placebo in Main Study-AMX0035 in OLE
n=34 Participants
AMX0035 twice daily--a combination of Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO)
|
AMX0035 in Main Study-AMX0035 in OLE
n=56 Participants
AMX0035 twice daily--a combination of Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO)
|
Total
n=90 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
58.5 years
STANDARD_DEVIATION 7.41 • n=34 Participants
|
57.8 years
STANDARD_DEVIATION 10.22 • n=56 Participants
|
58.0 years
STANDARD_DEVIATION 9.22 • n=90 Participants
|
|
Sex: Female, Male
Female
|
10 Participants
n=34 Participants
|
13 Participants
n=56 Participants
|
23 Participants
n=90 Participants
|
|
Sex: Female, Male
Male
|
24 Participants
n=34 Participants
|
43 Participants
n=56 Participants
|
67 Participants
n=90 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=34 Participants
|
4 Participants
n=56 Participants
|
5 Participants
n=90 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
33 Participants
n=34 Participants
|
52 Participants
n=56 Participants
|
85 Participants
n=90 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=34 Participants
|
0 Participants
n=56 Participants
|
0 Participants
n=90 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=34 Participants
|
0 Participants
n=56 Participants
|
0 Participants
n=90 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=34 Participants
|
2 Participants
n=56 Participants
|
3 Participants
n=90 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=34 Participants
|
0 Participants
n=56 Participants
|
0 Participants
n=90 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=34 Participants
|
1 Participants
n=56 Participants
|
1 Participants
n=90 Participants
|
|
Race (NIH/OMB)
White
|
33 Participants
n=34 Participants
|
52 Participants
n=56 Participants
|
85 Participants
n=90 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=34 Participants
|
0 Participants
n=56 Participants
|
0 Participants
n=90 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=34 Participants
|
1 Participants
n=56 Participants
|
1 Participants
n=90 Participants
|
|
Body Mass Index
|
27.1 kg/m^2
STANDARD_DEVIATION 6.50 • n=34 Participants
|
26.8 kg/m^2
STANDARD_DEVIATION 4.67 • n=56 Participants
|
26.9 kg/m^2
STANDARD_DEVIATION 5.40 • n=90 Participants
|
|
Del-FS: Rate of disease progression prior to entering the study
|
1.03 ScaleScore/Months post-symptom onset
STANDARD_DEVIATION 0.73 • n=32 Participants • 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.
|
0.92 ScaleScore/Months post-symptom onset
STANDARD_DEVIATION 0.46 • n=54 Participants • 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.
|
0.96 ScaleScore/Months post-symptom onset
STANDARD_DEVIATION 0.57 • n=86 Participants • 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.
|
|
Time since ALS diagnosis
|
11.9 months
STANDARD_DEVIATION 3.54 • n=32 Participants • 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.
|
11.7 months
STANDARD_DEVIATION 3.80 • n=54 Participants • 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.
|
11.8 months
STANDARD_DEVIATION 3.69 • n=86 Participants • 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.
|
|
Time since onset of ALS symptoms
|
19.2 months
STANDARD_DEVIATION 3.72 • n=32 Participants • 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.
|
19.4 months
STANDARD_DEVIATION 4.29 • n=54 Participants • 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.
|
19.3 months
STANDARD_DEVIATION 4.07 • n=86 Participants • 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.
|
|
Use of either edaravone or riluzole at or prior to study entry
|
28 Participants
n=32 Participants • 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.
|
45 Participants
n=54 Participants • 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.
|
73 Participants
n=86 Participants • 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.
|
|
Slow vital capacity (SVC) - % Predicted
|
72.7 percent of predicted
STANDARD_DEVIATION 21.22 • n=29 Participants • 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.
|
71.2 percent of predicted
STANDARD_DEVIATION 23.12 • n=53 Participants • 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.
|
71.7 percent of predicted
STANDARD_DEVIATION 22.34 • n=82 Participants • 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.
|
|
ALS Functional Rating Scale - Revised (ALSFRS-R) total score
|
29.8 scores on a scale
STANDARD_DEVIATION 9.36 • n=32 Participants • 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.
|
30.6 scores on a scale
STANDARD_DEVIATION 8.42 • n=54 Participants • 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.
|
30.3 scores on a scale
STANDARD_DEVIATION 8.74 • n=86 Participants • 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.
|
|
Accurate Test of Limb Isometric Strength (ATLIS) upper extremity score
|
43.29 percent of predicted normal values
STANDARD_DEVIATION 23.24 • n=28 Participants • 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.
|
39.91 percent of predicted normal values
STANDARD_DEVIATION 23.74 • n=45 Participants • 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.
|
41.21 percent of predicted normal values
STANDARD_DEVIATION 23.45 • n=73 Participants • 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.
|
|
Accurate Test of Limb Isometric Strength (ATLIS) lower extremity score
|
51.89 percent of predicted normal values
STANDARD_DEVIATION 25.22 • n=28 Participants • 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.
|
49.98 percent of predicted normal values
STANDARD_DEVIATION 22.52 • n=45 Participants • 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.
|
50.72 percent of predicted normal values
STANDARD_DEVIATION 23.45 • n=73 Participants • 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.
|
|
Accurate Test of Limb Isometric Strength (ATLIS) total score
|
43.21 percent of predicted normal values
STANDARD_DEVIATION 23.20 • n=28 Participants • 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.
|
39.82 percent of predicted normal values
STANDARD_DEVIATION 23.69 • n=45 Participants • 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.
|
41.12 percent of predicted normal values
STANDARD_DEVIATION 23.40 • n=73 Participants • 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.
|
PRIMARY outcome
Timeframe: 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)Population: Safety Population, which included all participants who received at least 1 dose of study medication in the OLE.
Number of participants with TEAEs from baseline in the OLE study through the last participant's last visit in the OLE
Outcome measures
| Measure |
Randomized to Placebo in Main Study and Did or Did Not Participate in OLE
n=90 Participants
AMX0035 twice daily--a combination of Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO)
|
Randomized to AMX0035 in Main Study and Did or Did Not Participate in OLE
AMX0035 twice daily--a combination of Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO)
|
|---|---|---|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
|
81 Participants
|
—
|
SECONDARY outcome
Timeframe: From baseline in the Main Study through Week 24 of the OLE (48 weeks overall)Population: mITT Population. Participants from the Main Study who did not enter the OLE were included.
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.
Outcome measures
| Measure |
Randomized to Placebo in Main Study and Did or Did Not Participate in OLE
n=48 Participants
AMX0035 twice daily--a combination of Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO)
|
Randomized to AMX0035 in Main Study and Did or Did Not Participate in OLE
n=87 Participants
AMX0035 twice daily--a combination of Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO)
|
|---|---|---|
|
Amyotrophic Lateral Sclerosis Rating Scale Revised Total Score (ALSFRS-R) Change in Slope
|
17.38 Change in ALSFRS-R Total Score per Month
Standard Error 1.545
|
21.61 Change in ALSFRS-R Total Score per Month
Standard Error 1.178
|
SECONDARY outcome
Timeframe: From date of first dose in the Main Study (each participant's Baseline Visit) through up to approximately 42 months (approximately 182 weeks)Population: ITT Population. Participants from the Main Study who did not enter the OLE were included.
Median survival in months
Outcome measures
| Measure |
Randomized to Placebo in Main Study and Did or Did Not Participate in OLE
n=48 Participants
AMX0035 twice daily--a combination of Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO)
|
Randomized to AMX0035 in Main Study and Did or Did Not Participate in OLE
n=89 Participants
AMX0035 twice daily--a combination of Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO)
|
|---|---|---|
|
Survival - Time to Death
|
18.7 Months
Interval 13.5 to 24.4
|
23.5 Months
Interval 19.5 to 26.2
|
SECONDARY outcome
Timeframe: From date of first dose in the Main Study (each participant's Baseline Visit) through up to approximately 42 months (approximately 182 weeks)Population: ITT population. Participants from the Main Study who did not enter the OLE were included.
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).
Outcome measures
| Measure |
Randomized to Placebo in Main Study and Did or Did Not Participate in OLE
n=48 Participants
AMX0035 twice daily--a combination of Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO)
|
Randomized to AMX0035 in Main Study and Did or Did Not Participate in OLE
n=89 Participants
AMX0035 twice daily--a combination of Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO)
|
|---|---|---|
|
Composite of Time to Hospitalization, Death or Death Equivalent
|
10.0 Months
Interval 6.1 to 12.7
|
14.8 Months
Interval 10.7 to 18.2
|
SECONDARY outcome
Timeframe: From baseline in the Main Study through Week 24 of the OLE (48 weeks overall)Population: mITT Population. Participants from the Main Study who did not enter the OLE were included in this analysis.
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.
Outcome measures
| Measure |
Randomized to Placebo in Main Study and Did or Did Not Participate in OLE
n=48 Participants
AMX0035 twice daily--a combination of Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO)
|
Randomized to AMX0035 in Main Study and Did or Did Not Participate in OLE
n=87 Participants
AMX0035 twice daily--a combination of Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO)
|
|---|---|---|
|
Accurate Testing of Limb Isometric Strength (ATLIS) Change in Slope - Upper Extremities
|
12.06 % of Predicted Normal Change per Month
Standard Error 3.283
|
19.83 % of Predicted Normal Change per Month
Standard Error 2.591
|
SECONDARY outcome
Timeframe: From baseline in the Main Study through Week 24 of the OLE (48 weeks overall)Population: mITT Population. Participants from the Main Study who did not enter the OLE were included in this analysis.
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.
Outcome measures
| Measure |
Randomized to Placebo in Main Study and Did or Did Not Participate in OLE
n=48 Participants
AMX0035 twice daily--a combination of Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO)
|
Randomized to AMX0035 in Main Study and Did or Did Not Participate in OLE
n=87 Participants
AMX0035 twice daily--a combination of Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO)
|
|---|---|---|
|
Accurate Testing of Limb Isometric Strength (ATLIS) Change in Slope - Lower Extremities
|
20.48 % of Predicted Normal Change per Month
Standard Error 3.653
|
25.24 % of Predicted Normal Change per Month
Standard Error 2.893
|
SECONDARY outcome
Timeframe: From baseline in the Main Study through Week 24 of the OLE (48 weeks overall)Population: mITT Population. Participants from the Main Study who did not enroll in the OLE were included.
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.
Outcome measures
| Measure |
Randomized to Placebo in Main Study and Did or Did Not Participate in OLE
n=48 Participants
AMX0035 twice daily--a combination of Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO)
|
Randomized to AMX0035 in Main Study and Did or Did Not Participate in OLE
n=87 Participants
AMX0035 twice daily--a combination of Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO)
|
|---|---|---|
|
Slow Vital Capacity Change in Slope
|
37.85 % of Predicted Normal Change per Month
Standard Error 4.427
|
48.52 % of Predicted Normal Change per Month
Standard Error 3.356
|
SECONDARY outcome
Timeframe: From Baseline in the Main Study through Week 24 in the OLE (48 weeks overall)Population: mITT Population. Participants from the Main Study who did not enter the OLE were included.
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.
Outcome measures
| Measure |
Randomized to Placebo in Main Study and Did or Did Not Participate in OLE
n=48 Participants
AMX0035 twice daily--a combination of Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO)
|
Randomized to AMX0035 in Main Study and Did or Did Not Participate in OLE
n=87 Participants
AMX0035 twice daily--a combination of Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO)
|
|---|---|---|
|
Accurate Testing of Limb Isometric Strength (ATLIS) Total Score Change in Slope
|
16.65 % of Predicted normal change per month
Standard Error 2.973
|
22.84 % of Predicted normal change per month
Standard Error 2.371
|
Adverse Events
AMX0035
Serious events: 31 serious events
Other events: 81 other events
Deaths: 19 deaths
Serious adverse events
| Measure |
AMX0035
n=90 participants at risk
AMX0035 twice daily--a combination of Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO)
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
12.2%
11/90 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia aspiration
|
4.4%
4/90 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory failure
|
1.1%
1/90 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
1.1%
1/90 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
1.1%
1/90 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
1.1%
1/90 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
|
1.1%
1/90 • 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.
|
|
Infections and infestations
Catheter site infection
|
1.1%
1/90 • 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.
|
|
Infections and infestations
Diverticulits
|
1.1%
1/90 • 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.
|
|
Infections and infestations
Gastroenteritis
|
1.1%
1/90 • 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.
|
|
Infections and infestations
Medical device site infection
|
1.1%
1/90 • 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.
|
|
Infections and infestations
Pneumonia
|
1.1%
1/90 • 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.
|
|
Infections and infestations
Pneumonia bacterial
|
1.1%
1/90 • 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.
|
|
Infections and infestations
Urosepsis
|
1.1%
1/90 • 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.
|
|
Gastrointestinal disorders
Dysphagia
|
1.1%
1/90 • 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.
|
|
Gastrointestinal disorders
Ileus
|
1.1%
1/90 • 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.
|
|
General disorders
Disease progression
|
2.2%
2/90 • 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.
|
|
Vascular disorders
Deep vein thrombosis
|
2.2%
2/90 • 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.
|
|
Cardiac disorders
Cardiac arrest
|
1.1%
1/90 • 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.
|
|
Hepatobiliary disorders
Cholecystitis acute
|
1.1%
1/90 • 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.
|
|
Injury, poisoning and procedural complications
Facial bones fracture
|
1.1%
1/90 • 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.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal stiffness
|
1.1%
1/90 • 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.
|
|
Nervous system disorders
Amyotrophic lateral sclerosis
|
1.1%
1/90 • 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.
|
|
Product Issues
Device malfunction
|
1.1%
1/90 • 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.
|
Other adverse events
| Measure |
AMX0035
n=90 participants at risk
AMX0035 twice daily--a combination of Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO)
|
|---|---|
|
Gastrointestinal disorders
Nausea
|
17.8%
16/90 • 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.
|
|
Gastrointestinal disorders
Diarrhoea
|
16.7%
15/90 • 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.
|
|
Gastrointestinal disorders
Constipation
|
11.1%
10/90 • 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.
|
|
Gastrointestinal disorders
Vomiting
|
5.6%
5/90 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
12.2%
11/90 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
8.9%
8/90 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
5.6%
5/90 • 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.
|
|
Infections and infestations
Urinary tract infection
|
6.7%
6/90 • 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.
|
|
Infections and infestations
Pneumonia
|
5.6%
5/90 • 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.
|
|
Injury, poisoning and procedural complications
Fall
|
22.2%
20/90 • 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.
|
|
Nervous system disorders
Dizziness
|
7.8%
7/90 • 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.
|
|
Nervous system disorders
Headache
|
6.7%
6/90 • 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.
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
8.9%
8/90 • 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.
|
|
Investigations
Weight decreased
|
6.7%
6/90 • 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.
|
|
General disorders
Disease progression
|
5.6%
5/90 • 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.
|
|
General disorders
Pyrexia
|
5.6%
5/90 • 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.
|
|
Psychiatric disorders
Anxiety
|
7.8%
7/90 • 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.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place