Trial · NCT05349721
Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS
NCT05349721, Phase 2, Terminated, PTC Therapeutics; results first posted 2025-08-01
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS. Trials, record NCT05349721. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05349721
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS
- NCT number
- NCT05349721
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 336 participants, actual
- Lead sponsor
- PTC Therapeutics
- Conditions
- Amyotrophic Lateral Sclerosis
- Interventions
- PTC857, Placebo
- First posted
- 2022-04-27
- Last updated
- 2025-08-01
- Other registries' IDs
- eudract:2021-006511-29
- Results first posted
- 2025-08-01
- Limitations and caveats
- The study was terminated early due to company decision.
Source ClinicalTrials.gov
Registered outcomes14
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Combined Assessment of Function (ALS Functional Rating Scale-Revised [ALSFRS-R]) and Survival (CAFS) Rank After 24 Weeks of Treatment (Intention-to-Treat [ITT] 1 Analysis Population) | Week 24 | The CAFS is a composite endpoint based on time to earlier occurrence of death and change from baseline in ALSFRS-R score. ALSFRS-R is a rating scale where 12 functions were rated on 5-point scales (from 0 to 4) with a maximum score of 48 (sum of all 12 items), with a higher score indicating better function. Each participant's outcome was compared to every other participant's outcome in a pairwise fashion by time to death and change on ALSFRS-R, assigned a score which was sum of comparisons (+1 \[better\], 0 \[tie\], -1 \[worse\]), and summed scores were ranked, from 1 to 306 (ITT1 Analysis Set) lowest rank corresponds to participant who died first and highest rank to the participant with best ALSFRS-R outcome among those who survived. Multiple imputation was used to impute participants with missing ALSFRS-R score at Week 24. A higher rank was considered a better outcome. Least square (LS) means and standard error (SE) were calculated using analysis of covariance (ANCOVA) model. |
| Secondary | Combined Assessment of Function (ALSFRS-R) and Survival (CAFS) Rank After 24 Weeks of Treatment (ITT2 Analysis Population) | Week 24 | The CAFS is a composite endpoint based on time to earlier occurrence of death and change from baseline in ALSFRS-R score. ALSFRS-R is a rating scale where 12 functions were rated on 5-point scales (from 0 to 4) with a maximum score of 48 (sum of all 12 items), with a higher score indicating better function. Each participant's outcome was compared to every other participant's outcome in a pairwise fashion by time to death and change on ALSFRS-R, assigned a score which was sum of comparisons (+1 \[better\], 0 \[tie\], -1 \[worse\]), and summed scores were ranked, from 1 to 334 (ITT2 Analysis Set) lowest rank corresponds to participant who died first and highest rank to the participant with best ALSFRS-R outcome among those who survived. A higher rank was considered a better outcome. LS mean and SE were calculated using ANCOVA model. |
| Secondary | Change From Baseline in ALSFRS-R Score at Week 24 (ITT1 Analysis Population) | Baseline, Week 24 | The ALSFRS-R is a quickly administered (5-minute) ordinal rating scale that assesses the participant's capability and independence in 12 functional activities across 4 subdomains of bodily function (bulbar, gross motor, fine motor, and respiratory) relevant in ALS. Each activity was recorded to the closest approximation from a list of 5 choices, scored 0 (total loss of function) to 4 (no loss of function), with the total score ranging from 0 to 48 and higher scores indicating less functional impairment. LS mean and SE were calculated using mixed model repeated measures (MMRM). |
| Secondary | Change From Baseline in ALSFRS-R Score at Week 24 (ITT2 Analysis Population) | Baseline, Week 24 | The ALSFRS-R is a quickly administered (5-minute) ordinal rating scale that assesses the participant's capability and independence in 12 functional activities across 4 subdomains of bodily function (bulbar, gross motor, fine motor, and respiratory) relevant in ALS. Each activity was recorded to the closest approximation from a list of 5 choices, scored 0 (total loss of function) to 4 (no loss of function), with the total score ranging from 0 to 48 and higher scores indicating less functional impairment. LS mean and SE were calculated using MMRM. |
| Secondary | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Day 1 through Week 24 | An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A TEAE was defined as an AE that began after the first administration of study drug or any existing AEs that worsened after the first dose of study drug. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'. |
| Secondary | Change From Baseline in Percent Predicted Slow Vital Capacity (SVC) at Week 24 | Baseline, Week 24 | The SVC is a measure of breathing function. SVC measures the volume that can be exhaled from a full inhalation after exhaling to a maximum as slowly as possible. The percent of predicted SVC is reported. LS mean and SE were calculated using MMRM. |
| Secondary | Change From Baseline in Modified Norris Scale Total Score at Week 24 | Baseline, Week 24 | Modified Norris scale is used to assess the motor/limb and bulbar function. The Modified Norris Scale is a rating scale for ALS that consists of 2 parts: the Limb Norris Scale and the Norris Bulbar Scale. The Limb Norris Scale has 21 items to evaluate extremity function, and the Norris Bulbar Scale has 13 items to evaluate bulbar function. Each item was rated in 4 ordinal categories, corresponding to the following values and ratings or functional scores: normal (3 points), somewhat impaired (2 points), inadequate (1 point), and "cannot do at all" (0 points). The total score was calculated by summing all the scales, ranging from 0 (worst) to 102 (best) where higher scores indicated better functional abilities. LS mean and SE were calculated using MMRM. |
| Secondary | Overall Survival Rate | Baseline to Week 24 | Overall survival rate was defined as percentage of participants who were alive at given timepoint. Overall survival was defined as the time in months from the date of first dose to the date of death from any cause or date last known alive for those who did not die. |
| Secondary | Overall Survival | Baseline to Week 24 | Overall survival was defined as the time in months from the date of first dose to the date of death from any cause or date last known alive for those who did not die. |
| Secondary | Change From Baseline in ALS Assessment Questionnaire (ALSAQ-40) Total Score at Week 24 | Baseline, Week 24 | The ALSAQ-40 is a disease-specific measure of health-related quality of life (QOL) for ALS. The ALSAQ-40 is comprised of 40 questions measuring 5 discrete dimensions of health status that are affected by the disease: physical mobility (10 items); activities of daily living and independence (10 items); eating and drinking (3 items); communication (7 items); emotional reactions (10 items). Participants were asked to indicate the frequency of each event by selecting one of 5 options (Likert scale: 0-4): never/rarely/sometimes/often/ always or cannot do at all. The total score ranging from 0 (no impairment) to 160 (severe impairment) was calculated by adding the 5 domain scores. A lower score indicates a better health state. LS mean and SE were calculated using MMRM. |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 registered outcomesOutcomes14
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Combined Assessment of Function (ALS Functional Rating Scale-Revised [ALSFRS-R]) and Survival (CAFS) Rank After 24 Weeks of Treatment (Intention-to-Treat [ITT] 1 Analysis Population) | Week 24 | ITT1 Analysis Set included all randomized participants who received at least 1 dose of double-blind study drug in the Treatment Period and who had a decrease in the ALSFRS-R score of ≤4 points during the Screening Period. | units on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Combined Assessment of Function (ALSFRS-R) and Survival (CAFS) Rank After 24 Weeks of Treatment (ITT2 Analysis Population) | Week 24 | ITT2 Analysis Set included all randomized participants who received at least 1 dose of double-blind study drug in the Treatment Period. | units on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in ALSFRS-R Score at Week 24 (ITT1 Analysis Population) | Baseline, Week 24 | ITT1 Analysis Set included all randomized participants who received at least 1 dose of double-blind study drug in the Treatment Period and who had a decrease in the ALSFRS-R score of ≤4 points during the Screening Period. | units on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in ALSFRS-R Score at Week 24 (ITT2 Analysis Population) | Baseline, Week 24 | ITT2 Analysis Set included all randomized participants who received at least 1 dose of double-blind study drug in the Treatment Period. | units on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Day 1 through Week 24 | Safety set included all randomized participants who received at least 1 dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in Percent Predicted Slow Vital Capacity (SVC) at Week 24 | Baseline, Week 24 | ITT1 Analysis Set included all randomized participants who received at least 1 dose of double-blind study drug in the Treatment Period and who had a decrease in the ALSFRS-R score of ≤4 points during the Screening Period. | percent predicted SVC | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in Modified Norris Scale Total Score at Week 24 | Baseline, Week 24 | ITT1 Analysis Set included all randomized participants who received at least 1 dose of double-blind study drug in the Treatment Period and who had a decrease in the ALSFRS-R score of ≤4 points during the Screening Period. | units on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Overall Survival Rate | Baseline to Week 24 | ITT1 Analysis Set included all randomized participants who received at least 1 dose of double-blind study drug in the Treatment Period and who had a decrease in the ALSFRS-R score of ≤4 points during the Screening Period. | percentage of participants | NUMBER |
| SECONDARY | Overall Survival | Baseline to Week 24 | ITT1 Analysis Set included all randomized participants who received at least 1 dose of double-blind study drug in the Treatment Period and who had a decrease in the ALSFRS-R score of ≤4 points during the Screening Period. | months | MEDIAN |
| SECONDARY | Change From Baseline in ALS Assessment Questionnaire (ALSAQ-40) Total Score at Week 24 | Baseline, Week 24 | ITT1 Analysis Set included all randomized participants who received at least 1 dose of double-blind study drug in the Treatment Period and who had a decrease in the ALSFRS-R score of ≤4 points during the Screening Period. | units on a scale | LEAST_SQUARES_MEAN |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 outcomesResults by group28
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Combined Assessment of Function (ALS Functional Rating Scale-Revised [ALSFRS-R]) and Survival (CAFS) Rank After 24 Weeks of Treatment (Intention-to-Treat [ITT] 1 Analysis Population) | Utreloxastat | 172.22 | Standard Error 13.25 | units on a scale | |
| Combined Assessment of Function (ALS Functional Rating Scale-Revised [ALSFRS-R]) and Survival (CAFS) Rank After 24 Weeks of Treatment (Intention-to-Treat [ITT] 1 Analysis Population) | Placebo | 165.36 | Standard Error 14.65 | units on a scale | |
| Combined Assessment of Function (ALSFRS-R) and Survival (CAFS) Rank After 24 Weeks of Treatment (ITT2 Analysis Population) | Utreloxastat | 188.58 | Standard Error 14.81 | units on a scale | |
| Combined Assessment of Function (ALSFRS-R) and Survival (CAFS) Rank After 24 Weeks of Treatment (ITT2 Analysis Population) | Placebo | 181.89 | Standard Error 15.96 | units on a scale | |
| Change From Baseline in ALSFRS-R Score at Week 24 (ITT1 Analysis Population) | Utreloxastat | 29.4 | Standard Error 0.73 | units on a scale | |
| Change From Baseline in ALSFRS-R Score at Week 24 (ITT1 Analysis Population) | Placebo | 31.4 | Standard Error 0.93 | units on a scale | |
| Change From Baseline in ALSFRS-R Score at Week 24 (ITT2 Analysis Population) | Utreloxastat | 27.7 | Standard Error 0.71 | units on a scale | |
| Change From Baseline in ALSFRS-R Score at Week 24 (ITT2 Analysis Population) | Placebo | 29.1 | Standard Error 0.88 | units on a scale | |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Utreloxastat | 174 | Participants | ||
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Placebo | 90 | Participants |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 results by groupParticipant flow24
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Part A (24 Weeks) | STARTED | Utreloxastat | 222 | |
| Part A (24 Weeks) | STARTED | Placebo | 114 | |
| Part A (24 Weeks) | Received at Least 1 Dose of Double-blind Study Drug in the Part A | Utreloxastat | 220 | |
| Part A (24 Weeks) | Received at Least 1 Dose of Double-blind Study Drug in the Part A | Placebo | 114 | |
| Part A (24 Weeks) | COMPLETED | Utreloxastat | 179 | |
| Part A (24 Weeks) | COMPLETED | Placebo | 100 | |
| Part A (24 Weeks) | NOT COMPLETED | Utreloxastat | 43 | |
| Part A (24 Weeks) | NOT COMPLETED | Placebo | 14 | |
| Part B (28 Weeks) | STARTED | Utreloxastat | 173 | |
| Part B (28 Weeks) | STARTED | Placebo | 100 |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 participant flowAdverse events1,864
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Diarrhoea | Gastrointestinal disorders | Part A: Utreloxastat | 21 | 220 | |
| other | Diarrhoea | Gastrointestinal disorders | Part A: Placebo | 17 | 114 | |
| other | Diarrhoea | Gastrointestinal disorders | Parts B and C: Utreloxastat/Utreloxastat | 6 | 173 | |
| other | Diarrhoea | Gastrointestinal disorders | Parts B and C: Placebo/ Utreloxastat | 5 | 100 | |
| other | Nausea | Gastrointestinal disorders | Part A: Utreloxastat | 17 | 220 | |
| other | Nausea | Gastrointestinal disorders | Part A: Placebo | 9 | 114 | |
| other | Nausea | Gastrointestinal disorders | Parts B and C: Utreloxastat/Utreloxastat | 7 | 173 | |
| other | Nausea | Gastrointestinal disorders | Parts B and C: Placebo/ Utreloxastat | 0 | 100 | |
| other | Constipation | Gastrointestinal disorders | Part A: Utreloxastat | 11 | 220 | |
| other | Constipation | Gastrointestinal disorders | Part A: Placebo | 6 | 114 |
1 to 10 of 1,864Source ClinicalTrials.gov
See all 1,864 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Part A: Utreloxastat | 9 | 220 |
| Total, all-cause mortality | Part A: Placebo | 3 | 114 |
| Total, all-cause mortality | Parts B and C: Utreloxastat/Utreloxastat | 23 | 173 |
| Total, all-cause mortality | Parts B and C: Placebo/ Utreloxastat | 15 | 100 |
| Total, other adverse events | Part A: Utreloxastat | 167 | 220 |
| Total, other adverse events | Part A: Placebo | 88 | 114 |
| Total, other adverse events | Parts B and C: Utreloxastat/Utreloxastat | 115 | 173 |
| Total, other adverse events | Parts B and C: Placebo/ Utreloxastat | 53 | 100 |
| Total, serious adverse events | Part A: Utreloxastat | 36 | 220 |
| Total, serious adverse events | Part A: Placebo | 11 | 114 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| Utreloxastat vs Placebo: Number of Participants With Treatment-emergent Adverse Events (TEAEs) |
Source Trial comparisons
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT05349721
GET https://conductscience.org/api/trials/v1/NCT05349721/results?page=2