Trial · NCT02496767
Ventilatory Investigation of Tirasemtiv and Assessment of Longitudinal Indices After Treatment for a Year
NCT02496767, Phase 3, Completed, Cytokinetics; results first posted 2020-06-19
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Ventilatory Investigation of Tirasemtiv and Assessment of Longitudinal Indices After Treatment for a Year. Trials, record NCT02496767. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02496767
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Ventilatory Investigation of Tirasemtiv and Assessment of Longitudinal Indices After Treatment for a Year
- NCT number
- NCT02496767
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 744 participants, actual
- Lead sponsor
- Cytokinetics
- Conditions
- Amyotrophic Lateral Sclerosis
- Interventions
- Tirasemtiv, Placebo tablets
- First posted
- 2015-07-14
- Last updated
- 2020-09-09
- Other registries' IDs
- eudract:2014-005413-23
- Results first posted
- 2020-06-19
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline to Week 24 of the Double-blind, Placebo-controlled Phase in Percent Predicted Slow Vital Capacity (SVC) | 24 weeks | SVC was measured using a spirometer (in units of liters). Following 3 to 5 breaths at rest, the patients were instructed to take as deep an inspiration as possible followed by a maximum exhalation (blowing out all the air in their lungs). Values obtained were converted to % predicted values (ie, the test result as a percent of predicted values for patients of similar demographic and baseline characteristics \[eg, height, age, sex\], based on Knudson 83 normative values). |
| Secondary | Change From Baseline in the ALSFRS-R Respiratory Domain Score at the End of 48 Weeks of Double-blind, Placebo-controlled Treatment | 48 weeks | The ALSFRS-R is used to measure the progression and severity of disease; it consists of 12 questions, assessing a patient's capability and independence in functional activities relevant to ALS, categorized in 4 domains: bulbar functions, fine motor tasks, gross motor tasks, and respiratory function. Respiratory function consists of 3 of the 12 questions, which assess dyspnea, orthopnea, and respiratory insufficiency. Each question is scored from 0 (indicating incapable or dependent) to 4 (normal). The sum of the response to these 3 questions represents the respiratory domain score. The respiratory domain score ranges from 0 to 12, with higher scores reflecting more normal function and lower scores reflecting more impaired function. |
| Secondary | Slope of Mega-score of Muscle Strength During the 48 Weeks of Double-blind, Placebo-controlled Treatment | 48 weeks | A hand-held dynomometer, with a scale of 0 to 300 pounds, was used to measure muscle strength and handgrip strength (bilateral); the muscle groups tested were: elbow flexion (bilateral), wrist extension (bilateral), knee extension (bilateral), and ankle dorsiflexion (bilateral). The muscle strength mega-score was calculated as the average of responses to all tested muscles as well as handgrip strength. The slope of muscle strength mega-score was the change over time (48 weeks) and analyzed using a mixed model that assumed a random slope effect. For this endpoint, negative values indicate a decline in muscle strength over time. |
| Secondary | Time to the First Occurrence of a Decline From Baseline in Percent Predicted SVC ≥ 20 Percentage Points or the Onset of Respiratory Insufficiency or Death All 48 Weeks of Double-blind, Placebo-controlled Treatment | 48 weeks | This endpoint evaluated the time to occurrence of a decline in percent predicted SVC (as measured by spirometry) of ≥ 20 percentage points, or the onset of respiratory insufficiency (defined as tracheostomy or the use of non-invasive ventilation for ≥ 22 hours per day for ≥10 consecutive days), or death, whichever was first, during the 48-week double-blind, placebo-controlled treatment phase. Note: The median time to a ≥ 20% decline in percent predicted SVC, onset of respiratory insufficiency, or death was 302 days for the placebo group and 359, 334, and 337 days for the 250 mg, 375 mg, and 500 mg tirasemtiv groups, respectively. The data presented for this endpoint are the number and percent of patients who met the endpoint. |
| Secondary | Time to the First Occurrence of a Decline in SVC to ≤ 50% Predicted, or the Onset of Respiratory Insufficiency, or Death During the 48 Weeks of Double-blind, Placebo-controlled Treatment | 48 weeks | This endpoint evaluated the time to occurrence of a decline in SVC (as measured by spirometry) to ≤ 50% predicted, or the onset of respiratory insufficiency (defined as tracheostomy or the use of non-invasive ventilation for ≥ 22 hours per day for ≥10 consecutive days), or death, whichever was first, during the 48-week double-blind, placebo-controlled treatment phase. Note: The median time to a decline in SVC to ≤ 50% predicted, onset of respiratory insufficiency, or death was not estimable for the placebo group or the 375 mg tirasemtiv group. The median time was estimated as 363 and 351 days for the 250 mg and 500 mg tirasemtiv groups, respectively. The data presented for this endpoint are the number and percent of patients who met the endpoint. |
| Secondary | Change From Baseline in the ALSFRS-R Total Score to the End of 48 Weeks of the Double-blind, Placebo-controlled Treatment | 48 weeks | The ALSFRS-R is used to measure the progression and severity of disease; it consists of 12 questions, assessing a patient's capability and independence in functional activities relevant to ALS, categorized in 4 domains: gross motor tasks, fine motor tasks, bulbar functions, and respiratory function. Each question is scored from 0 (indicating incapable or dependent) to 4 (normal). The total score ranges from 0 to 48, with higher scores reflecting more normal function and lower scores reflecting more impaired function. |
| Secondary | Time to the First Use of Mechanical Ventilatory Assistance or Death During All 48 Weeks of Double-blind, Placebo-controlled Treatment | 48 weeks | This endpoint evaluated the time to occurrence of mechanical ventilatory assistance (defined as invasive or non-invasive ventilation for at least 2 hours over a 24-hour period for at least 5 consecutive days) or death, whichever was first, during the 48-week double-blind, placebo-controlled treatment phase. Note: The median time to first use of mechanical ventilatory assistance or death was not estimable for all but the 375 mg tirasemtiv group (with a value of 367 days). As such the number and percent of patients who met the endpoint (ie, had mechanical ventilatory assistance or died) are presented. |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline to Week 24 of the Double-blind, Placebo-controlled Phase in Percent Predicted Slow Vital Capacity (SVC) | 24 weeks | The data presented are for the Full Analysis Set, comprised of patients who received at least 1 dose of study drug during the randomized double-blind, placebo-controlled phase and had at least 1 post-randomization efficacy assessment. | % predicted | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in the ALSFRS-R Respiratory Domain Score at the End of 48 Weeks of Double-blind, Placebo-controlled Treatment | 48 weeks | The data provided are for the Full Analysis Set, comprised of patients who received at least 1 dose of study drug during the randomized double-blind, placebo-controlled phase and had at least 1 post-randomization efficacy assessment. | units on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Slope of Mega-score of Muscle Strength During the 48 Weeks of Double-blind, Placebo-controlled Treatment | 48 weeks | The data provided are for the Full Analysis Set, comprised of patients who received at least 1 dose of study drug during the randomized double-blind, placebo-controlled phase and had at least 1 post-randomization efficacy assessment. | pounds/day | MEAN |
| SECONDARY | Time to the First Occurrence of a Decline From Baseline in Percent Predicted SVC ≥ 20 Percentage Points or the Onset of Respiratory Insufficiency or Death All 48 Weeks of Double-blind, Placebo-controlled Treatment | 48 weeks | The data provided are for the Full Analysis Set, comprised of patients who received at least 1 dose of study drug during the randomized double-blind, placebo-controlled phase and had at least 1 post-randomization efficacy assessment. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Time to the First Occurrence of a Decline in SVC to ≤ 50% Predicted, or the Onset of Respiratory Insufficiency, or Death During the 48 Weeks of Double-blind, Placebo-controlled Treatment | 48 weeks | The data provided are for the Full Analysis Set, comprised of patients who received at least 1 dose of study drug during the randomized double-blind, placebo-controlled phase and had at least 1 post-randomization efficacy assessment. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in the ALSFRS-R Total Score to the End of 48 Weeks of the Double-blind, Placebo-controlled Treatment | 48 weeks | The data provided are for the Full Analysis Set, comprised of patients who received at least 1 dose of study drug during the randomized double-blind, placebo-controlled phase and had at least 1 post-randomization efficacy assessment. | units on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Time to the First Use of Mechanical Ventilatory Assistance or Death During All 48 Weeks of Double-blind, Placebo-controlled Treatment | 48 weeks | The data provided are for the Full Analysis Set, comprised of patients who received at least 1 dose of study drug during the randomized double-blind, placebo-controlled phase and had at least 1 post-randomization efficacy assessment. | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group28
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline to Week 24 of the Double-blind, Placebo-controlled Phase in Percent Predicted Slow Vital Capacity (SVC) | Double-blind, Placebo-controlled: Group 1 - Placebo | -14.354 | Standard Error 1.2270 | % predicted | |
| Change From Baseline to Week 24 of the Double-blind, Placebo-controlled Phase in Percent Predicted Slow Vital Capacity (SVC) | Double-blind, Placebo-controlled: Group 2 - 250 mg Tirasemtiv | -12.648 | Standard Error 1.5165 | % predicted | |
| Change From Baseline to Week 24 of the Double-blind, Placebo-controlled Phase in Percent Predicted Slow Vital Capacity (SVC) | Double-blind, Placebo-controlled: Group 3 - 375 mg Tirasemtiv | -13.742 | Standard Error 1.6076 | % predicted | |
| Change From Baseline to Week 24 of the Double-blind, Placebo-controlled Phase in Percent Predicted Slow Vital Capacity (SVC) | Double-blind, Placebo-controlled: Group 4 - 500 mg Tirasemtiv | -13.927 | Standard Error 1.7213 | % predicted | |
| Change From Baseline in the ALSFRS-R Respiratory Domain Score at the End of 48 Weeks of Double-blind, Placebo-controlled Treatment | Double-blind, Placebo-controlled: Group 1 - Placebo | -2.26 | Standard Error 0.258 | units on a scale | |
| Change From Baseline in the ALSFRS-R Respiratory Domain Score at the End of 48 Weeks of Double-blind, Placebo-controlled Treatment | Double-blind, Placebo-controlled: Group 2 - 250 mg Tirasemtiv | -1.95 | Standard Error 0.320 | units on a scale | |
| Change From Baseline in the ALSFRS-R Respiratory Domain Score at the End of 48 Weeks of Double-blind, Placebo-controlled Treatment | Double-blind, Placebo-controlled: Group 3 - 375 mg Tirasemtiv | -2.41 | Standard Error 0.340 | units on a scale | |
| Change From Baseline in the ALSFRS-R Respiratory Domain Score at the End of 48 Weeks of Double-blind, Placebo-controlled Treatment | Double-blind, Placebo-controlled: Group 4 - 500 mg Tirasemtiv | -2.59 | Standard Error 0.355 | units on a scale | |
| Slope of Mega-score of Muscle Strength During the 48 Weeks of Double-blind, Placebo-controlled Treatment | Double-blind, Placebo-controlled: Group 1 - Placebo | -0.1501 | 95% Confidence Interval -0.1729 to -0.1272 | pounds/day | |
| Slope of Mega-score of Muscle Strength During the 48 Weeks of Double-blind, Placebo-controlled Treatment | Double-blind, Placebo-controlled: Group 2 - 250 mg Tirasemtiv | -0.1512 | 95% Confidence Interval -0.1794 to -0.1230 | pounds/day |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 results by groupParticipant flow72
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Open-label Lead-in Phase | STARTED | Open-label Lead-in: 250 mg Tirasemtiv | 744 | |
| Open-label Lead-in Phase | STARTED | Double-blind, Placebo-controlled: Group 1 - Placebo | 0 | |
| Open-label Lead-in Phase | STARTED | Double-blind, Placebo-controlled: Group 2 - 250 mg Tirasemtiv | 0 | |
| Open-label Lead-in Phase | STARTED | Double-blind, Placebo-controlled: Group 3 - 375 mg Tirasemtiv | 0 | |
| Open-label Lead-in Phase | STARTED | Double-blind, Placebo-controlled: Group 4 - 500 mg Tirasemtiv | 0 | |
| Open-label Lead-in Phase | STARTED | Double-blind, Withdrawal: Placebo | 0 | |
| Open-label Lead-in Phase | STARTED | Double-blind, Withdrawal: Tirasemtiv to Placebo | 0 | |
| Open-label Lead-in Phase | STARTED | Double-blind, Withdrawal: Tirasemtiv | 0 | |
| Open-label Lead-in Phase | COMPLETED | Open-label Lead-in: 250 mg Tirasemtiv | 565 | |
| Open-label Lead-in Phase | COMPLETED | Double-blind, Placebo-controlled: Group 1 - Placebo | 0 |
1 to 10 of 72Source ClinicalTrials.gov
See all 72 participant flowAdverse events1,280
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 | Abdominal distension | Gastrointestinal disorders | Open-label Lead-in: 250 mg Tirasemtiv | 3 | 744 | |
| other | Abdominal distension | Gastrointestinal disorders | Double-blind, Placebo-controlled: Group 1 - Placebo | 2 | 188 | |
| other | Abdominal distension | Gastrointestinal disorders | Double-blind, Placebo-controlled: Group 2 - 250 mg Tirasemtiv | 4 | 126 | |
| other | Abdominal distension | Gastrointestinal disorders | Double-blind, Placebo-controlled: Group 3 - 375 mg Tirasemtiv | 5 | 126 | |
| other | Abdominal distension | Gastrointestinal disorders | Double-blind, Placebo-controlled: Group 4 - 500 mg Tirasemtiv | 3 | 125 | |
| other | Abdominal distension | Gastrointestinal disorders | Double-blind, Withdrawal: Placebo | 2 | 132 | |
| other | Abdominal distension | Gastrointestinal disorders | Double-blind, Withdrawal: Tirasemtiv to Placebo | 1 | 103 | |
| other | Abdominal distension | Gastrointestinal disorders | Double-blind, Withdrawal: Tirasemtiv | 5 | 101 | |
| other | Constipation | Gastrointestinal disorders | Open-label Lead-in: 250 mg Tirasemtiv | 21 | 744 | |
| other | Constipation | Gastrointestinal disorders | Double-blind, Placebo-controlled: Group 1 - Placebo | 40 | 188 |
1 to 10 of 1,280Source ClinicalTrials.gov
See all 1,280 adverse eventsAdverse event totals24
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Open-label Lead-in: 250 mg Tirasemtiv | 1 | 744 |
| Total, all-cause mortality | Double-blind, Placebo-controlled: Group 1 - Placebo | 17 | 188 |
| Total, all-cause mortality | Double-blind, Placebo-controlled: Group 2 - 250 mg Tirasemtiv | 8 | 126 |
| Total, all-cause mortality | Double-blind, Placebo-controlled: Group 3 - 375 mg Tirasemtiv | 10 | 126 |
| Total, all-cause mortality | Double-blind, Placebo-controlled: Group 4 - 500 mg Tirasemtiv | 8 | 125 |
| Total, all-cause mortality | Double-blind, Withdrawal: Placebo | 5 | 132 |
| Total, all-cause mortality | Double-blind, Withdrawal: Tirasemtiv to Placebo | 4 | 103 |
| Total, all-cause mortality | Double-blind, Withdrawal: Tirasemtiv | 5 | 101 |
| Total, other adverse events | Open-label Lead-in: 250 mg Tirasemtiv | 640 | 744 |
| Total, other adverse events | Double-blind, Placebo-controlled: Group 1 - Placebo | 182 | 188 |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 adverse event totalsPapers2
The papers the trial lists on ClinicalTrials.gov.
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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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.
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