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A Study of CK-2127107 in Patients With Spinal Muscular Atrophy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02644668
Recruitment Status : Completed
First Posted : January 1, 2016
Results First Posted : August 31, 2020
Last Update Posted : August 31, 2020
Sponsor:
Collaborator:
Astellas Pharma Global Development, Inc.
Information provided by (Responsible Party):
Cytokinetics

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Triple (Participant, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Spinal Muscular Atrophy
Interventions Drug: Placebo
Drug: Reldesemtiv 150 mg
Drug: Reldesemtiv 450 mg
Enrollment 70
Recruitment Details Patients with SMA were enrolled at 18 sites in Canada and the United States. The first patient was enrolled on 14 January 2016 and the last patient completed on 31 May 2018.
Pre-assignment Details Eligible patients were male or female, ≥12 y of age and had a genetically confirmed diagnosis of SMA. Ambulatory patients were able to walk ≥50 m in 6 min; and non-ambulatory patients were able to tolerate upright sitting with support for 3 h. Patients had an FVC >20% predicted, an HFMS-E score ≥10 and ≤54, and elbow and knee flexion ≤90 degrees.
Arm/Group Title Placebo Reldesemtiv 150 mg Twice Daily Reldesemtiv 450 mg Twice Daily
Hide Arm/Group Description

Patients randomized to this treatment arm received a placebo suspension twice daily for 8 weeks.

Placebo: Granules for oral suspension (placebo)

Patient randomized to this treatment arm received reldesemtiv suspension at a dose of 150 mg, twice daily for 8 weeks.

Reldesemtiv 150 mg: Granules for oral suspension, 18.7% reldesemtiv

Patients randomized to this treatment arm received reldesemtiv suspension at a dose of 450 mg, twice daily for 8 weeks.

Reldesemtiv 450 mg: Granules for oral suspension, 56.0% reldesemtiv
Period Title: Overall Study
Started 26 24 20
Completed 24 24 17
Not Completed 2 0 3
Reason Not Completed
Adverse Event             2             0             1
Protocol Violation             0             0             1
Withdrawal by Subject             0             0             1
Arm/Group Title Placebo Reldesemtiv 150 mg Twice Daily Reldesemtiv 450 mg Twice Daily Total
Hide Arm/Group Description Patients randomized to this treatment arm received a placebo suspension twice daily for 8 weeks. Patient randomized to this treatment arm received reldesemtiv suspension at a dose of 150 mg, twice daily for 8 weeks. Patients randomized to this treatment arm received reldesemtiv suspension at a dose of 450 mg, twice daily for 8 weeks. Total of all reporting groups
Overall Number of Baseline Participants 26 24 20 70
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 26 participants 24 participants 20 participants 70 participants
28.5  (16.03) 27.8  (11.96) 32.6  (17.92) 29.4  (15.27)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 26 participants 24 participants 20 participants 70 participants
Female
11
  42.3%
10
  41.7%
8
  40.0%
29
  41.4%
Male
15
  57.7%
14
  58.3%
12
  60.0%
41
  58.6%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 26 participants 24 participants 20 participants 70 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
2
   7.7%
1
   4.2%
1
   5.0%
4
   5.7%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
1
   3.8%
0
   0.0%
1
   5.0%
2
   2.9%
White
22
  84.6%
23
  95.8%
18
  90.0%
63
  90.0%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
1
   3.8%
0
   0.0%
0
   0.0%
1
   1.4%
Time since SMA symptom onset  
Mean (Standard Deviation)
Unit of measure:  Months
Number Analyzed 26 participants 24 participants 20 participants 70 participants
302.5  (170.11) 246.8  (105.93) 299.8  (174.32) 282.6  (152.56)
Time since genetically confirmed diagnosis  
Mean (Standard Deviation)
Unit of measure:  Months
Number Analyzed 26 participants 24 participants 20 participants 70 participants
139.2  (75.09) 138.8  (81.45) 101.4  (82.27) 128.2  (80.06)
1.Primary Outcome
Title Change From Baseline to Week 8 in Forced Vital Capacity (FVC)
Hide Description FVC was measured using a calibrated spirometer (in units of liters). Patients were instructed to take as deep an inspiration as possible followed by a maximum exhalation (blowing out all the air in their lungs).
Time Frame baseline and 8 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population includes patients who received at least 1 dose of reldesemtiv and had results from the relevant outcome measure at Week 8.
Arm/Group Title Placebo Reldesemtiv 150 mg Twice Daily Reldesemtiv 450 mg Twice Daily
Hide Arm/Group Description:
Patients randomized to this treatment arm received a placebo suspension twice daily for 8 weeks.
Patient randomized to this treatment arm received reldesemtiv suspension at a dose of 150 mg, twice daily for 8 weeks.
Patients randomized to this treatment arm received reldesemtiv suspension at a dose of 450 mg, twice daily for 8 weeks.
Overall Number of Participants Analyzed 25 24 18
Least Squares Mean (Standard Error)
Unit of Measure: liters
-0.02  (0.042) -0.03  (0.043) -0.07  (0.050)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Reldesemtiv 150 mg Twice Daily
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.9086
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Least squares mean difference
Estimated Value -0.01
Confidence Interval (2-Sided) 95%
-0.13 to 0.11
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.060
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, Reldesemtiv 450 mg Twice Daily
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.4361
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Least squares mean difference
Estimated Value -0.05
Confidence Interval (2-Sided) 95%
-0.18 to 0.08
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.065
Estimation Comments [Not Specified]
2.Primary Outcome
Title Change From Baseline to Week 8 in Maximum Inspiratory Pressure (MIP)
Hide Description MIP was measured (in units of cm H20) using a calibrated spirometer with an inspiratory pressure valve attached. For the test, patients were asked to inhale as forcefully as possible, to their maximum pressure.
Time Frame baseline and 8 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population includes patients who received at least 1 dose of reldesemtiv and had results from the relevant outcome measure at Week 8.
Arm/Group Title Placebo Reldesemtiv 150 mg Twice Daily Reldesemtiv 450 mg Twice Daily
Hide Arm/Group Description:
Patients randomized to this treatment arm received a placebo suspension twice daily for 8 weeks.
Patient randomized to this treatment arm received reldesemtiv suspension at a dose of 150 mg, twice daily for 8 weeks.
Patients randomized to this treatment arm received reldesemtiv suspension at a dose of 450 mg, twice daily for 8 weeks.
Overall Number of Participants Analyzed 25 24 18
Least Squares Mean (Standard Error)
Unit of Measure: cm H2O
-2.62  (3.317) -5.56  (3.401) -1.63  (3.901)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Reldesemtiv 150 mg Twice Daily
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.5382
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Least squares mean difference
Estimated Value -2.94
Confidence Interval (2-Sided) 95%
-12.43 to 6.55
Parameter Dispersion
Type: Standard Error of the Mean
Value: 4.749
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, Reldesemtiv 450 mg Twice Daily
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.8464
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Least squares mean difference
Estimated Value 0.99
Confidence Interval (2-Sided) 95%
-9.19 to 11.18
Parameter Dispersion
Type: Standard Error of the Mean
Value: 5.099
Estimation Comments [Not Specified]
3.Primary Outcome
Title Change From Baseline to Week 8 in Maximum Expiratory Pressure (MEP)
Hide Description MEP was measured (in units of cm H20) using a calibrated spirometer with an exspiratory pressure valve attached. For the test, patients were asked to maximally inhale then perform a forced exhalation with as forcefully as possible.
Time Frame baseline and 8 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population includes patients who received at least 1 dose of reldesemtiv and had results from the relevant outcome measure at Week 8.
Arm/Group Title Placebo Reldesemtiv 150 mg Twice Daily Reldesemtiv 450 mg Twice Daily
Hide Arm/Group Description:
Patients randomized to this treatment arm received a placebo suspension twice daily for 8 weeks.
Patient randomized to this treatment arm received reldesemtiv suspension at a dose of 150 mg, twice daily for 8 weeks.
Patients randomized to this treatment arm received reldesemtiv suspension at a dose of 450 mg, twice daily for 8 weeks.
Overall Number of Participants Analyzed 25 24 18
Least Squares Mean (Standard Error)
Unit of Measure: cm H2O
-4.22  (3.843) 7.47  (3.941) 8.93  (4.517)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Reldesemtiv 150 mg Twice Daily
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0378
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Least squares mean difference
Estimated Value 11.69
Confidence Interval (2-Sided) 95%
0.68 to 22.70
Parameter Dispersion
Type: Standard Error of the Mean
Value: 5.506
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, Reldesemtiv 450 mg Twice Daily
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0298
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Least squares mean difference
Estimated Value 13.15
Confidence Interval (2-Sided) 95%
1.33 to 24.97
Parameter Dispersion
Type: Standard Error of the Mean
Value: 5.913
Estimation Comments [Not Specified]
4.Primary Outcome
Title Muscle Strength Mega-Score at Week 8
Hide Description

Muscle strength of 3 muscle groups (elbow flexion, knee extension, and shoulder abduction) were measured bilaterally using a hand-held dynamometer. Muscle strength was measured twice for each body location; if the variability between the 2 measures was > 15%, a third measure was obtained.

The maximum muscle strength of the 2 measurements was identified and transformed as a percent change from baseline using the equation: ([postbaseline value - baseline value] / baseline value) × 100.

The mega-score was a composite score that averaged strength across the 3 muscle groups. It was calculated as the mean of the non-missing transformed muscle strength scores among the 3 muscle groups each measure bilaterally (totaling 6 body locations).
Time Frame baseline and 8 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population includes patients who received at least 1 dose of reldesemtiv and had results from the relevant outcome measure at Week 8.
Arm/Group Title Placebo Reldesemtiv 150 mg Twice Daily Reldesemtiv 450 mg Twice Daily
Hide Arm/Group Description:
Patients randomized to this treatment arm received a placebo suspension twice daily for 8 weeks.
Patient randomized to this treatment arm received reldesemtiv suspension at a dose of 150 mg, twice daily for 8 weeks.
Patients randomized to this treatment arm received reldesemtiv suspension at a dose of 450 mg, twice daily for 8 weeks.
Overall Number of Participants Analyzed 25 24 18
Least Squares Mean (Standard Error)
Unit of Measure: percent change
14.34  (5.298) 9.76  (5.393) -0.88  (6.247)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Reldesemtiv 150 mg Twice Daily
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.5461
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Least squares mean difference
Estimated Value -4.59
Confidence Interval (2-Sided) 95%
-19.69 to 10.52
Parameter Dispersion
Type: Standard Error of the Mean
Value: 7.560
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, Reldesemtiv 450 mg Twice Daily
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0672
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Least squares mean difference
Estimated Value -15.23
Confidence Interval (2-Sided) 95%
-31.56 to 1.11
Parameter Dispersion
Type: Standard Error of the Mean
Value: 8.175
Estimation Comments [Not Specified]
5.Primary Outcome
Title Change From Baseline to Week 8 in the Hammersmith Functional Motor Scale-Expanded (HFMS-E)
Hide Description The HFMS-E evaluated the level of independent mobility and motor skills through assessment of 33 test-items, each scored from 0 (worse) to 2 (better). The total score was calculated as the sum of the scores among the 33 test items, and has a range from 0 to 66.
Time Frame baseline and 8 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population includes patients who received at least 1 dose of reldesemtiv and had results from the relevant outcome measure at Week 8.
Arm/Group Title Placebo Reldesemtiv 150 mg Twice Daily Reldesemtiv 450 mg Twice Daily
Hide Arm/Group Description:
Patients randomized to this treatment arm received a placebo suspension twice daily for 8 weeks.
Patient randomized to this treatment arm received reldesemtiv suspension at a dose of 150 mg, twice daily for 8 weeks.
Patients randomized to this treatment arm received reldesemtiv suspension at a dose of 450 mg, twice daily for 8 weeks.
Overall Number of Participants Analyzed 25 22 18
Least Squares Mean (Standard Error)
Unit of Measure: score on a scale
1.40  (0.637) 1.02  (0.675) 0.39  (0.748)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Reldesemtiv 150 mg Twice Daily
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.6849
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Least squares mean difference
Estimated Value -0.38
Confidence Interval (2-Sided) 95%
-2.23 to 1.48
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.928
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, Reldesemtiv 450 mg Twice Daily
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3091
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Least squares mean difference
Estimated Value -1.00
Confidence Interval (2-Sided) 95%
-2.96 to 0.95
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.980
Estimation Comments [Not Specified]
6.Primary Outcome
Title Change From Baseline to Week 8 in Revised Upper Limb Module (RULM)
Hide Description The RULM assessed motor function in the upper limbs (specifically shoulder, elbow, wrist, and hand function) that related to activities of everyday life. The RULM consisted of 20 items, 1 of which was scored on a 7-point scale (from 0 to 6), 18 were scored on a 3-point scale (from 0 to 2), and 1 was scored on a 2-point scale (0 or 1). The total score was the sum of each response and could range from a minimum of 0 to a maximum of 43 points. Higher scores reflected better motor function.
Time Frame baseline and 8 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population includes patients who received at least 1 dose of reldesemtiv and had results from the relevant outcome measure at Week 8.
Arm/Group Title Placebo Reldesemtiv 150 mg Twice Daily Reldesemtiv 450 mg Twice Daily
Hide Arm/Group Description:
Patients randomized to this treatment arm received a placebo suspension twice daily for 8 weeks.
Patient randomized to this treatment arm received reldesemtiv suspension at a dose of 150 mg, twice daily for 8 weeks.
Patients randomized to this treatment arm received reldesemtiv suspension at a dose of 450 mg, twice daily for 8 weeks.
Overall Number of Participants Analyzed 25 24 18
Least Squares Mean (Standard Error)
Unit of Measure: score on a scale
0.54  (0.397) 1.15  (0.406) 0.43  (0.468)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Reldesemtiv 150 mg Twice Daily
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.2878
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Least squares mean difference
Estimated Value 0.61
Confidence Interval (2-Sided) 95%
-0.53 to 1.75
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.571
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, Reldesemtiv 450 mg Twice Daily
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.8512
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Least squares mean difference
Estimated Value -0.12
Confidence Interval (2-Sided) 95%
-1.34 to 1.11
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.616
Estimation Comments [Not Specified]
7.Primary Outcome
Title Change From Baseline to Week 8 in the TUG Test
Hide Description The TUG test measured the time (in seconds) it took for a patient to rise from a chair, walk 3 meters, turn around, walk back to the chair and sit down.
Time Frame baseline and 8 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
This test was evaluated in ambulatory patients only.
Arm/Group Title Placebo Reldesemtiv 150 mg Twice Daily Reldesemtiv 450 mg Twice Daily
Hide Arm/Group Description:
Patients randomized to this treatment arm received a placebo suspension twice daily for 8 weeks.
Patient randomized to this treatment arm received reldesemtiv suspension at a dose of 150 mg, twice daily for 8 weeks.
Patients randomized to this treatment arm received reldesemtiv suspension at a dose of 450 mg, twice daily for 8 weeks.
Overall Number of Participants Analyzed 8 10 4
Least Squares Mean (Standard Error)
Unit of Measure: seconds
0.24  (1.837) -0.54  (1.708) -2.87  (2.675)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Reldesemtiv 150 mg Twice Daily
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.7612
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Least squares mean difference
Estimated Value -0.78
Confidence Interval (2-Sided) 95%
-6.08 to 4.52
Parameter Dispersion
Type: Standard Error of the Mean
Value: 2.528
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, Reldesemtiv 450 mg Twice Daily
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3502
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Least squares mean difference
Estimated Value -3.10
Confidence Interval (2-Sided) 95%
-9.91 to 3.70
Parameter Dispersion
Type: Standard Error of the Mean
Value: 3.231
Estimation Comments [Not Specified]
8.Primary Outcome
Title Change From Baseline to Week 8 in the 6MWT
Hide Description The 6MWT measured the distance (in meters) a patient walked in 6 minutes.
Time Frame baseline and 8 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
This test was evaluated in ambulatory patients only.
Arm/Group Title Placebo Reldesemtiv 150 mg Twice Daily Reldesemtiv 450 mg Twice Daily
Hide Arm/Group Description:
Patients randomized to this treatment arm received a placebo suspension twice daily for 8 weeks.
Patient randomized to this treatment arm received reldesemtiv suspension at a dose of 150 mg, twice daily for 8 weeks.
Patients randomized to this treatment arm received reldesemtiv suspension at a dose of 450 mg, twice daily for 8 weeks.
Overall Number of Participants Analyzed 9 12 6
Least Squares Mean (Standard Error)
Unit of Measure: meters
1.39  (7.692) 9.11  (6.826) 26.28  (9.641)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Reldesemtiv 150 mg Twice Daily
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.4684
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Least squares mean difference
Estimated Value 7.72
Confidence Interval (2-Sided) 95%
-13.86 to 29.30
Parameter Dispersion
Type: Standard Error of the Mean
Value: 10.484
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, Reldesemtiv 450 mg Twice Daily
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0584
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Least squares mean difference
Estimated Value 24.89
Confidence Interval (2-Sided) 95%
-0.95 to 50.74
Parameter Dispersion
Type: Standard Error of the Mean
Value: 12.550
Estimation Comments [Not Specified]
9.Primary Outcome
Title Patient Global Assessment at the End of Week 8
Hide Description Patients assessed whether they felt the same, better, or worse than prior to dosing on Day 1.
Time Frame 8 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population includes patients who received at least 1 dose of reldesemtiv and had results from the relevant outcome measure at Week 8.
Arm/Group Title Placebo Reldesemtiv 150 mg Twice Daily Reldesemtiv 450 mg Twice Daily
Hide Arm/Group Description:
Patients randomized to this treatment arm received a placebo suspension twice daily for 8 weeks.
Patient randomized to this treatment arm received reldesemtiv suspension at a dose of 150 mg, twice daily for 8 weeks.
Patients randomized to this treatment arm received reldesemtiv suspension at a dose of 450 mg, twice daily for 8 weeks.
Overall Number of Participants Analyzed 25 24 18
Measure Type: Count of Participants
Unit of Measure: Participants
Better than pre-dose
12
  48.0%
7
  29.2%
7
  38.9%
Same as pre-dose
13
  52.0%
15
  62.5%
11
  61.1%
Worse than pre-dose
0
   0.0%
2
   8.3%
0
   0.0%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Reldesemtiv 150 mg Twice Daily
Comments Odds ratio of active vs placebo comparison of the patients' global assessment of better than pre-dose vs same or worse than pre-dose
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1686
Comments [Not Specified]
Method Regression, Logistic
Comments [Not Specified]
Method of Estimation Estimation Parameter Odds Ratio (OR)
Estimated Value 0.43
Confidence Interval (2-Sided) 95%
0.13 to 1.43
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, Reldesemtiv 450 mg Twice Daily
Comments Odds ratio of active vs placebo comparison of the patients' global assessment of better than pre-dose vs same or worse than pre-dose
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.5259
Comments [Not Specified]
Method Regression, Logistic
Comments [Not Specified]
Method of Estimation Estimation Parameter Odds Ratio (OR)
Estimated Value 0.67
Confidence Interval (2-Sided) 95%
0.20 to 2.30
Estimation Comments [Not Specified]
10.Primary Outcome
Title Investigator Global Assessment at the End of Week 8
Hide Description The Investigator assessed whether patient appeared the same, better, or worse than prior to dosing on Day 1.
Time Frame 8 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population includes patients who received at least 1 dose of reldesemtiv and had results from the relevant outcome measure at Week 8.
Arm/Group Title Placebo Reldesemtiv 150 mg Twice Daily Reldesemtiv 450 mg Twice Daily
Hide Arm/Group Description:
Patients randomized to this treatment arm received a placebo suspension twice daily for 8 weeks.
Patient randomized to this treatment arm received reldesemtiv suspension at a dose of 150 mg, twice daily for 8 weeks.
Patients randomized to this treatment arm received reldesemtiv suspension at a dose of 450 mg, twice daily for 8 weeks.
Overall Number of Participants Analyzed 24 24 17
Measure Type: Count of Participants
Unit of Measure: Participants
Better than pre-dose
6
  25.0%
7
  29.2%
6
  35.3%
Same as pre-dose
18
  75.0%
17
  70.8%
11
  64.7%
Worse than pre-dose
0
   0.0%
0
   0.0%
0
   0.0%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Reldesemtiv 150 mg Twice Daily
Comments Odds ratio of active vs placebo comparison of the patients' global assessment of better than pre-dose vs same or worse than pre-dose
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.7655
Comments [Not Specified]
Method Regression, Logistic
Comments [Not Specified]
Method of Estimation Estimation Parameter Odds Ratio (OR)
Estimated Value 1.22
Confidence Interval (2-Sided) 95%
0.34 to 4.40
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, Reldesemtiv 450 mg Twice Daily
Comments Odds ratio of active vs placebo comparison of the patients' global assessment of better than pre-dose vs same or worse than pre-dose
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.4920
Comments [Not Specified]
Method Regression, Logistic
Comments [Not Specified]
Method of Estimation Estimation Parameter Odds Ratio (OR)
Estimated Value 1.61
Confidence Interval (2-Sided) 95%
0.41 to 6.25
Estimation Comments [Not Specified]
11.Secondary Outcome
Title Reldesemtiv Maximum Observed Plasma Concentration (Cmax)
Hide Description Determined by evaluation of reldesemtiv plasma concentrations from blood samples collected prior to dosing and at 1, 3, and 6 hours following dosing
Time Frame End of Week 8
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population includes all patients who had an evaluable reldesemtiv Cmax value at Week 8.
Arm/Group Title Reldesemtiv 150 mg Twice Daily Reldesemtiv 450 mg Twice Daily
Hide Arm/Group Description:
Patient randomized to this treatment arm received reldesemtiv suspension at a dose of 150 mg, twice daily for 8 weeks.
Patients randomized to this treatment arm received reldesemtiv suspension at a dose of 450 mg, twice daily for 8 weeks.
Overall Number of Participants Analyzed 23 15
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: microgram/milliliter
1.40
(43.18%)
3.34
(48.77%)
12.Secondary Outcome
Title Reldesemtiv Area Under the Plasma Concentration-time Curve From 0 to 12 Hours (AUC0-12)
Hide Description Determined by evaluation of reldesemtiv plasma concentrations from blood samples collected prior to dosing and at 1, 3, and 6 hours following dosing
Time Frame End of Week 8
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population includes patients who had an evaluable reldesemtiv AUC0-12 value at Week 8.
Arm/Group Title Reldesemtiv 150 mg Twice Daily Reldesemtiv 450 mg Twice Daily
Hide Arm/Group Description:
Patient randomized to this treatment arm received reldesemtiv suspension at a dose of 150 mg, twice daily for 8 weeks.
Patients randomized to this treatment arm received reldesemtiv suspension at a dose of 450 mg, twice daily for 8 weeks.
Overall Number of Participants Analyzed 23 15
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: hour x microgram/milliliter
9.0
(49.38%)
25.0
(52.19%)
Time Frame Adverse events (AEs) were collected over approximately 12 weeks: from administration of the first dose of study drug through the treatment period [8 weeks] and through the follow-up period [4 weeks after the last dose of study drug]).
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Placebo Reldesemtiv 150 mg Twice Daily Reldesemtiv 450 mg Twice Daily
Hide Arm/Group Description Patients randomized to this treatment arm received a placebo suspension twice daily for 8 weeks. Patient randomized to this treatment arm received reldesemtiv suspension at a dose of 150 mg, twice daily for 8 weeks. Patients randomized to this treatment arm received reldesemtiv suspension at a dose of 450 mg, twice daily for 8 weeks.
All-Cause Mortality
Placebo Reldesemtiv 150 mg Twice Daily Reldesemtiv 450 mg Twice Daily
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/26 (0.00%)      0/24 (0.00%)      0/20 (0.00%)    
Hide Serious Adverse Events
Placebo Reldesemtiv 150 mg Twice Daily Reldesemtiv 450 mg Twice Daily
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/26 (0.00%)      2/24 (8.33%)      2/20 (10.00%)    
Cardiac disorders       
Myocarditis † 1  0/26 (0.00%)  0 1/24 (4.17%)  1 0/20 (0.00%)  0
General disorders       
Facial pain † 1  0/26 (0.00%)  0 1/24 (4.17%)  1 0/20 (0.00%)  0
Infections and infestations       
Gastroenteritis Escherichia coli † 1  0/26 (0.00%)  0 1/24 (4.17%)  1 0/20 (0.00%)  0
Gastroenteritis salmonella † 1  0/26 (0.00%)  0 1/24 (4.17%)  1 0/20 (0.00%)  0
Injury, poisoning and procedural complications       
Traumatic fracture † 1  0/26 (0.00%)  0 0/24 (0.00%)  0 1/20 (5.00%)  1
Investigations       
Blood creatine phosphokinase increased † 1  0/26 (0.00%)  0 0/24 (0.00%)  0 1/20 (5.00%)  1
Aspartate aminotransferase increased † 1  0/26 (0.00%)  0 0/24 (0.00%)  0 1/20 (5.00%)  1
Nervous system disorders       
Hypoaesthesia † 1  0/26 (0.00%)  0 1/24 (4.17%)  1 0/20 (0.00%)  0
1
Term from vocabulary, MedDRA (18.0)
†
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Placebo Reldesemtiv 150 mg Twice Daily Reldesemtiv 450 mg Twice Daily
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   24/26 (92.31%)      20/24 (83.33%)      17/20 (85.00%)    
Gastrointestinal disorders       
Constipation † 1  0/26 (0.00%)  0 3/24 (12.50%)  3 2/20 (10.00%)  2
Nausea † 1  5/26 (19.23%)  5 3/24 (12.50%)  4 2/20 (10.00%)  2
Diarrhoea † 1  2/26 (7.69%)  2 2/24 (8.33%)  2 1/20 (5.00%)  2
Dyspepsia † 1  0/26 (0.00%)  0 2/24 (8.33%)  2 1/20 (5.00%)  1
Abdominal pain upper † 1  1/26 (3.85%)  1 2/24 (8.33%)  2 0/20 (0.00%)  0
Dry mouth † 1  2/26 (7.69%)  2 0/24 (0.00%)  0 0/20 (0.00%)  0
Abdominal pain lower † 1  0/26 (0.00%)  0 0/24 (0.00%)  0 1/20 (5.00%)  1
Vomiting † 1  0/26 (0.00%)  0 0/24 (0.00%)  0 1/20 (5.00%)  2
General disorders       
Fatigue † 1  4/26 (15.38%)  5 2/24 (8.33%)  2 2/20 (10.00%)  2
Pyrexia † 1  2/26 (7.69%)  2 0/24 (0.00%)  0 0/20 (0.00%)  0
Feeling hot † 1  0/26 (0.00%)  0 0/24 (0.00%)  0 1/20 (5.00%)  1
Influenza like illness † 1  0/26 (0.00%)  0 0/24 (0.00%)  0 1/20 (5.00%)  1
Pain † 1  1/26 (3.85%)  1 0/24 (0.00%)  0 1/20 (5.00%)  1
Infections and infestations       
Nasopharyngitis † 1  3/26 (11.54%)  3 3/24 (12.50%)  4 0/20 (0.00%)  0
Upper respiratory tract infection † 1  4/26 (15.38%)  4 0/24 (0.00%)  0 2/20 (10.00%)  2
Respiratory tract infection † 1  0/26 (0.00%)  0 1/24 (4.17%)  1 1/20 (5.00%)  1
Bronchitis † 1  0/26 (0.00%)  0 0/24 (0.00%)  0 1/20 (5.00%)  1
Viral upper respiratory tract infection † 1  0/26 (0.00%)  0 0/24 (0.00%)  0 1/20 (5.00%)  1
Injury, poisoning and procedural complications       
Fall † 1  3/26 (11.54%)  5 1/24 (4.17%)  2 2/20 (10.00%)  3
Contusion † 1  0/26 (0.00%)  0 2/24 (8.33%)  2 0/20 (0.00%)  0
Skin abrasion † 1  0/26 (0.00%)  0 0/24 (0.00%)  0 2/20 (10.00%)  3
Ligament sprain † 1  1/26 (3.85%)  2 0/24 (0.00%)  0 1/20 (5.00%)  2
Investigations       
Blood creatine phosphokinase increased † 1  0/26 (0.00%)  0 0/24 (0.00%)  0 1/20 (5.00%)  2
Blood bilirubin increased † 1  0/26 (0.00%)  0 0/24 (0.00%)  0 1/20 (5.00%)  1
Blood thyroid stimulating hormone increased † 1  0/26 (0.00%)  0 0/24 (0.00%)  0 1/20 (5.00%)  1
Cystatin C increased † 1  0/26 (0.00%)  0 0/24 (0.00%)  0 1/20 (5.00%)  1
Electrocardiogram abnormal † 1  0/26 (0.00%)  0 0/24 (0.00%)  0 1/20 (5.00%)  1
Metabolism and nutrition disorders       
Increased appetite † 1  2/26 (7.69%)  2 0/24 (0.00%)  0 1/20 (5.00%)  1
Decreased appetite † 1  1/26 (3.85%)  1 1/24 (4.17%)  1 1/20 (5.00%)  1
Musculoskeletal and connective tissue disorders       
Arthralgia † 1  2/26 (7.69%)  2 1/24 (4.17%)  1 0/20 (0.00%)  0
Back pain † 1  2/26 (7.69%)  3 0/24 (0.00%)  0 0/20 (0.00%)  0
Coccydynia † 1  2/26 (7.69%)  2 0/24 (0.00%)  0 0/20 (0.00%)  0
Muscle spasms † 1  0/26 (0.00%)  0 0/24 (0.00%)  0 1/20 (5.00%)  1
Nervous system disorders       
Headache † 1  5/26 (19.23%)  5 6/24 (25.00%)  6 5/20 (25.00%)  7
Psychiatric disorders       
Bruxism † 1  0/26 (0.00%)  0 0/24 (0.00%)  0 1/20 (5.00%)  1
Renal and urinary disorders       
Proteinuria † 1  0/26 (0.00%)  0 0/24 (0.00%)  0 1/20 (5.00%)  1
Respiratory, thoracic and mediastinal disorders       
Respiratory tract congestion † 1  0/26 (0.00%)  0 2/24 (8.33%)  2 0/20 (0.00%)  0
Nasal congestion † 1  2/26 (7.69%)  2 1/24 (4.17%)  1 0/20 (0.00%)  0
Skin and subcutaneous tissue disorders       
Dermatitis contact † 1  0/26 (0.00%)  0 0/24 (0.00%)  0 1/20 (5.00%)  1
1
Term from vocabulary, MedDRA (18.0)
†
Indicates events were collected by systematic assessment
[Not Specified]
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: MD Cytokinetics
Organization: Cytokinetics, Inc.
Phone: 650-624-2929
EMail: medicalaffairs@cytokinetics.com
Layout table for additonal information
Responsible Party: Cytokinetics
ClinicalTrials.gov Identifier: NCT02644668    
Other Study ID Numbers: CY 5021
First Submitted: December 23, 2015
First Posted: January 1, 2016
Results First Submitted: August 5, 2020
Results First Posted: August 31, 2020
Last Update Posted: August 31, 2020