Amyotrophic Lateral Sclerosis
Conditions
Keywords
ALS, Amyotrophic Lateral Sclerosis, Lou Gehrig, Lou Gehrig's, Lou Gehrig's disease, Motor Neuron Disease, Nervous System Diseases, KNS-760704, RTPB
Brief summary
This is an open-label, multi-center study designed to extend the evaluation of the safety, tolerability, and clinical effects of oral administration of KNS-760704 in patients with ALS.
Detailed description
Patients who complete the Part 2 Week 28 visit in study KNS-760704-CL201 and patients with ALS who were actively receiving RTPB \[(6R)-4,5,6,7-tetrahydro-N6-propyl-2,6-benzothiazolediamine dihydro-chloride monohydrate\] under Research IND #60,948 were eligible to participate in this study. Eligible patients received 1 tablet of KNS-760704 (150 mg) every 12 hours (Q12H) (300 mg total daily dose) for up to 180 weeks.
Interventions
DRUGKNS-760704
150 mg Q12H KNS-760704 given orally (300 mg total daily dose)
Sponsors
Knopp Biosciences
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
21 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
1. Patient has provided signed informed consent for this trial before the commencement of any study-related procedure 2. Patient is actively participating in Knopp Protocol KNS-760704-CL201 and has completed the Part 2 Week 28 visit in that study or patient is actively receiving RTPB under Research IND #60,948
Exclusion criteria
1. Patient did not participate in Knopp Protocol KNS-760704-CL201 or patient did not receive RTPB under Research IND #60,948. 2. Patient discontinued from KNS-760704-CL201 for any reason other than enrollment into this study (applies to patients enrolled in KNS-760704-CL201 only) 3. Patient was not taking RTPB under Research IND #60,948 prior to April 30, 2009 (applies to patients enrolled under Research IND #60,948 only)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Potentially Clinically Significant Hematology Results | 180 weeks | Number of Participants with Potentially Clinically Significant Hematology Results by Treatment Group. Percentages based on number of patients with at least one non-missing post-baseline value in each treatment group. Patients are only counted once per criterion per laboratory test. |
| Number of Participants With Potentially Clinically Significant Liver Enzyme Abnormalities | 180 weeks | Number of participants with potentially clinically significant liver enzyme abnormalities for the safety population are presented. Percentages are based on the number of patients with at least one non-missing, post-baseline value for each parameter. |
| Number of Participants With Potentially Clinically ECG Abnormalities | 180 weeks | Number of participants with potentially clinically significant ECG abnormalities for the safety population are presented. Percentages are based on the number of patients in the safety population who had at least one non-missing, post-baseline value. |
| Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities | 180 weeks | Number of participants with potentially clinically significant vital sign abnormalities for the safety population are presented. Percentages are based on the number of patients with at least one non-missing, post-baseline value for each parameter. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Upright Vital Capacity From Baseline to Week 24 | 24 weeks | Change in Percent Predicted Upright Vital Capacity from Baseline to Week 24. A negative change indicates clinical worsening. |
| Change in Upright Vital Capacity From Baseline to Week 48 | 48 weeks | Change in Percent Predicted Upright Vital Capacity from Baseline to Week 48. A negative change indicates clinical worsening. |
| Change in McGill Single-Item Scale (SIS) From Baseline to Week 12 | 12 weeks | The McGill SIS consists of a single question designed to assess the improvement of or the rate of deterioration of the subject's quality-of-life. Subjects rated their quality of life over the prior 2 days on a scale of 0 (very bad) to 10 (excellent). Decreases from Baseline indicate deterioration of a subject's quality of life. |
| Change in Upright Vital Capacity From Baseline to Week 12 | 12 weeks | Change in Percent Predicted Upright Vital Capacity from Baseline to Week 12. A negative change indicates clinical worsening. |
| Change in McGill Single-Item Scale (SIS) From Baseline to Week 48 | 48 weeks | The McGill SIS consists of a single question designed to assess the improvement of or the rate of deterioration of the subject's quality-of-life. Subjects rated their quality of life over the prior 2 days on a scale of 0 (very bad) to 10 (excellent). Decreases from Baseline indicate deterioration of a subject's quality of life. |
| Number of Subjects With Feeding Tube Placed During the Study. | 144 weeks | Number of participants who had a feeding tube placed during the study. |
| Change in McGill Single-Item Scale (SIS) From Baseline to Week 24 | 24 weeks | The McGill SIS consists of a single question designed to assess the improvement of or the rate of deterioration of the subject's quality-of-life. Subjects rated their quality of life over the prior 2 days on a scale of 0 (very bad) to 10 (excellent). Decreases from Baseline indicate deterioration of a subject's quality of life. |
| Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 12 | 12 weeks | The ALSFRS-R (ALS functional rating scale with respiratory component) is a validated scale which measures 4 functional domains, comprising respiratory function, bulbar function, gross motor skills, and fine motor skills. There are a total of 12 questions, each scored from 0 to 4 for a total possible score of 48. Therefore, the score ranges from 0 to 48 with higher scores representing better function. |
| Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 24 | 24 weeks | The ALSFRS-R (ALS functional rating scale with respiratory component) is a validated scale which measures 4 functional domains, comprising respiratory function, bulbar function, gross motor skills, and fine motor skills. There are a total of 12 questions, each scored from 0 to 4 for a total possible score of 48. Therefore, the score ranges from 0 to 48 with higher scores representing better function. |
| Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 48 | 48 weeks | The ALSFRS-R (ALS functional rating scale with respiratory component) is a validated scale which measures 4 functional domains, comprising respiratory function, bulbar function, gross motor skills, and fine motor skills. There are a total of 12 questions, each scored from 0 to 4 for a total possible score of 48. Therefore, the score ranges from 0 to 48 with higher scores representing better function. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| 300 mg Per Day KNS-760704 (dexpramipexole) - 150 mg BID | 74 |
| Total | 74 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 4 |
| Overall Study | Death | 28 |
| Overall Study | Other | 2 |
| Overall Study | W/D Consent (due to ALS progression)) | 19 |
| Overall Study | Withdrawal Consent (due to other) | 2 |
Baseline characteristics
| Characteristic | 300 mg Per Day |
|---|---|
| Age, Continuous | 57.4 years STANDARD_DEVIATION 11.14 |
| Age, Customized <=50 | 24 Participants |
| Age, Customized 51-65 | 30 Participants |
| Age, Customized >65 | 20 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 6 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 68 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 1 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 72 Participants |
| Region of Enrollment United States | 74 participants |
| Sex: Female, Male Female | 22 Participants |
| Sex: Female, Male Male | 52 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 38 / 74 |
| other Total, other adverse events | 74 / 74 |
| serious Total, serious adverse events | 48 / 74 |
Outcome results
Primary
Number of Participants With Potentially Clinically ECG Abnormalities
Number of participants with potentially clinically significant ECG abnormalities for the safety population are presented. Percentages are based on the number of patients in the safety population who had at least one non-missing, post-baseline value.
Time frame: 180 weeks
Population: Number of subjects evaluated is the number of subjects who had a baseline assessment and at least one post-baseline assessment.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically ECG Abnormalities | Axis - LEFT AXIS DEVIATION | 5 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically ECG Abnormalities | Axis - RIGHT AXIS DEVIATION | 1 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically ECG Abnormalities | Conduction - LEFT ANTERIOR HEMIBLOCK | 2 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically ECG Abnormalities | Conduction - RSR' SUGGESTS RIGHT VENTRICULAR CONDUCTION DELAY | 3 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically ECG Abnormalities | Myocardial Ischemia/Infarction - CANNOT RULE OUT POSSIBLE MYOCARDIAL INFARCTION (INFERIOR) | 1 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically ECG Abnormalities | Conduction - SHORT P-R INTERVAL | 3 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically ECG Abnormalities | Morphology - LEFT ATRIAL ENLARGEMENT | 2 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically ECG Abnormalities | Morphology - LEFT VENTRICULAR HYPERTROPHY BY VOLTAGE CRITERIA ONLY | 2 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically ECG Abnormalities | Morphology - LEFT VENTRICULAR HYPERTROPHY WITH REPOLARIZATION ABNORMALITIES | 1 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically ECG Abnormalities | Morphology - POSSIBLE RIGHT VENTRICULAR HYPERTROPHY | 1 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically ECG Abnormalities | Morphology - RIGHT VENTRICULAR HYPERTROPHY | 1 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically ECG Abnormalities | Myocardial Ischemia/Infarction - ANTEROSEPTAL MYOCARDIAL INFARCTION - AGE UNDETERMINED | 1 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically ECG Abnormalities | Rhythm - VENTRICULAR PREMATURE BEATS | 1 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically ECG Abnormalities | ST-T Wave - COUNTERCLOCKWISE ROTATION | 1 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically ECG Abnormalities | ST-T Wave - EARLY REPOLARIZATION | 1 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically ECG Abnormalities | ST-T Wave - NON-SPECIFIC ST ABNORMALITY | 2 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically ECG Abnormalities | ST-T Wave - NON-SPECIFIC ST AND T WAVE ABNORMALITY | 1 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically ECG Abnormalities | ST-T Wave - NON-SPECIFIC T WAVE ABNORMALITY | 14 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically ECG Abnormalities | Myocardial Isch/Infar. CANNOT RULE OUT POSSIBLE MYOCARDIAL INFARCTION MAY BE IMPROPER LEAD POSITIONS | 1 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically ECG Abnormalities | Myocardial Ischemia/Infarction - INFERIOR MYOCARDIAL INFARCTION - AGE UNDETERMINED | 1 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically ECG Abnormalities | Myocardial Ischemia/Infarction - OLD INFERIOR MYOCARDIAL INFARCTION | 1 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically ECG Abnormalities | Other - POOR PRECORDIAL R WAVE PROGRESSION. POSSIBLE IMPROPER PLACEMENT OF V LEADS. | 1 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically ECG Abnormalities | Other - POOR R WAVE PROGRESSION, PRECORDIAL LEADS | 3 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically ECG Abnormalities | QT Interval - QTCB BORDERLINE PROLONGED | 9 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically ECG Abnormalities | Rhythm - ATRIAL FLUTTER WITH 3:1 VENTRICULAR RESPONSE | 1 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically ECG Abnormalities | Rhythm - FREQUENT VENTRICULAR PREMATURE BEATS | 1 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically ECG Abnormalities | Rhythm - RARE VENTRICULAR PREMATURE BEATS | 1 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically ECG Abnormalities | Rhythm - SINUS BRADYCARDIA | 2 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically ECG Abnormalities | Rhythm - SINUS TACHYCARDIA | 25 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically ECG Abnormalities | Rhythm - UNUSUAL P AXIS, POSSIBLE ECTOPIC ATRIAL RHYTHM | 1 Participants |
Primary
Number of Participants With Potentially Clinically Significant Hematology Results
Number of Participants with Potentially Clinically Significant Hematology Results by Treatment Group. Percentages based on number of patients with at least one non-missing post-baseline value in each treatment group. Patients are only counted once per criterion per laboratory test.
Time frame: 180 weeks
Population: Number of subjects evaluated is the number of subjects who had a baseline assessment and at least one post-baseline assessment.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically Significant Hematology Results | WBC (<3.0 x 10^9/L) | 5 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically Significant Hematology Results | WBC (>=16.0 x 10^9/L) | 2 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically Significant Hematology Results | Neutrophils (<1.5 x 10^9/L) | 3 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically Significant Hematology Results | Neutrophils (>13.5 x 10^9/L) | 4 Participants |
Primary
Number of Participants With Potentially Clinically Significant Liver Enzyme Abnormalities
Number of participants with potentially clinically significant liver enzyme abnormalities for the safety population are presented. Percentages are based on the number of patients with at least one non-missing, post-baseline value for each parameter.
Time frame: 180 weeks
Population: Number of subjects evaluated is the number of subjects who had a baseline assessment and at least one post-baseline assessment.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically Significant Liver Enzyme Abnormalities | ALT >3 times ULN | 5 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically Significant Liver Enzyme Abnormalities | AST >3 times ULN | 2 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically Significant Liver Enzyme Abnormalities | Total Bilirubin >1.5 times ULN | 3 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically Significant Liver Enzyme Abnormalities | Creatinine >1.5 times ULN | 0 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically Significant Liver Enzyme Abnormalities | ALT or AST >3 times ULN and total bilirubin >2 times ULN (Hy's Law) | 0 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically Significant Liver Enzyme Abnormalities | Alkaline phosphatase >2.5 times ULN (upper limit of the 'normal' reference range) | 1 Participants |
Primary
Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities
Number of participants with potentially clinically significant vital sign abnormalities for the safety population are presented. Percentages are based on the number of patients with at least one non-missing, post-baseline value for each parameter.
Time frame: 180 weeks
Population: Number of subjects evaluated is the number of subjects in the Safety Population who had a baseline assessment and at least one post-baseline assessment.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities | Pulse Rate - >120 bpm post-baseline or an increase from baseline of >20 bpm | 26 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities | Pulse Rate - >120 bpm post-baseline and an increase from baseline of >20 bpm | 1 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities | Pulse Rate - <50 bpm post-baseline or a decrease from baseline of >20 bpm | 9 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities | Systolic Blood Pressure - >180 mmHg post-baseline and an increase from baseline of >40 mmHg | 1 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities | Systolic Blood Pressure - <90 mmHg post-baseline or a decrease from baseline of >30 mmHg | 11 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities | Systolic Blood Pressure - <90 mmHg post-baseline and a decrease from baseline of >30 mmHg | 1 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities | Diastolic Blood Pressure - >105 mmHg post-baseline or an increase from baseline of >30 mmHg | 4 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities | Diastolic Blood Pressure - >105 mmHg post-baseline and an increase from baseline of >30 mmHg | 0 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities | Diastolic Blood Pressure - <50 mmHg post-baseline or a decrease from baseline of >20 mmHg | 13 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities | Weight - Decrease from baseline of >7% | 32 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities | Pulse Rate - <50 bpm post-baseline and a decrease from baseline of >20 bpm | 0 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities | Systolic Blood Pressure - >180 mmHg post-baseline or an increase from baseline of >40 mmHg | 3 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities | Diastolic Blood Pressure - <50 mmHg post-baseline and a decrease from baseline of >20 mmHg | 0 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities | Weight - Increase from baseline of >7% | 4 Participants |
| KNS-760704 300 mg/Day | Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities | Temperature - >38 degree C. post-baseline and an increase from baseline of at least 1 degree Celsius | 4 Participants |
Secondary
Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 12
The ALSFRS-R (ALS functional rating scale with respiratory component) is a validated scale which measures 4 functional domains, comprising respiratory function, bulbar function, gross motor skills, and fine motor skills. There are a total of 12 questions, each scored from 0 to 4 for a total possible score of 48. Therefore, the score ranges from 0 to 48 with higher scores representing better function.
Time frame: 12 weeks
Population: All subjects who received at least 1 dose of study treatment during the study and who at least one valid post-baseline measurement and a measurement at the timepoint being analyzed.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| KNS-760704 300 mg/Day | Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 12 | -2.48 units on a scale | Standard Deviation 3.424 |
Secondary
Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 24
The ALSFRS-R (ALS functional rating scale with respiratory component) is a validated scale which measures 4 functional domains, comprising respiratory function, bulbar function, gross motor skills, and fine motor skills. There are a total of 12 questions, each scored from 0 to 4 for a total possible score of 48. Therefore, the score ranges from 0 to 48 with higher scores representing better function.
Time frame: 24 weeks
Population: All subjects who received at least 1 dose of study treatment during the study and who at least one valid post-baseline measurement and a measurement at the timepoint being analyzed.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| KNS-760704 300 mg/Day | Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 24 | -4.29 units on a scale | Standard Deviation 4.589 |
Secondary
Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 48
The ALSFRS-R (ALS functional rating scale with respiratory component) is a validated scale which measures 4 functional domains, comprising respiratory function, bulbar function, gross motor skills, and fine motor skills. There are a total of 12 questions, each scored from 0 to 4 for a total possible score of 48. Therefore, the score ranges from 0 to 48 with higher scores representing better function.
Time frame: 48 weeks
Population: All subjects who received at least 1 dose of study treatment during the study and who at least one valid post-baseline measurement and a measurement at the timepoint being analyzed.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| KNS-760704 300 mg/Day | Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 48 | -7.49 units on a scale | Standard Deviation 8.476 |
Secondary
Change in McGill Single-Item Scale (SIS) From Baseline to Week 12
The McGill SIS consists of a single question designed to assess the improvement of or the rate of deterioration of the subject's quality-of-life. Subjects rated their quality of life over the prior 2 days on a scale of 0 (very bad) to 10 (excellent). Decreases from Baseline indicate deterioration of a subject's quality of life.
Time frame: 12 weeks
Population: All subjects who received at least 1 dose of study treatment during the study and who at least one valid post-baseline measurement and a measurement at the timepoint being analyzed.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| KNS-760704 300 mg/Day | Change in McGill Single-Item Scale (SIS) From Baseline to Week 12 | -0.1 units on a scale | Standard Deviation 1.48 |
Secondary
Change in McGill Single-Item Scale (SIS) From Baseline to Week 24
The McGill SIS consists of a single question designed to assess the improvement of or the rate of deterioration of the subject's quality-of-life. Subjects rated their quality of life over the prior 2 days on a scale of 0 (very bad) to 10 (excellent). Decreases from Baseline indicate deterioration of a subject's quality of life.
Time frame: 24 weeks
Population: All subjects who received at least 1 dose of study treatment during the study and who at least one valid post-baseline measurement and a measurement at the timepoint being analyzed.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| KNS-760704 300 mg/Day | Change in McGill Single-Item Scale (SIS) From Baseline to Week 24 | -0.5 units on a scale | Standard Deviation 2.05 |
Secondary
Change in McGill Single-Item Scale (SIS) From Baseline to Week 48
The McGill SIS consists of a single question designed to assess the improvement of or the rate of deterioration of the subject's quality-of-life. Subjects rated their quality of life over the prior 2 days on a scale of 0 (very bad) to 10 (excellent). Decreases from Baseline indicate deterioration of a subject's quality of life.
Time frame: 48 weeks
Population: All subjects who received at least 1 dose of study treatment during the study and who at least one valid post-baseline measurement and a measurement at the timepoint being analyzed.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| KNS-760704 300 mg/Day | Change in McGill Single-Item Scale (SIS) From Baseline to Week 48 | -0.8 units on a scale | Standard Deviation 1.76 |
Secondary
Change in Upright Vital Capacity From Baseline to Week 12
Change in Percent Predicted Upright Vital Capacity from Baseline to Week 12. A negative change indicates clinical worsening.
Time frame: 12 weeks
Population: All subjects who received at least 1 dose of study treatment during the study and who at least one valid post-baseline measurement and a measurement at the timepoint being analyzed.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| KNS-760704 300 mg/Day | Change in Upright Vital Capacity From Baseline to Week 12 | -7.7 percentage of predicted vital capacity | Standard Deviation 14.03 |
Secondary
Change in Upright Vital Capacity From Baseline to Week 24
Change in Percent Predicted Upright Vital Capacity from Baseline to Week 24. A negative change indicates clinical worsening.
Time frame: 24 weeks
Population: All subjects who received at least 1 dose of study treatment during the study and who at least one valid post-baseline measurement and a measurement at the timepoint being analyzed.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| KNS-760704 300 mg/Day | Change in Upright Vital Capacity From Baseline to Week 24 | -12.5 percentage of predicted vital capacity | Standard Deviation 18.9 |
Secondary
Change in Upright Vital Capacity From Baseline to Week 48
Change in Percent Predicted Upright Vital Capacity from Baseline to Week 48. A negative change indicates clinical worsening.
Time frame: 48 weeks
Population: All subjects who received at least 1 dose of study treatment during the study and who at least one valid post-baseline measurement and a measurement at the timepoint being analyzed.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| KNS-760704 300 mg/Day | Change in Upright Vital Capacity From Baseline to Week 48 | -15.8 percentage of predicted vital capacity | Standard Deviation 17.59 |
Secondary
Number of Subjects With Feeding Tube Placed During the Study.
Number of participants who had a feeding tube placed during the study.
Time frame: 144 weeks
Population: Randomized subjects who had at least 1 post-baseline clinical status evaluation. Subjects who had a feeding tube in place at the beginning of the study were not included in this analysis.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| KNS-760704 300 mg/Day | Number of Subjects With Feeding Tube Placed During the Study. | 24 participants |