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.
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
74
150 mg Q12H KNS-760704 given orally (300 mg total daily dose)
University of Arkansas for Medical Sciences
Little Rock, Arkansas, United States
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
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
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
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
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.
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UCLA, Dept. of Neurology - Neuromuscular/ALS Research Center
Los Angeles, California, United States
The Forbes Norris MDA/ALS Research Center
San Francisco, California, United States
University of Miami Miller School of Medicine
Miami, Florida, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
Johns Hopkins University School of Medicine
Baltimore, Maryland, United States
Massachusettes General Hospital
Boston, Massachusetts, United States
Washington University School of Medicine
St Louis, Missouri, United States
Bryan LGH Medical Center East
Lincoln, Nebraska, United States
Columbia University, Lou Gehrig MDA/ALS Research Center
New York, New York, United States
...and 9 more locations
Time frame: 12 weeks
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
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
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
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
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
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
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
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
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