Open-Label, Safety and Tolerability Extension Study of KNS-760704 in Amyotrophic Lateral Sclerosis (ALS) (CL211)
- Registration Number
- NCT00931944
- Lead Sponsor
- Knopp Biosciences
- 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 74
- Patient has provided signed informed consent for this trial before the commencement of any study-related procedure
- 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
- Patient did not participate in Knopp Protocol KNS-760704-CL201 or patient did not receive RTPB under Research IND #60,948.
- Patient discontinued from KNS-760704-CL201 for any reason other than enrollment into this study (applies to patients enrolled in KNS-760704-CL201 only)
- 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)
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description KNS-760704 300 mg/day KNS-760704 Open-label KNS-760704 (150 mg Q12H)
- Primary Outcome Measures
Name Time Method 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 Outcome Measures
Name Time Method 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.
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 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 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 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.
Trial Locations
- Locations (19)
SUNY Upstate Medical University
🇺🇸Syracuse, New York, United States
Penn State Hershey Medical Center
🇺🇸Hershey, Pennsylvania, United States
Columbia University, Lou Gehrig MDA/ALS Research Center
🇺🇸New York, New York, United States
Johns Hopkins University School of Medicine
🇺🇸Baltimore, Maryland, United States
Massachusettes General Hospital
🇺🇸Boston, Massachusetts, United States
Washington University School of Medicine
🇺🇸Saint Louis, Missouri, 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
Vanderbilt University Medical Center
🇺🇸Nashville, Tennessee, United States
University of Texas Health Sciences Center of San Antonio
🇺🇸San Antonio, Texas, United States
University of Utah
🇺🇸Salt Lake City, Utah, United States
UCLA, Dept. of Neurology - Neuromuscular/ALS Research Center
🇺🇸Los Angeles, California, United States
University of Arkansas for Medical Sciences
🇺🇸Little Rock, Arkansas, United States
Bryan LGH Medical Center East
🇺🇸Lincoln, Nebraska, United States
University of Pittsburgh School of Medicine
🇺🇸Pittsburgh, Pennsylvania, United States
Drexel University College Of Medicine
🇺🇸Philadelphia, Pennsylvania, United States
University of Washington
🇺🇸Seattle, Washington, United States
University of Virginia Health System
🇺🇸Charlottesville, Virginia, United States
University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States