Open-Label, Safety and Tolerability Extension Study of KNS-760704 in Amyotrophic Lateral Sclerosis (ALS) (CL211)

Amyotrophic Lateral Sclerosis Clinical Trial

— CL211  

Official title:

An Open-Label, Safety and Tolerability, Study Evaluating KNS-760704 in Patients With Amyotrophic Lateral Sclerosis (ALS)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00931944
• Other study ID #: KNS-760704-CL211
• Status: Completed
• Phase: Phase 2
• Start date: July 2009
• Est. completion date: July 2013

Study information

• Verified date: July 2021
• Source: Knopp Biosciences
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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 information / eligibility

• Status: Completed
• Enrollment: 74
• Est. completion date: July 2013
• Est. primary completion date: July 2013
• Accepts healthy volunteers: No
• Gender: All
• Age group: 21 Years to 80 Years

Eligibility

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)

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• Sclerosis

Intervention

Drug:
• KNS-760704
150 mg Q12H KNS-760704 given orally (300 mg total daily dose)

Locations

Country	Name	City	State
United States	Johns Hopkins University School of Medicine	Baltimore	Maryland
United States	Massachusettes General Hospital	Boston	Massachusetts
United States	University of Virginia Health System	Charlottesville	Virginia
United States	Penn State Hershey Medical Center	Hershey	Pennsylvania
United States	University of Kansas Medical Center	Kansas City	Kansas
United States	Bryan LGH Medical Center East	Lincoln	Nebraska
United States	University of Arkansas for Medical Sciences	Little Rock	Arkansas
United States	UCLA, Dept. of Neurology - Neuromuscular/ALS Research Center	Los Angeles	California
United States	University of Miami Miller School of Medicine	Miami	Florida
United States	Vanderbilt University Medical Center	Nashville	Tennessee
United States	Columbia University, Lou Gehrig MDA/ALS Research Center	New York	New York
United States	Drexel University College Of Medicine	Philadelphia	Pennsylvania
United States	University of Pittsburgh School of Medicine	Pittsburgh	Pennsylvania
United States	Washington University School of Medicine	Saint Louis	Missouri
United States	University of Utah	Salt Lake City	Utah
United States	University of Texas Health Sciences Center of San Antonio	San Antonio	Texas
United States	The Forbes Norris MDA/ALS Research Center	San Francisco	California
United States	University of Washington	Seattle	Washington
United States	SUNY Upstate Medical University	Syracuse	New York

Sponsors (1)

Lead Sponsor	Collaborator
Knopp Biosciences

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
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.	180 weeks
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.	180 weeks
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.	180 weeks
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.	180 weeks
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.	12 weeks
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.	24 weeks
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.	48 weeks
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.	12 weeks
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.	24 weeks
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.	48 weeks
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.	12 weeks
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.	24 weeks
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.	48 weeks
Secondary	Number of Subjects With Feeding Tube Placed During the Study.	Number of participants who had a feeding tube placed during the study.	144 weeks

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