search
Back to results

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

Primary Purpose

Amyotrophic Lateral Sclerosis

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
KNS-760704
Sponsored by
Knopp Biosciences
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Amyotrophic Lateral Sclerosis focused on measuring ALS, Amyotrophic Lateral Sclerosis, Lou Gehrig, Lou Gehrig's, Lou Gehrig's disease, Motor Neuron Disease, Nervous System Diseases, KNS-760704, RTPB

Eligibility Criteria

21 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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)

Sites / Locations

  • University of Arkansas for Medical Sciences
  • UCLA, Dept. of Neurology - Neuromuscular/ALS Research Center
  • The Forbes Norris MDA/ALS Research Center
  • University of Miami Miller School of Medicine
  • University of Kansas Medical Center
  • Johns Hopkins University School of Medicine
  • Massachusettes General Hospital
  • Washington University School of Medicine
  • Bryan LGH Medical Center East
  • Columbia University, Lou Gehrig MDA/ALS Research Center
  • SUNY Upstate Medical University
  • Penn State Hershey Medical Center
  • Drexel University College Of Medicine
  • University of Pittsburgh School of Medicine
  • Vanderbilt University Medical Center
  • University of Texas Health Sciences Center of San Antonio
  • University of Utah
  • University of Virginia Health System
  • University of Washington

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

KNS-760704 300 mg/day

Arm Description

Open-label KNS-760704 (150 mg Q12H)

Outcomes

Primary Outcome Measures

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.
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.
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.
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.

Secondary Outcome Measures

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.
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.
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.
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.
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.
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.
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.
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.
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.
Number of Subjects With Feeding Tube Placed During the Study.
Number of participants who had a feeding tube placed during the study.

Full Information

First Posted
June 30, 2009
Last Updated
July 22, 2021
Sponsor
Knopp Biosciences
search

1. Study Identification

Unique Protocol Identification Number
NCT00931944
Brief Title
Open-Label, Safety and Tolerability Extension Study of KNS-760704 in Amyotrophic Lateral Sclerosis (ALS) (CL211)
Acronym
CL211
Official Title
An Open-Label, Safety and Tolerability, Study Evaluating KNS-760704 in Patients With Amyotrophic Lateral Sclerosis (ALS)
Study Type
Interventional

2. Study Status

Record Verification Date
July 2021
Overall Recruitment Status
Completed
Study Start Date
July 2009 (undefined)
Primary Completion Date
July 2013 (Actual)
Study Completion Date
July 2013 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Knopp Biosciences

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Amyotrophic Lateral Sclerosis
Keywords
ALS, Amyotrophic Lateral Sclerosis, Lou Gehrig, Lou Gehrig's, Lou Gehrig's disease, Motor Neuron Disease, Nervous System Diseases, KNS-760704, RTPB

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
74 (Actual)

8. Arms, Groups, and Interventions

Arm Title
KNS-760704 300 mg/day
Arm Type
Experimental
Arm Description
Open-label KNS-760704 (150 mg Q12H)
Intervention Type
Drug
Intervention Name(s)
KNS-760704
Other Intervention Name(s)
dexpramipexole
Intervention Description
150 mg Q12H KNS-760704 given orally (300 mg total daily dose)
Primary Outcome Measure Information:
Title
Number of Participants With Potentially Clinically Significant Hematology Results
Description
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
Title
Number of Participants With Potentially Clinically Significant Liver Enzyme Abnormalities
Description
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
Title
Number of Participants With Potentially Clinically ECG Abnormalities
Description
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
Title
Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities
Description
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
Secondary Outcome Measure Information:
Title
Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 12
Description
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
Title
Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 24
Description
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
Title
Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 48
Description
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
Title
Change in Upright Vital Capacity From Baseline to Week 12
Description
Change in Percent Predicted Upright Vital Capacity from Baseline to Week 12. A negative change indicates clinical worsening.
Time Frame
12 weeks
Title
Change in Upright Vital Capacity From Baseline to Week 24
Description
Change in Percent Predicted Upright Vital Capacity from Baseline to Week 24. A negative change indicates clinical worsening.
Time Frame
24 weeks
Title
Change in Upright Vital Capacity From Baseline to Week 48
Description
Change in Percent Predicted Upright Vital Capacity from Baseline to Week 48. A negative change indicates clinical worsening.
Time Frame
48 weeks
Title
Change in McGill Single-Item Scale (SIS) From Baseline to Week 12
Description
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
Title
Change in McGill Single-Item Scale (SIS) From Baseline to Week 24
Description
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
Title
Change in McGill Single-Item Scale (SIS) From Baseline to Week 48
Description
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
Title
Number of Subjects With Feeding Tube Placed During the Study.
Description
Number of participants who had a feeding tube placed during the study.
Time Frame
144 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
21 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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 Exclusion Criteria: 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)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Merit Cudkowicz, M.D., MSc
Organizational Affiliation
NeuroClinical Trials Unit (Massachusetts General Hospital)
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Arkansas for Medical Sciences
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72205
Country
United States
Facility Name
UCLA, Dept. of Neurology - Neuromuscular/ALS Research Center
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
The Forbes Norris MDA/ALS Research Center
City
San Francisco
State/Province
California
ZIP/Postal Code
94115
Country
United States
Facility Name
University of Miami Miller School of Medicine
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
University of Kansas Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
Johns Hopkins University School of Medicine
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21228
Country
United States
Facility Name
Massachusettes General Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02129
Country
United States
Facility Name
Washington University School of Medicine
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Bryan LGH Medical Center East
City
Lincoln
State/Province
Nebraska
ZIP/Postal Code
68506
Country
United States
Facility Name
Columbia University, Lou Gehrig MDA/ALS Research Center
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
SUNY Upstate Medical University
City
Syracuse
State/Province
New York
ZIP/Postal Code
13210
Country
United States
Facility Name
Penn State Hershey Medical Center
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033
Country
United States
Facility Name
Drexel University College Of Medicine
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19102
Country
United States
Facility Name
University of Pittsburgh School of Medicine
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213
Country
United States
Facility Name
Vanderbilt University Medical Center
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Facility Name
University of Texas Health Sciences Center of San Antonio
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78229
Country
United States
Facility Name
University of Utah
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
Facility Name
University of Virginia Health System
City
Charlottesville
State/Province
Virginia
ZIP/Postal Code
22908
Country
United States
Facility Name
University of Washington
City
Seattle
State/Province
Washington
ZIP/Postal Code
98195
Country
United States

12. IPD Sharing Statement

Learn more about this trial

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

We'll reach out to this number within 24 hrs