Clinical trial NCT00931944: Open-Label, Safety and Tolerability Extension Study of KNS-760704 in Amyotrophic Lateral Sclerosis (ALS) (CL211)
Official title: An Open-Label, Safety and Tolerability, Study Evaluating KNS-760704 in Patients With Amyotrophic Lateral Sclerosis (ALS)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: 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
Lead sponsor: Knopp Biosciences
Sponsor class: INDUSTRY
First posted: 2009-07-02
Enrollment: 74
Sex: ALL
Minimum age (years): 21
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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)

Primary outcomes:
measure: Number of Participants With Potentially Clinically Significant Hematology Results
timeFrame: 180 weeks
measure: Number of Participants With Potentially Clinically Significant Liver Enzyme Abnormalities
timeFrame: 180 weeks
measure: Number of Participants With Potentially Clinically ECG Abnormalities
timeFrame: 180 weeks
measure: Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities
timeFrame: 180 weeks
Secondary outcomes:
measure: Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 12
timeFrame: 12 weeks
measure: Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 24
timeFrame: 24 weeks
measure: Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 48
timeFrame: 48 weeks
measure: Change in Upright Vital Capacity From Baseline to Week 12
timeFrame: 12 weeks
measure: Change in Upright Vital Capacity From Baseline to Week 24
timeFrame: 24 weeks
measure: Change in Upright Vital Capacity From Baseline to Week 48
timeFrame: 48 weeks
measure: Change in McGill Single-Item Scale (SIS) From Baseline to Week 12
timeFrame: 12 weeks
measure: Change in McGill Single-Item Scale (SIS) From Baseline to Week 24
timeFrame: 24 weeks
measure: Change in McGill Single-Item Scale (SIS) From Baseline to Week 48
timeFrame: 48 weeks
measure: Number of Subjects With Feeding Tube Placed During the Study.
timeFrame: 144 weeks
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: University of Arkansas for Medical Sciences
state: Arkansas
city: Los Angeles
country: United States
facility: UCLA, Dept. of Neurology - Neuromuscular/ALS Research Center
state: California
city: San Francisco
country: United States
facility: The Forbes Norris MDA/ALS Research Center
state: California
city: Miami
country: United States
facility: University of Miami Miller School of Medicine
state: Florida
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Baltimore
country: United States
facility: Johns Hopkins University School of Medicine
state: Maryland
city: Boston
country: United States
facility: Massachusettes General Hospital
state: Massachusetts
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: Lincoln
country: United States
facility: Bryan LGH Medical Center East
state: Nebraska
city: New York
country: United States
facility: Columbia University, Lou Gehrig MDA/ALS Research Center
state: New York
city: Syracuse
country: United States
facility: SUNY Upstate Medical University
state: New York
city: Hershey
country: United States
facility: Penn State Hershey Medical Center
state: Pennsylvania
city: Philadelphia
country: United States
facility: Drexel University College Of Medicine
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh School of Medicine
state: Pennsylvania
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: San Antonio
country: United States
facility: University of Texas Health Sciences Center of San Antonio
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
city: Charlottesville
country: United States
facility: University of Virginia Health System
state: Virginia
city: Seattle
country: United States
facility: University of Washington
state: Washington
Study officials:
affiliation: NeuroClinical Trials Unit (Massachusetts General Hospital)
name: Merit Cudkowicz, M.D., MSc
role: PRINCIPAL_INVESTIGATOR
References: