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

NCT00931944 | Completed Phase 2 Results

• 1 other identifier: KNS-760704-CL211
• Study Type: interventional
• Target: 74
• Locations: 1 country
• Sites: 19

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.

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

Outcome Measures

Primary Outcomes (4)

• 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

• 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

• 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

• 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 Outcomes (10)

• Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 12

  12 weeks

• Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 24

  24 weeks

• Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 48

  48 weeks

• Change in Upright Vital Capacity From Baseline to Week 12

  12 weeks

• Change in Upright Vital Capacity From Baseline to Week 24

  24 weeks

Study Arms (1)

KNS-760704 300 mg/day

EXPERIMENTAL

Open-label KNS-760704 (150 mg Q12H)

Drug: KNS-760704

Interventions

KNS-760704 DRUG

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

Also known as: dexpramipexole

KNS-760704 300 mg/day

Eligibility Criteria

• Age: 21 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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)

Sponsors & Collaborators

• Knopp Biosciences: lead

Study Sites (19)

University of Arkansas for Medical Sciences

Little Rock, Arkansas, 72205, United States

Location

UCLA, Dept. of Neurology - Neuromuscular/ALS Research Center

Los Angeles, California, 90095, United States

Location

The Forbes Norris MDA/ALS Research Center

San Francisco, California, 94115, United States

Location

University of Miami Miller School of Medicine

Miami, Florida, 33136, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

Johns Hopkins University School of Medicine

Baltimore, Maryland, 21228, United States

Location

Massachusettes General Hospital

Boston, Massachusetts, 02129, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Bryan LGH Medical Center East

Lincoln, Nebraska, 68506, United States

Location

Columbia University, Lou Gehrig MDA/ALS Research Center

New York, New York, 10032, United States

Location

SUNY Upstate Medical University

Syracuse, New York, 13210, United States

Location

Penn State Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

Location

Drexel University College Of Medicine

Philadelphia, Pennsylvania, 19102, United States

Location

University of Pittsburgh School of Medicine

Pittsburgh, Pennsylvania, 15213, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

Location

University of Texas Health Sciences Center of San Antonio

San Antonio, Texas, 78229, United States

Location

University of Utah

Salt Lake City, Utah, 84132, United States

Location

University of Virginia Health System

Charlottesville, Virginia, 22908, United States

Location

University of Washington

Seattle, Washington, 98195, United States

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis; Motor Neuron Disease; Nervous System Diseases

Interventions

Dexpramipexole

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Benzothiazoles; Thiazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Results Point of Contact

• Title: Head of Regulatory
• Organization: Knopp Biosciences

greg@knoppbio.com

4124881776

Study Officials

• Merit Cudkowicz, M.D., MSc

  NeuroClinical Trials Unit (Massachusetts General Hospital)

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 30, 2009
• First Posted: July 2, 2009
• Study Start: July 1, 2009
• Primary Completion: July 1, 2013
• Study Completion: July 1, 2013
• Last Updated: August 16, 2021
• Results First Posted: August 16, 2021

Record last verified: 2021-07

Locations

US (19)