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

Study Purpose:

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.

Study Status:

Not recruiting


Amyotrophic Lateral Sclerosis

Study Type:


Type of Intervention:


Intervention Name:




Phase 2

Study Chair(s)/Principal Investigator(s):

Merit Cudkowicz, M.D., MSc, NeuroClinical Trials Unit (Massachusetts General Hospital) ID:


Neals Affiliated?


Coordinating Center Contact Information


149 13th Street, Bldg 149, MGH East
Charlestown, Massachusetts, 02129 United States

Full Study Summary:

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.

Study Sponsor:

Knopp Biosciences

Participant Duration:

up to 180 weeks

Estimated Enrollment:


Estimated Study Start Date:

05 / 31 / 2009

Estimated Study Completion Date:

07 / 01 / 2013

Posting Last Modified Date:

08 / 16 / 2021

Date Study Added to

07 / 02 / 2009

Minimum Age:

21 Years

Maximum Age:

80 Years

Can participants use Riluzole?


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)

University of Arkansas for Medical Sciences

Little Rock, Arkansas 72205
United States

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

Los Angeles, California 90095
United States

The Forbes Norris MDA/ALS Research Center

San Francisco, California 94115
United States

University of Miami Miller School of Medicine

Miami, Florida 33136
United States

University of Kansas Medical Center

Kansas City, Kansas 66160
United States

Johns Hopkins University School of Medicine

Baltimore, Maryland 21228
United States

Massachusettes General Hospital

Boston, Massachusetts 02129
United States

Washington University School of Medicine

Saint Louis, Missouri 63110
United States

Bryan LGH Medical Center East

Lincoln, Nebraska 68506
United States

Columbia University, Lou Gehrig MDA/ALS Research Center

New York, New York 10032
United States

SUNY Upstate Medical University

Syracuse, New York 13210
United States

Penn State Hershey Medical Center

Hershey, Pennsylvania 17033
United States

Drexel University College Of Medicine

Philadelphia, Pennsylvania 19102
United States

University of Pittsburgh School of Medicine

Pittsburgh, Pennsylvania 15213
United States

Vanderbilt University Medical Center

Nashville, Tennessee 37232
United States

University of Texas Health Sciences Center of San Antonio

San Antonio, Texas 78229
United States

University of Utah

Salt Lake City, Utah 84132
United States

University of Virginia Health System

Charlottesville, Virginia 22908
United States

University of Washington

Seattle, Washington 98195
United States