Clinical Trial of  KNS-760704

The purpose of the study is to evaluate safety, tolerability and effect of oral KNS-760704 in amyotrophic lateral sclerosis (ALS). It is known that muscle-controlling nerve cells called motor neurons die in the brains and spinal cords of people with ALS. However, the cause of the cell death is unknown.

Investigators at Knopp Neurosciences say this small- molecule drug, KNS-760704, has demonstrated neuroprotective properties. Phase 1 studies in healthy volunteers have shown that up to 300 milligrams of the drug was safe and well tolerated. Recently, a similar drug was found to be safe and well tolerated in 100 people with ALS and suggested that the functional changes were slowed in patient on drug. This study will evaluate the potential benefits of KNS-760704 over a broad dose range in 80 patients with ALS.


This is a double-blind, randomized, placebo-controlled study. This means that neither participants nor investigators will know who is receiving KNS-760704 and who is not; and that a portion of the study participants will be randomly assigned to receive KNS-760704, while another portion will receive a placebo (an inert substance that looks like the study drug).

This trial will be conducted in two parts. In part 1, approximately 80 eligible patients will be randomized to one of four treatment groups for 12 weeks of treatment. The groups will be placebo, or low-dose, mid-dose or high-dose KNS-760704.

Participants meeting eligibility requirements will be enrolled at approximately 20 centers in theUnited States. In addition to the visit to determine eligibility and the first visit to take study drug, participants will be required to make five additional research clinic visits during part 1. 

Participants who complete all 12 weeks of part 1 will be eligible for a group assignment in part 2. The duration of part 2 of the study is 28 weeks. Participants will be randomly assigned to one of two active treatment groups (low-dose or high-dose KNS-760704) for 24 weeks and to a placebo group for the remaining four-week period in part 2. Participants will not be told when the four weeks of placebo treatment will be given. During part 2, participants will be required to make six research clinic visits, including the baseline visit.

During each visit throughout both parts of the study, participants will be subjected to a series of tests designed to study the safety of the study drug, as well and undergo tests to evaluate the effects of KNS-760704 on measures of clinical function (for example, respiratory capacity, quality of life and general functioning).

To be eligible participants must be diagnosed with ALS and have first been seen for ALS symptoms within the last 24 months. The upright vital capacity (breathing measurement) must be greater than 65 percent of normal. People cannot participate if they are on non-invasive ventilation or are taking other clinical trial medications including Lithium.

If you are interested please contact:

Drexel University College Of Medicine
Philadelphia, Pennsylvania, United States, 19102    
Contact: Christine Barr    
Phone: 215-762-5186    




The ALS Association Greater Philadelphia Chapter
321 Norristown Road - Suite 260, Ambler, PA 19002

The ALS Association is a 501(c)3 nonprofit organization and donations are tax deductible to the full extent of the law.