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Efficacy and Safety of MSC-NTF (NurOwn) in Participants With Early Symptomatic ALS and Moderate Disease Presentation in ALS (ENDURANCE STUDY)
Sponsor: Brainstorm-Cell Therapeutics
Summary
The goal of this two-part clinical trial is: 1\) to evaluate the safety and efficacy of Debamestrocel - MSC-NTF (NurOwn) compared to placebo in participants with early symptomatic ALS and moderate disease presentation in ALS; followed by 2) further evaluation by providing NurOwn to all participants in an open label extension period. Researchers will compare NurOwn to a placebo (a look-alike substance that contains no drug) to evaluate the efficacy of NurOwn compared to placebo in the treatment of participants with ALS. Participants will: Receive NurOwn or a placebo every 8 weeks for 24 weeks. After that, every participant will receive NurOwn every 8 weeks for an additional 24 weeks. They will visit the clinic approximately every 8 weeks for checkups and tests.
Official title: A Two-Part Phase 3b Randomized, Double-Blind, Placebo-Controlled, Followed by Open-Label Extension, Multicenter Study of the Efficacy and Safety of MSC-NTF (NurOwn) in Participants With With Early Symptomatic and Moderate Disease Presentation in Amyotrophic Lateral Sclerosis (ALS)
Key Details
Gender
All
Age Range
18 Years - 75 Years
Study Type
INTERVENTIONAL
Enrollment
200
Start Date
2025-06-30
Completion Date
2029-05-01
Last Updated
2025-05-22
Healthy Volunteers
No
Interventions
Debamestrocel - MSC-NTF (NurOwn)
MSC-NTF cells suspended in excipient Dulbecco Modified Eagle Medium (DMEM).
Placebo
Dulbecco Modified Eagle Medium (DMEM).
Locations (15)
Barrow Neurological Institute
Phoenix, Arizona, United States
University of California San Diego Medical Center
La Jolla, California, United States
University of Southern California
Los Angeles, California, United States
California Pacific Medical Center
San Francisco, California, United States
University of California, San Francisco
San Francisco, California, United States
University of Colorado Anschutz Medical Campus School of Medicine
Aurora, Colorado, United States
Nova Southeastern University
Davie, Florida, United States
Mayo Clinic
Jacksonville, Florida, United States
University of South Florida
Tampa, Florida, United States
Northwestern Medicine
Chicago, Illinois, United States
Sean M. Healey & AMG Center For ALS At Massachusetts General Hospital
Boston, Massachusetts, United States
University of Massachusetts Medical School
Worcester, Massachusetts, United States
Mayo Clinic
Rochester, Minnesota, United States
Providence ALS Center
Portland, Oregon, United States
Temple University Of The Commonwealth System of Higher Education
Philadelphia, Pennsylvania, United States