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)
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.
Description
This is a multicenter, Phase 3b study to assess the efficacy and safety of NurOwn in participants with early symptomatic ALS and moderate disease presentation in ALS. The study comprises two parts: a 24-week randomized, double blind placebo controlled period (Part A) followed by a 24-week open label expansion period (Part B). Up to approximately 200 participants are planned to be enrolled and randomized 1:1 to the NurOwn and placebo groups in Part A. All eligible participants who complete Part A will have the option of entering Part B. The trial includes a 9-week screening period. After the…
Eligibility
- Age range
- 18–75 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Male and female participants 18 to 75 years old (inclusive) at Screening Visit 1. 2. ALS diagnosed as laboratory-supported probable, clinically probable, or definite as defined by the revised El Escorial criteria. 3. Having onset of ALS symptoms, including muscle weakness, within 24 months from Screening Visit 1. 4. ≥2 points on each item of the ALSFRS-R at the Screening Visit 1. 5. ≤45 points on ALSFRS-R total score at Screening Visit 1. 6. Upright slow vital capacity (SVC) measure ≥65% of predicted for gender, height, and age at Screening Visit 1. 7. Participants must…
Interventions
- BiologicalDebamestrocel - MSC-NTF (NurOwn)
MSC-NTF cells suspended in excipient Dulbecco Modified Eagle Medium (DMEM).
- BiologicalPlacebo
Dulbecco Modified Eagle Medium (DMEM).
Locations (15)
- Barrow Neurological InstitutePhoenix, Arizona
- University of California San Diego Medical CenterLa Jolla, California
- University of Southern CaliforniaLos Angeles, California
- California Pacific Medical CenterSan Francisco, California
- University of California, San FranciscoSan Francisco, California
- University of Colorado Anschutz Medical Campus School of MedicineAurora, Colorado