NCT03280056

Safety and Efficacy of Repeated Administrations of NurOwn® in ALS Patients

A Phase 3, Randomized Double-Blind, Placebo-Controlled Multicenter Study to Evaluate Efficacy and Safety of Repeated Administration of NurOwn® (Autologous Mesenchymal Stem Cells Secreting Neurotrophic Factors) in Participants With ALS

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 196 (actual)
• Sponsor: Brainstorm-Cell Therapeutics · Industry
• Sex: All
• Age: 18 Years – 60 Years
• Healthy volunteers: Not accepted

Summary

This study will evaluate the safety and efficacy of repeated administration of NurOwn® (MSC-NTF cells) therapy, which is based on transplantation of autologous bone marrow derived mesenchymal stromal cells (MSC), which are enriched from the patient's own bone marrow, propagated ex vivo and induced to secrete Neurotrophic factors (NTFs). The autologous NurOwn® (MSC-NTF cells) are back-transplanted into the patient intrathecally by standard lumbar puncture where neurons and glial cells are expected to take up the neurotrophic factors secreted by the transplanted cells

Detailed description

Neurotrophic factors (NTFs) are potent survival factors for embryonic, neonatal, and adult neurons and are considered potential therapeutic candidates for ALS. Delivery of multiple NTFs to the immediate environment of afflicted neurons in ALS patients is expected to improve their survival and thus slow down disease progression and alleviate symptoms. NTF-secreting mesenchymal stromal cells (MSC-NTF cells) are a novel cell-therapeutic approach aimed at effectively delivering NTFs directly to the site of damage in ALS patients. Participants meeting the inclusion and exclusion criteria will be randomized and will undergo bone-marrow aspiration. MSC of the participants randomized to the treatment group will be induced into MSC-NTF cells. Participants will undergo a total of three intrathecal (IT) transplantations with NurOwn® (MSC-NTF cells) or matching placebo at three bi-monthly intervals

Conditions

• Amyotrophic Lateral Sclerosis (ALS)

Interventions

Type	Name	Description
BIOLOGICAL	NurOwn® (MSC-NTF cells)	NurOwn® (MSC-NTF): One course of treatment that includes three separate intrathecal injections of 100-125 x 10\^6 cells every 8 weeks NurOwn® (MSC-NTF): Autologous, bone marrow-derived, mesenchymal stem cells secreting neurotrophic factors
OTHER	Placebo	One course of treatment that includes three separate intrathecal injections of Placebo every 8 weeks Placebo: liquid solution in syringe for injection
OTHER	Bone Marrow aspiration	Bone Marrow aspiration

Timeline

• Start date: 2017-08-28
• Primary completion: 2020-09-29
• Completion: 2020-09-29
• First posted: 2017-09-12
• Last updated: 2024-02-29
• Results posted: 2024-02-29

Locations

6 sites across 1 country: United States

Regulatory

• FDA-regulated drug study