This is the classic website, which will be retired eventually. Please visit the modernized instead.
Working… Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03280056
Recruitment Status : Completed
First Posted : September 12, 2017
Last Update Posted : October 6, 2021
California Institute for Regenerative Medicine (CIRM)
Information provided by (Responsible Party):
Brainstorm-Cell Therapeutics

Brief 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

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis (ALS) Biological: NurOwn® (MSC-NTF cells) Other: Placebo Other: Bone Marrow aspiration Phase 3

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

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 263 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Randomized, Double-Blind, Placebo-Controlled Multicenter Study
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: This is a double-blind study where the investigators, participants and all sponsor and CRO personnel involved in the conduct, data management or analysis of the study will remain blinded to the treatment assignments
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : August 28, 2017
Actual Primary Completion Date : October 30, 2020
Actual Study Completion Date : October 30, 2020

Arm Intervention/treatment
Active Comparator: NurOwn® (MSC-NTF cells)
Three Intrathecal administrations of NurOwn® (MSC-NTF cells) at bi-monthly intervals
Biological: NurOwn® (MSC-NTF cells)
Autologous transplantation of bone marrow derived mesenchymal stem cells propagated ex vivo and induced to secrete neurotrophic factors

Other: Bone Marrow aspiration
Bone Marrow aspiration

Placebo Comparator: Placebo
Three Intrathecal administrations of Placebo at bi-monthly intervals
Other: Placebo

Other: Bone Marrow aspiration
Bone Marrow aspiration

Primary Outcome Measures :
  1. To evaluate the efficacy and safety of NurOwn® (autologous MSC-NTF cells) as compared to placebo as measured by the amyotrophic lateral sclerosis functional rating scale (ALSFRS-R) [ Time Frame: 28 weeks following the first treatment ]
    To determine efficacy and safety of repeat intrathecal injections of NurOwn® as compared to Placebo given three times two months apart to participants with Amyotrophic Lateral Sclerosis

Secondary Outcome Measures :
  1. Biomarkers [ Time Frame: Through selected post-treatment time points up to 20 weeks post transplant ]
    To evaluate biomarkers (such as cell-secreted neurothrophic factors, inflammatory factors, and cytokines in pg/ml) in the cerebrospinal fluid (CSF) as well as in serum samples throughout the study to evaluate their relationship to treatment with NurOwn® (MSC-NTF cells)

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   18 Years to 60 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • ALS diagnosed as possible, laboratory-supported probable, probable, or definite as defined by revised El Escorial criteria.
  • Having onset of ALS disease symptoms, including limb weakness within 24 months at the Screening Visit.
  • ALSFRS-R ≥ 25 at the screening Visit.
  • Upright slow vital capacity (SVC) measure ≥ 65% of predicted for gender, height, and age at the screening Visit.
  • Rapid progressors
  • Participants taking a stable dose of Riluzole are permitted in the study
  • Citizen or permanent resident of the United States or Canadian citizen able to travel to a US site for all follow-up study visits

Exclusion Criteria:

  • Prior stem cell therapy of any kind
  • History of autoimmune or other serious disease (including malignancy and immune deficiency) that may confound study results
  • Current use of immunosuppressant medication or anticoagulants (per Investigator discretion)
  • Exposure to any other experimental agent or participation in an ALS clinical trial within 30 days prior to Screening Visit
  • Use of RADICAVA (edaravone injection) within 30 days of screening or intent to use edaravone at any time during the course of the study including the follow up period
  • Use of non-invasive ventilation (BIPAP), diaphragm pacing system or invasive ventilation (tracheostomy)
  • Feeding tube
  • Pregnant women or women currently breastfeeding

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03280056

Layout table for location information
United States, California
University of California Irvine Alpha Stem Cell Clinic
Irvine, California, United States, 92697
Cedars-Sinai Medical Center
Los Angeles, California, United States, 90048
California Pacific Medical Center
San Francisco, California, United States, 94115
United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States, 02115
University of Massachusetts Medical School
Worcester, Massachusetts, United States, 01655
United States, Minnesota
Mayo Clinic
Rochester, Minnesota, United States, 55905
Sponsors and Collaborators
Brainstorm-Cell Therapeutics
California Institute for Regenerative Medicine (CIRM)
Layout table for investigator information
Principal Investigator: Merit E. Cudkowicz, MD Massachusetts General Hospital
Principal Investigator: Robert H. Brown, MD, PhD UMass Medical School
Principal Investigator: Anthony J. Windebank, MD Mayo Clinic
Principal Investigator: Namita A. Goyal, MD UC Irvine
Principal Investigator: Robert G. Miller, MD California Pacific Medical Center (CPM) Research Institute
Principal Investigator: Robert Baloh, MD, Ph.D. Cedars-Sinai Medical Center
Layout table for additonal information
Responsible Party: Brainstorm-Cell Therapeutics Identifier: NCT03280056    
Other Study ID Numbers: BCT-002-US
First Posted: September 12, 2017    Key Record Dates
Last Update Posted: October 6, 2021
Last Verified: October 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Brainstorm-Cell Therapeutics:
Neurotrophic factors
Additional relevant MeSH terms:
Layout table for MeSH terms
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases