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

This study is currently recruiting participants.
Verified August 2017 by Brainstorm-Cell Therapeutics
Sponsor:
ClinicalTrials.gov Identifier:
NCT03280056
First Posted: September 12, 2017
Last Update Posted: September 18, 2017
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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Collaborator:
California Institute for Regenerative Medicine
Information provided by (Responsible Party):
Brainstorm-Cell Therapeutics
  Purpose

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 Intervention Phase
Amyotrophic Lateral Sclerosis (ALS) Biological: NurOwn® (MSC-NTF cells) Other: Placebo Phase 3

Study Type: Interventional
Study Design: 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

Resource links provided by NLM:


Further study details as provided by Brainstorm-Cell Therapeutics:

Primary Outcome Measures:
  • 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:
  • 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)


Estimated Enrollment: 200
Actual Study Start Date: August 28, 2017
Estimated Study Completion Date: July 30, 2019
Estimated Primary Completion Date: April 30, 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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
Placebo Comparator: Placebo
Three Intrathecal administrations of Placebo at bi-monthly intervals
Other: Placebo
Placebo

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

  Eligibility

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.


Ages Eligible for Study:   18 Years to 60 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.
  • Participants taking a stable dose of Riluzole are permitted in the study

Exclusion Criteria:

  • Prior stem cell therapy of any kind
  • History of autoimmune or other serious disease (including malignancy and immune deficiency)
  • 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
  Contacts and Locations
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 ClinicalTrials.gov identifier (NCT number): NCT03280056


Contacts
Contact: Ralph Z. Kern, MD 201-488-0460 rkern@brainstorm-cell.com
Contact: Yael D. Gothelf, Ph.D. ygothelf@brainstorm-cell.com

Locations
United States, California
University of California Irvine Alpha Stem Cell Clinic Recruiting
Irvine, California, United States, 92697
Contact: Robert Zhou    949-824-3990    stemcell@uci.edu   
Principal Investigator: Namita A. Goyal, MD         
United States, Massachusetts
Massachusetts General Hospital Recruiting
Boston, Massachusetts, United States, 02115
Contact: Taylor J Mezoian    617-643-0312    TMEZOIAN@mgh.harvard.edu   
Principal Investigator: James D Berry, MD         
Sponsors and Collaborators
Brainstorm-Cell Therapeutics
California Institute for Regenerative Medicine
Investigators
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
  More Information

Responsible Party: Brainstorm-Cell Therapeutics
ClinicalTrials.gov Identifier: NCT03280056     History of Changes
Other Study ID Numbers: BCT-002-US
First Submitted: August 29, 2017
First Posted: September 12, 2017
Last Update Posted: September 18, 2017
Last Verified: August 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Brainstorm-Cell Therapeutics:
MSC
Autologous
Neurotrophic factors

Additional relevant 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