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

Study 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

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Stem Cell

Study Status:

Active, currently recruiting

Phase:

Phase III

Study Chair(s)/Principal Investigator(s):

Merit E. Cudkowicz, MD Massachusetts General Hospital
Robert H. Brown, MD, PhD UMass Medical School
Anthony J. Windebank, MD Mayo Clinic
Namita A. Goyal, MD UC Irvine
Robert G. Miller, MD California Pacific Medical Center (CPM) Research Institute

Clinicaltrials.gov ID (11 digit #):

NCT03280056

Neals Affiliated?

No

Coordinating Center Contact Information


Ralph Z. Kern, MD / .(JavaScript must be enabled to view this email address) / 201-488-0460
Yael D. Gothelf, Ph.D. / .(JavaScript must be enabled to view this email address) IL

Full Study Summary:

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.

Study Sponsor:

Brainstorm-Cell Therapeutics

Participant Duration:

Estimated Enrollment:

200

Estimated Study Start Date:

08/28/2017

Estimated Study Completion Date:

07/30/2019

Posting Last Modified Date:

09/18/2017

Date Study Added to alsconsortium.org:

09/18/2017
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    60

    Min Vital Capacity (% predicted normal):

    65

    Time since Symptom Onset:

    <24 months

    Time since Diagnosis:

    N/A

    Can participants use Riluzole?

    Yes


    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

  • Site Contact Information

    University of California Irvine Alpha Stem Cell Clinic
    Robert Zhou / .(JavaScript must be enabled to view this email address) / 949-824-3990
    Irvine, California 92697
    United States

    Massachusetts General Hospital
    Taylor Mezoian / .(JavaScript must be enabled to view this email address) / 617-643-0312
    Boston, Massachusetts 02115
    United States