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
Sponsor:
Brief Summary:
(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
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
Locations
- 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
