A Phase 2 Open-label Multicenter Study to Evaluate the Safety and Efficacy of Repeated Administration of NurOwn® [Autologous Mesenchymal Stem Cells Secreting Neurotrophic Factors (NTF), MSC-NTF] Cells in Participants With Progressive MS
Neurotrophic Factors (MSC-NTF cells) involving 20 participants with progressive MS at
multiple investigational study sites.
1. Males and females ages 18 to 65 years old, inclusive, at the Screening Visit.
2. Clinical diagnosis of Progressive MS (Primary and Secondary) based on the 2017 revised
MacDonald Criteria and confirmation by the Investigator that the disease has entered
the progressive stage for at least 6 months prior to enrollment.
3. No evidence of clinical MS relapse or high dose pulse corticosteroid treatment within
6 months prior to screening
4. Disability status at screening with an Expanded Disability Status Scale (EDSS)
5. Able to walk 25 feet in 60 seconds or less.
6. Stable dose of non-excluded MS Disease Modifying Therapy for at least 6 months prior
to Screening Visit (Visit 1).
1. Prior stem cell therapy of any kind.
2. Active participation in any other MS interventional study or use of unapproved MS
investigational therapy within 90 days prior to the Screening Visit (Visit 1).
3. Inability to lie flat for the duration of intrathecal cell transplantation and/or bone
marrow biopsy, or inability to tolerate study procedures for any other reason.
4. History of clinically significant autoimmune disease (excluding thyroid disease) that
may confound study results, myelodysplastic or myeloproliferative disorder, leukemia
or lymphoma, whole body irradiation, hip fracture, or severe scoliosis.
5. Any unstable clinically significant medical condition other than multiple sclerosis
(e.g., within six months of Screening Visit (Visit 1), had myocardial infarction,
angina pectoris, and/or congestive heart failure), treatment with anticoagulants that,
in the opinion of the investigator, would compromise the safety of participants.
6. Any history of malignancy within the previous 5 years, except for non-melanoma
localized skin cancers (with no evidence of metastasis, significant invasion, or
reoccurrence within three years of Screening Visit (Visit 1)).
7. Serum aspartate aminotransferase (AST) or alanine aminotransferase (ALT) value >3.0
times the upper normal limit.
8. Serum creatinine value >2.0 times the upper normal limit.
9. Positive test for Hepatitis B (HBV; surface antigen (HBsAg) and antibodies to core
antigen (IgG and IgM anti-HBc)), Hepatitis C (HCV), or human immunodeficiency virus
(HIV) 1 and 2.
10. Current use of immunosuppressant medication or use of such medication within 6 months
of study enrollment (aside from Rituximab or other approved B-cell immunotherapy).
Alemtuzumab (Lemtrada), Cladribine (NDA submitted), Natalizumab (Tysabri), S1P
modulators (Gilenya) are excluded for safety reasons due to the known risk of systemic
autoimmune disease, malignancy, opportunistic infections, and cardiovascular toxicity
associated with these therapies, as well as theoretical effects on MSC-NTF cell homing
and migration, that may be associated with Natalizumab and/or S1P modulators
11. Any history of acquired or inherited immune deficiency syndrome.
12. Any history of either substance abuse within the past year, or unstable psychiatric
disease according to the Investigator’s judgment.
13. Pregnant women or women currently breastfeeding.
14. Subjects for whom MRI is contraindicated (i.e., have a pacemaker or other metallic
implanted device, or are unable to remain in the machine for period of time needed to
acquire a scan.
- University of Southern California, Los Angeles, California, United States, 90033
- Stanford University School of Medicine, Redwood City, California, United States, 943063