Safety and Efficacy of Repeated Administration of NurOwn (MSC-NTF Cells) in Participants With Progressive MS

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I'm Interested

Trial ID: NCT03799718

Purpose

A multidose open-label study with autologous Mesenchymal Stromal Stem Cells Secreting Neurotrophic Factors (MSC-NTF cells) involving 20 participants with progressive MS at multiple investigational study sites.

Official Title

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

Stanford Investigator(s)

Christopher Lock
Christopher Lock

Clinical Associate Professor, Neurology & Neurological Sciences

Eligibility


Inclusion Criteria:

   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)
   3.0-6.5, inclusive.

   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).

   7. Women of childbearing potential shall either be surgically sterile, or must agree not
   to become pregnant for the duration of the study. Women must be willing to undergo a
   serum pregnancy test at screening, and at the conclusion of the study. Participants of
   childbearing potential must agree to use a medically approved form of birth control
   (abstinence, intrauterine device (IUD), oral contraception, barrier and spermicide or
   hormonal implant) throughout the duration of the study and for at least 3 months
   following the last transplantation. For those women who are sexually active and using
   oral contraceptives, a second form of barrier contraception is required. Men must be
   willing to consistently use two forms of contraceptive if their partners are of
   childbearing age.

   8. Capable of providing informed consent and willing and able to follow study procedures,
   including willingness to undergo multiple/repeated lumbar puncture.

Exclusion Criteria:

   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, in the opinion of the Investigator and the medical
   monitor, 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
   (Gilenya).

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.

Intervention(s):

biological: NurOwn (MSC-NTF cells)

Recruiting

I'm Interested

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Yamuna Joseph