Intermediate-size Patient Population Expanded Access Protocol: Repeated Administration of Nurown® (Autologous Mesenchymal Stem Cells Secreting Neurotrophic Factors) for the Treatment of Amyotrophic Lateral Sclerosis (ALS)

BCT-003-US

Expanded Access for treatment with investigational product MSC-NTF cells(NurOwn®) for participants who completed all scheduled treatments and follow-up assessments in the BCT-002-US study

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Inclusion Criteria: 1. Participation in the BCT-002-US study, successful completion of all scheduled treatments and follow-up assessments. 2 Able to provide to the Investigator written informed consent regarding the investigational drug, or, as applicable, on whose behalf a legally authorized representative of the participant has provided such consent. 3. Able to safely undergo all study procedures. Exclusion Criteria: 1. Inability to lie flat for the duration of intrathecal cell transplantation and/or bone marrow biopsy, or inability to tolerate treatment procedures for any other reason (lying flat with BiPAP or NIV are not exclusionary). 2. History of clinically significant autoimmune disease (excluding thyroid disease) myelodysplastic or myeloproliferative disorder, leukemia or lymphoma, whole body irradiation, hip fracture, or severe scoliosis. 3. Any unstable clinically significant medical condition other than ALS (e.g., within six months of baseline, 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 the participant. 4. Any history of malignancy within the previous 5 years, with the exception of non- melanoma localized skin cancers (with no evidence of metastasis, significant invasion, or re-occurrence within three years of baseline). 5. Current use of immunosuppressant medication or use of such medication within 4 weeks of the pre-treatment visit. 6. Any history of acquired or inherited immune deficiency syndrome. 7. Tracheostomy and/or mechanical ventilation. Feeding tube use, BiPAP or NIV are not exclusionary. 8. Pregnant women or women currently breastfeeding or unwilling to use effective birth control methods during the treatment (if of childbearing age). 9. Positive test result for Hepatitis B virus (HBV; surface antigen (HBsAg) and IgM antibodies to core antigen (IgM anti-HBc)), Hepatitis C virus (HCV), Human Immune deficiency virus (HIV) 1 and 2.

{'studyType': 'EXPANDED_ACCESS', 'expandedAccessTypes': {'intermediate': True, 'treatment': True}}

2024-04-23