A Compassionate Use Study to Administer NurOwn® (Autologous Mesenchymal Stem Cells Secreting Neurotrophic Factors) to Treat Amyotrophic Lateral Sclerosis (ALS)

Overview

About this study

The purpose of this study is to provide up to three intrathecal injections of NurOwn® (MSC-NTF cells, autologous Mesenchymal Stem Cells [MSC] Secreting Neurotrophic Factors [NTF]) given two months apart to patients who have completed all scheduled treatments and follow-up assessments in the BCT-002-US clinical trial. A bone marrow aspiration will be required as part of this program. It will take about 30 weeks for participants to complete the program.

 

 

 

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria:

  • Participation in the BCT-002-US study, completed all study visits and have received three IT treatments.
  • Able to provide to the Investigator written informed consent regarding the eligible investigational drug, or, as applicable, on whose behalf a legally authorized representative of the participant has provided such consent.
  • Able to safely undergo a bone marrow aspiration.
  • Feeding tube use is permitted.

Exclusion Criteria:

  • Inability to lie flat for the duration of intrathecal cell transplantation and/or bone marrow aspiration, or inability to tolerate treatment procedures for any other reason (lying flat with BiPAP or NIV is not exclusionary).
  • History of clinically significant autoimmune disease (excluding thyroid disease) myelodysplastic or myeloproliferative disorder, leukemia or lymphoma, whole body irradiation, hip fracture, or severe scoliosis.
  • 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.
  • Any history of malignancy within the previous 5 years, with the exception of nonmelanoma localized skin cancers (with no evidence of metastasis, significant invasion, or re-occurrence within three years of baseline).
  • Current use of immunosuppressant medication or use of such medication within 4 weeks of the screening visit.
  • Any history of acquired or inherited immune deficiency syndrome.
  • Tracheostomy and/or mechanical ventilation (BiPAP or NIV are not exclisonary).
  • Pregnant women or women currently breastfeeding or unwilling to use effective birth control methods during the treatment (if of childbearing age).
  • 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.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Anthony Windebank, M.D.

Closed for enrollment

Contact information:

Carol Denny

(507) 284-5476

Denny.Carol@mayo.edu

More information

Publications

Publications are currently not available
.
CLS-20508604

Mayo Clinic Footer