Safety and Efficacy of Repeated Administrations of NurOwn® in ALS Patients

Overview

About this study

This study will evaluate the safety and efficacy of repeated administration of NurOwn® (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

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:

 

  • Males and females ages 18 to 60 years old, inclusive, at the Screening Visit.
  • 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.
  • Decline in ALSFRS-R total score of 3 or more points in the three months before randomization.
  • Taking a stable dose of riluzole, or no riluzole at all (riluzole-naïve participants are permitted in the study), for at least 30 days prior to the Screening Visit and be willing to maintain the riluzole dose for the duration of the study.

Exclusion Criteria:

 

  • Prior stem cell therapy of any kind.
  • Active participation in any other ALS interventional study.
  • 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.
  • History of autoimmune disease that may confound study results 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 participants.

 

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

Rochester, Minn.

Mayo Clinic principal investigator

Anthony Windebank, M.D.

Closed for enrollment

More information

Publications

Publications are currently not available

Study Results Summary

Not yet available

Supplemental Study Information

Not yet available

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CLS-20398307

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