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Mesenchymal Stem Cells for Treatment of Amyotrophic Lateral Sclerosis (ALS)

This study has been completed.
Information provided by:
Mayo Clinic Identifier:
First received: June 10, 2010
Last updated: May 2, 2012
Last verified: May 2012
The purpose of this study is to determine the safety of injecting mesenchymal stem cells through intraspinal delivery for the treatment of ALS.

Condition Intervention Phase
Amyotrophic Lateral Sclerosis
Biological: autologous mesenchymal stem cells
Phase 1

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Single Patient Treatment Protocol for Autologous Mesenchymal Stem Cell Intraspinal Therapy in Amyotrophic Lateral Sclerosis (ALS)

Resource links provided by NLM:

Further study details as provided by Mayo Clinic:

Primary Outcome Measures:
  • Safety [ Time Frame: 2 year follow-up ]
    Clinical monitoring of possible reaction to intrathecal MSC delivery including blood pressure, temperature and pain score.

Secondary Outcome Measures:
  • Neurologic disability score [ Time Frame: Two year follow-up ]
    Quantitative, summated, manual muscle testing

Enrollment: 1
Study Start Date: June 2010
Study Completion Date: April 2011
Primary Completion Date: April 2011 (Final data collection date for primary outcome measure)
Intervention Details:
    Biological: autologous mesenchymal stem cells
    single intrathecal dose of 10x6 cells by lumbar puncture.
Detailed Description:
A single patient safety study for harvesting, expanding ex vivo and injecting autologous mesenchymal stem cells (MSC's) into the subarachnoid space of a patient with amyotrophic lateral sclerosis (ALS). Cells will be isolated from adipose tissue by subcutaneous biopsy and expanded using an FDA-approved protocol. They will then be injected by lumbar puncture into the cerebrospinal fluid. Injection will be completed in the in- patient clinical research unit (CRU). the patient will be followed for two years.

Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • age greater than 18 years, if female, must be menopausal or had hysterectomy
  • resident and citizen of the United States
  • history of a chronic onset of a progressive motor weakness
  • able to comply with protocol requirements
  • can provide written consent

Exclusion Criteria:

  • does not have renal disease (Creatine > 2.0)
  • does not have active systemic disease
  • does not have any clinically significant abnormalities on prestudy laboratory evaluation
  • does not have any clinically significant medical condition (e.g.,within 6 months of baseline, had a myocardial infarct, angina pectoris, and/or congestive heart failure), that in the opinion of the investigator, would compromise the safety of the patient
  • does not have a history of cancer including melanoma with the exception of localized skin cancers (with no evidence of metastasis, significant invasion,or re-occurrence within 3 years of baseline).
  • has not used an investigational drug within 30 days of baseline visit
  • does not have a tracheostomy
  • does not have a Beck's Depression Inventory score >16
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Please refer to this study by its identifier: NCT01142856

United States, Minnesota
Mayo Clinic
Rochester, Minnesota, United States, 55905
Sponsors and Collaborators
Mayo Clinic
Principal Investigator: Anthony J. Windebank, MD Mayo Clinic
  More Information

Responsible Party: Anthony J. Windebank, M.D., Mayo Clinic Identifier: NCT01142856     History of Changes
Other Study ID Numbers: 09-001995
Study First Received: June 10, 2010
Last Updated: May 2, 2012

Additional relevant MeSH terms:
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases processed this record on April 24, 2017