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

This study has been completed.
Sponsor:
Information provided by:
Mayo Clinic
ClinicalTrials.gov Identifier:
NCT01142856
First received: June 10, 2010
Last updated: May 2, 2012
Last verified: May 2012
History of Changes
  Purpose

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: Endpoint Classification: Safety Study
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:

U.S. FDA Resources

Further study details as provided by Mayo Clinic:

Primary Outcome Measures:
  • Safety [ Time Frame: 2 year follow-up ] [ Designated as safety issue: Yes ]
    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 ] [ Designated as safety issue: No ]
    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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01142856

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

No publications provided

Responsible Party: Anthony J. Windebank, M.D., Mayo Clinic
ClinicalTrials.gov Identifier: NCT01142856     History of Changes
Other Study ID Numbers: 09-001995
Study First Received: June 10, 2010
Last Updated: May 2, 2012
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Amyotrophic Lateral Sclerosis
Sclerosis
Motor Neuron Disease
Spinal Cord Diseases
Central Nervous System Diseases
Nervous System Diseases
 Neurodegenerative Diseases
TDP-43 Proteinopathies
Neuromuscular Diseases
Proteostasis Deficiencies
Metabolic Diseases
Pathologic Processes

ClinicalTrials.gov processed this record on May 21, 2013