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

A Single Patient Treatment Protocol for Autologous Mesenchymal Stem Cell Intraspinal Therapy in Amyotrophic Lateral Sclerosis (ALS)

The purpose of this study is to determine the safety of injecting mesenchymal stem cells through intraspinal delivery for the treatment of ALS.

Study Overview

• Status: Completed
• Conditions: Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Biological: autologous mesenchymal stem cells

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.

• Study Type: Interventional
• Enrollment (Actual): 1
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Mayo Clinic

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety	Clinical monitoring of possible reaction to intrathecal MSC delivery including blood pressure, temperature and pain score.	2 year follow-up

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Neurologic disability score	Quantitative, summated, manual muscle testing	Two year follow-up

Collaborators and Investigators

• Sponsor: Mayo Clinic

Study record dates

Study Major Dates

• Study Start: June 1, 2010
• Primary Completion (ACTUAL): April 1, 2011
• Study Completion (ACTUAL): April 1, 2011

Study Registration Dates

• First Submitted: June 10, 2010
• First Submitted That Met QC Criteria: June 10, 2010
• First Posted (ESTIMATE): June 11, 2010

Study Record Updates

• Last Update Posted (ESTIMATE): May 3, 2012
• Last Update Submitted That Met QC Criteria: May 2, 2012
• Last Verified: May 1, 2012

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Metabolic Diseases; Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Sclerosis; Motor Neuron Disease; Amyotrophic Lateral Sclerosis
• Other Study ID Numbers: 09-001995