Back to resultsMesenchymal Stem Cells for Treatment of Amyotrophic Lateral Sclerosis (ALS)
Primary Purpose
Amyotrophic Lateral Sclerosis
Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
autologous mesenchymal stem cells
Sponsored by
About this trial
This is an interventional treatment trial for Amyotrophic Lateral Sclerosis
Eligibility 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
Sites / Locations
- Mayo Clinic
Outcomes
Primary Outcome Measures
Safety
Clinical monitoring of possible reaction to intrathecal MSC delivery including blood pressure, temperature and pain score.
Secondary Outcome Measures
Neurologic disability score
Quantitative, summated, manual muscle testing
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01142856
Brief Title
Mesenchymal Stem Cells for Treatment of Amyotrophic Lateral Sclerosis (ALS)
Official Title
A Single Patient Treatment Protocol for Autologous Mesenchymal Stem Cell Intraspinal Therapy in Amyotrophic Lateral Sclerosis (ALS)
Study Type
Interventional
2. Study Status
Record Verification Date
May 2012
Overall Recruitment Status
Completed
Study Start Date
June 2010 (undefined)
Primary Completion Date
April 2011 (Actual)
Study Completion Date
April 2011 (Actual)
3. Sponsor/Collaborators
Name of the Sponsor
Mayo Clinic
4. Oversight
5. Study Description
Brief Summary
The purpose of this study is to determine the safety of injecting mesenchymal stem cells through intraspinal delivery for the treatment of ALS.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Amyotrophic Lateral Sclerosis
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
1 (Actual)
8. Arms, Groups, and Interventions
Intervention Type
Biological
Intervention Name(s)
autologous mesenchymal stem cells
Intervention Description
single intrathecal dose of 10x6 cells by lumbar puncture.
Primary Outcome Measure Information:
Title
Safety
Description
Clinical monitoring of possible reaction to intrathecal MSC delivery including blood pressure, temperature and pain score.
Time Frame
2 year follow-up
Secondary Outcome Measure Information:
Title
Neurologic disability score
Description
Quantitative, summated, manual muscle testing
Time Frame
Two year follow-up
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Anthony J. Windebank, MD
Organizational Affiliation
Mayo Clinic
Official's Role
Principal Investigator
Facility Information:
Facility Name
Mayo Clinic
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Mesenchymal Stem Cells for Treatment of Amyotrophic Lateral Sclerosis (ALS)
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