Mesenchymal Stem Cells for Treatment of Amyotrophic Lateral Sclerosis (ALS) - Full Text View

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:	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:

Genetics Home Reference related topics: amyotrophic lateral sclerosis

MedlinePlus related topics: Amyotrophic Lateral Sclerosis

Genetic and Rare Diseases Information Center resources: Amyotrophic Lateral Sclerosis

U.S. FDA Resources

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:

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 (Adult, Senior)
Sexes Eligible for Study:	All
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

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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


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

Additional relevant MeSH terms:


Sclerosis 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

ClinicalTrials.gov processed this record on June 27, 2017