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

Study Purpose:

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

Study Status:

Not recruiting

Disease:

Amyotrophic Lateral Sclerosis

Study Type:

Interventional

Type of Intervention:

Biological

Intervention Name:

autologous mesenchymal stem cells

Placebo:

Phase:

Phase 1

Study Chair(s)/Principal Investigator(s):

Anthony J. Windebank, MD, Mayo Clinic

Clinicaltrials.gov ID:

NCT01142856

Neals Affiliated?

No

Coordinating Center Contact Information

Mayo Clinic

Rochester, Minnesota, 55905 United States

Full Study Summary:

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 Sponsor:

Mayo Clinic

Participant Duration:

Two years

Estimated Enrollment:

1

Estimated Study Start Date:

11 / 02 / 2016

Estimated Study Completion Date:

04 / 01 / 2011

Posting Last Modified Date:

05 / 03 / 2012

Date Study Added to neals.org:

06 / 11 / 2010

Minimum Age:

18 Years

Maximum Age:

N/A

Can participants use Riluzole?

Yes

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

Mayo Clinic

Rochester, Minnesota 55905
United States