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

Amyotrophic Lateral Sclerosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01142856
• Other study ID #: 09-001995
• Status: Completed
• Phase: Phase 1
• First received: June 10, 2010
• Last updated: May 2, 2012
• Start date: June 2010
• Est. completion date: April 2011

Study information

• Verified date: May 2012
• Source: Mayo Clinic
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 1
• Est. completion date: April 2011
• Est. primary completion date: April 2011
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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 Design

Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• Sclerosis

Intervention

Biological:
• autologous mesenchymal stem cells
single intrathecal dose of 10x6 cells by lumbar puncture.

Locations

Country	Name	City	State
United States	Mayo Clinic	Rochester	Minnesota

Sponsors (1)

Lead Sponsor	Collaborator
Mayo Clinic

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety	Clinical monitoring of possible reaction to intrathecal MSC delivery including blood pressure, temperature and pain score.	2 year follow-up	Yes
Secondary	Neurologic disability score	Quantitative, summated, manual muscle testing	Two year follow-up	No