Amyotrophic Lateral Sclerosis
Conditions
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.
Interventions
BIOLOGICALautologous mesenchymal stem cells
single intrathecal dose of 10x6 cells by lumbar puncture.
Sponsors
Mayo Clinic
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety | 2 year follow-up | Clinical monitoring of possible reaction to intrathecal MSC delivery including blood pressure, temperature and pain score. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Neurologic disability score | Two year follow-up | Quantitative, summated, manual muscle testing |
Countries
United States
Outcome results
None listed