The purpose of this study is to determine the safety and efficacy of intrathecal treatment delivered to the cerebrospinal fluid (CSF) of mesenchymal stem cells in ALS patients every 3 months for a total of 4 injections over 12 months. Mesenchymal stem cells (MSCs) are a type of stem cell that can be grown into a number of different kinds of cells. In this study, MSCs will be taken from the subject's body fat and grown. CSF is the fluid surrounding the spine. The use of mesenchymal stem cells is considered investigational, which means it has not been approved by the Food and Drug Administration (FDA) for routine clinical use. However, the FDA has allowed the use of mesenchymal stem cells in this research study.
The Goal of the Proposed Study is to perform an open label, 60 subject, Phase II multi-site clinical trial to investigate the safety and efficacy of intrathecal treatment of aaMSCs in ALS. Patients will be treated with 10-100 million aaMSCs every 3 months for a total of 4 intrathecal injections over 12 months. Reduced dose treatments will be allowed based on specific adverse events. Multiple biomarkers will be tracked throughout the clinical trial and correlated with response to treatment. This study was initially performed at Mayo Clinic in Rochester and subsequently expanded to the two other Mayo Clinic sites in Arizona and Florida. All biopsies and stem cell injections take place at Mayo Clinic Rochester, regardless of where the subject initially enrolls into the study.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
75
The investigational product consists of autologous adipose-derived Mesenchymal Stromal Cells (MSCs), suspended in 5-10 mL Lactated Ringer's. The MSC are provided in a sterile syringe labelled with appropriate patient and product identifiers ready for intrathecal injection.
Mayo Clinic
Scottsdale, Arizona, United States
Mayo Clinic
Jacksonville, Florida, United States
Mayo Clinic in Rochester
Rochester, Minnesota, United States
Adverse Events
The number of adverse events experienced by subjects
Time frame: 12-months
Unexpected Severe Adverse Events
The number of unexpected severe adverse events experienced by subjects
Time frame: 12-months
Change in Slope of ALS Functional Rating Scale - Revised (ALSFRS-R)
The ALSFRS-R includes 12 questions. Each task is rated on a five-point scale from 0 = can't do, to 4 = normal ability. Individual item scores are summed to produce a reported score of between 0=worst and 48=best. The change in slope is the rate at which a patient's functional ability declines over time, as measured by the change in the ALFSFRS score over 1 year. A steeper slope signifies a faster decline in function. A negative change in slope indicates a decline in function and positive change in slope indicates an improvement in function.
Time frame: baseline, approximately 1 year
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