The purpose of this study is to determine whether mesenchymal stem cells (MSCs) can be safely delivered to the cerebrospinal fluid (CSF) of patients with multiple system atrophy (MSA). Funding Source - FDA OOPD.
The primary aim is to evaluate the safety and tolerability of intrathecal injection of autologous MSCs in a dose escalation study in patients with MSA. Safety secondary goals include to monitor changes in peripheral blood and in components of CSF, and monitor for any changes of nervous system structures using MRI. Efficacy secondary goals include evaluating potential efficacy by providing a number of studies and instruments that will detect changes in the course of the disease in terms of autonomic and neurologic symptoms and deficits.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
30
single dose of 1 × 10(7) cells intrathecally
2 doses of 5 × 10(7) cells intrathecally each 1 month (±4 days) apart
2 doses of 1 × 10(8) cells intrathecally each 1 month apart
Mayo Clinic in Rochester
Rochester, Minnesota, United States
Adverse event frequency (by severity, type, attribution, and intervention dose).
Time frame: 14 months
Rate of change of Unified Multiple System Atrophy Rating Scale (UMSARS) I score from baseline to 12 months (or last available date), compared with placebo limb of Rifampicin trial (historical control cohort).
Time frame: 12 months
Rate of change from baseline to 12 months (or last available date) in UMSARS II score.
Time frame: 12 months
Rate of change from baseline to 12 months (or last available date) in UMSARS total score.
Time frame: 12 months
Rate of change in COMPASS-select score from baseline to 12 months.
Time frame: 12 months
Change in CASS score and thermoregulatory sweat test (TST) % from baseline to 12 months.
Time frame: 12 months
MRI morphometric changes using dedicated algorithms to evaluate rate of atrophy of defined areas of brain from baseline to 12 months.
Time frame: 12 months
Change in CSF biomarkers from baseline to 2 months.
Time frame: 2 months
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Ten doses of 5 x 10(7) (±20%) cells intrathecally six months (±1 month) apart
Ten doses of 2.5 x 10(7) (±20%) cells intrathecally six months (±1 month) apart