NCT02315027

Mesenchymal Stem Cell Therapy in Multiple System Atrophy

Intrathecal Autologous Mesenchymal Stem Cell Therapy in Multiple System Atrophy (MSA) - Effect of Dose and Natural History

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 30 (actual)
• Sponsor: Mayo Clinic · Academic / Other
• Sex: All
• Age: 30 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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.

Conditions

• MSA

Interventions

Type	Name	Description
BIOLOGICAL	Autologous Mesenchymal Stem Cells	single dose of 1 × 10(7) cells intrathecally
BIOLOGICAL	Autologous Mesenchymal Stem Cells	2 doses of 5 × 10(7) cells intrathecally each 1 month (±4 days) apart
BIOLOGICAL	Autologous Mesenchymal Stem Cells	2 doses of 1 × 10(8) cells intrathecally each 1 month apart
BIOLOGICAL	Autologous Mesenchymal Stem Cells	Ten doses of 5 x 10(7) (±20%) cells intrathecally six months (±1 month) apart
BIOLOGICAL	Autologous Mesenchymal Stem Cells	Ten doses of 2.5 x 10(7) (±20%) cells intrathecally six months (±1 month) apart

Timeline

• Start date: 2012-10-01
• Primary completion: 2027-03-01
• Completion: 2027-03-01
• First posted: 2014-12-11
• Last updated: 2026-04-08

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study