Mesenchymal Stem Cell Therapy in Multiple System Atrophy

Phase 1

Active, not recruiting

• Conditions: MSA
• Interventions: Biological: Autologous Mesenchymal Stem Cells

• Registration Number: NCT02315027
• Lead Sponsor: Mayo Clinic

Brief 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.

Study Timeline

• Study Start: 2012-10
• Study First Submitted: 2014-10-31
• Study First Submitted QC: 2014-12-09
• Study First Posted: 2014-12-11
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-01
• Last Update Posted: 2025-04-03
• Primary Completion: 2026-03
• Study Completion: 2026-03

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
10 doses of 5 x 10(7) (±20%) MSCs	Autologous Mesenchymal Stem Cells	Group 4: Participants will receive up to 10 doses of 5 x 10(7) (±20%) mesenchymal stem cells (MSCs) approximately 6 months apart.
2 doses of 1 × 10(8) MSCs	Autologous Mesenchymal Stem Cells	Group 3: Participants will receive one intrathecal dose of 1 × 10(8) mesenchymal stem cells (MSCs), followed by a second intrathecal dose of 1 × 10(8) MSCs approximately one month later
1 dose of 1 × 10(7) MSCs	Autologous Mesenchymal Stem Cells	Group 1: Participants will receive a single intrathecal dose of 1 × 10(7) mesenchymal stem cells (MSCs)
10 doses of 2.5 x 10(7) (±20%) MSCs	Autologous Mesenchymal Stem Cells	Group 5: Participants will receive up to 10 doses of 2.5 x 10(7) (±20%) mesenchymal stem cells (MSCs) approximately 6 months apart.
2 doses of 5 × 10(7) MSCs	Autologous Mesenchymal Stem Cells	Group 2: Participants will receive one intrathecal dose of 5 × 10(7) mesenchymal stem cells (MSCs), followed by a second intrathecal dose of 5 × 10(7) MSCs approximately one month later

Primary Outcome Measures

Name	Time	Method
Adverse event frequency (by severity, type, attribution, and intervention dose).	14 months

Secondary Outcome Measures

Name	Time	Method
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).	12 months
Rate of change from baseline to 12 months (or last available date) in UMSARS II score.	12 months
Change in CASS score and thermoregulatory sweat test (TST) % from baseline to 12 months.	12 months
Change in CSF biomarkers from baseline to 2 months.	2 months
Rate of change in COMPASS-select score from baseline to 12 months.	12 months
Rate of change from baseline to 12 months (or last available date) in UMSARS total score.	12 months
MRI morphometric changes using dedicated algorithms to evaluate rate of atrophy of defined areas of brain from baseline to 12 months.	12 months

Trial Locations

Locations (1)

Mayo Clinic in Rochester; 🇺🇸 Rochester, Minnesota, United States