NCT03268603

Intrathecal Autologous Adipose-derived Mesenchymal Stromal Cells for Amyotrophic Lateral Sclerosis (ALS)

A Phase II Study of Intrathecal Autologous Adipose-derived Mesenchymal Stromal Cells for Amyotrophic Lateral Sclerosis

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 75 (actual)
• Sponsor: Mayo Clinic · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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.

Conditions

• ALS
• Amyotrophic Lateral Sclerosis

Interventions

Type	Name	Description
DRUG	Autologous Adipose-derived Mesenchymal Stromal Cells	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.

Timeline

• Start date: 2017-10-10
• Primary completion: 2024-01-31
• Completion: 2026-12-31
• First posted: 2017-08-31
• Last updated: 2026-03-09
• Results posted: 2025-04-02

Locations

3 sites across 1 country: United States

Regulatory

• FDA-regulated drug study