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

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

Quick Info:

Status:
Currently Recruiting
Estimated Enrollment:
60
Phase:
2
Treatment Type:
Stem cells: Autologous Adipose-derived Mesenchymal Stromal Cells
Trial Type:
Single group assignment, open-label
Sponsor:
Mayo Clinic
Primary Investigator:
Contact Information:

Enrollment Criteria:

Forced Ventilation

Breathing Ability

Percent lung function (FVC) or (SVC)
≥ 65%
Months/Onset

Months Since Onset

Number of months since first
symptoms of ALS
No specified
BiPap Allowed

Non-Invasive Ventilation (NIV)

Can PALS use a BiPAP in the trial?
no
DPS Allowed

Diaphragm Pacer (DPS)

Can PALS use a DPS in the trial?
no
Edaravone Usage

Edaravone Usage

Can a PALS use edaravone (Radicut/Radicava)
while enrolled in the trial?
Unknown

Update Notes:

4/11/2019No significant updates
1/31/2018No significant updates
9/20/2017Recruitment status updated
9/1/2017Trial added

Locations:

Mayo Clinic in Rochester, Rochester, 55905

Other Information:

Purpose: 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.
Eligibility:
Details: 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 1 x 10^8 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 will initially be performed at Mayo Clinic in Rochester. Subsequently, the study will expand to the two other Mayo Clinic sites in Arizona and Florida as the manufacturing capabilities are finalized.
Collaborator(s):
News Articles and Summaries:
ALS Forum:
First Published on Clinicaltrials.gov: 8/30/2017
ClinicalTrials.gov ID: NCT03268603
Trial Protocol as Published on Clinicaltrials.gov:
ClinicalTrials.gov