Intrathecal Autologous Adipose-derived Mesenchymal Stromal Cells for Amyotrophic Lateral Sclerosis (ALS)
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|ClinicalTrials.gov Identifier: NCT03268603|
Recruitment Status : Recruiting
First Posted : August 31, 2017
Last Update Posted : April 11, 2019
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.
|Condition or disease||Intervention/treatment||Phase|
|ALS Amyotrophic Lateral Sclerosis||Drug: Autologous Adipose-derived Mesenchymal Stromal Cells||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||60 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase II Study of Intrathecal Autologous Adipose-derived Mesenchymal Stromal Cells for Amyotrophic Lateral Sclerosis|
|Actual Study Start Date :||October 10, 2017|
|Estimated Primary Completion Date :||December 2019|
|Estimated Study Completion Date :||December 2019|
Experimental: Mesenchymal Stromal Cells
Autologous Adipose-derived Mesenchymal Stromal Cells (aaMSCs) will be administered intrathecally at a single dose in a volume of 5-10 mL, all patients will receive 1 x 10^8 intrathecal aaMSCs at the first injection (Visit 4). Subsequent doses may be reduced to 5 x 10^7, based on Dose Modification Rules. One further dose reduction to 1 x 10^7 may occur in later injections.
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.
- Number of Adverse Events [ Time Frame: approximately 2 years ]Number of adverse events will be recorded from the time of enrollment until the end of the follow-up period or, in the case of early withdrawal, to the time of study withdrawal.
- Change in slope of ALS Functional Rating Scale - Revised (ALSFRS-R) [ Time Frame: baseline, approximately 1 year ]The ALSFRS-R includes 12 questions. Each task is rated on a five-point scale from 0 = can't do, to 4 = normal ability. Individual item scores are summed to produce a reported score of between 0=worst and 48=best.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03268603
|Contact: Michelle M Turner||507-284-2676||RSTALSRESEARCH@mayo.edu|
|Contact: Carol Denny||507-284-2676||RSTALSRESEARCH@mayo.edu|
|Principal Investigator:||Nathan P Staff, MD, PhD||Mayo Clinic|
|Principal Investigator:||Anthony J Windebank, MD||Mayo Clinic|