A Dose-escalation Safety Trial for Intrathecal Autologous Mesenchymal Stem Cell Therapy in Amyotrophic Lateral Sclerosis
The primary objective of this study is to determine the safety of intrathecal delivery of
autologous mesenchymal stem cells (MSCs) to the cerebrospinal fluid (CSF) of patients with
ALS using a dose-escalation study. The trial will include 25 adult, non-ventilator-dependent
patients with clinically definite amyotrophic lateral sclerosis (ALS). Cells will be
isolated from adipose tissue, expanded ex vivo and then, after ~8 weeks, intrathecal (IT)
autologous delivery of MSCs will be performed. There will be 5 treatment groups of up to 5
patients each. Groups 1, 2, and 4 will receive a single dose of cells. Groups 3 and 5 will
receive 2 doses of cells separated by 1 month. Groups will be completed sequentially so that
patients will not be enrolled into the next treatment group until at least 3 patients in the
preceding group have completed the treatment and 1 month of additional observation without
significant toxicity. All patients will be followed on a regular basis until death or for a
minimum of 2 years after completion of the final infusion. Initial clinical follow-up will
be weekly with scheduled blood, CSF and magnetic resonance imaging (MRI) evaluations. After
1 month, patients will have clinical evaluations at 3 month intervals, or earlier if
indicated by clinical status.
Interventional
Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Number of patients with dose-limiting toxicities
2 years after completion of the final infusion
Yes
United States: Food and Drug Administration
11-008415
NCT01609283
May 2012
May 2014
Name | Location |
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Mayo Clinic | Rochester, Minnesota 55905 |