A Phase 1 interventional study of autologous mesenchymal stem cells in Amyotrophic Lateral Sclerosis, sponsored by Mayo Clinic. Completed at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2019-09-03.
Sponsored by Mayo Clinic · Phase 1, Interventional, and Treatment
The purpose of this study is to determine determine the safety of intraspinal delivery of mesenchymal stem cells (MSCs) to the cerebral spinal fluid of patients with Amyotrophic Lateral Sclerosis (ALS) using a dose-escalation study.
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.
Addendum: Five subjects from the Group 5 dosing level will received additional injections of MSCs in an extension of the original study, if tolerated.
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This study's enrollment of 27 is below the median of 35 across 461 interventional studies indexed under Motor Neuron Disease.
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Exclusion Criteria:
Biological: autologous mesenchymal stem cells
There will be five treatment groups of up to five 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 one month. Intrathecal injections into new subjects will be timed so that there is a minimum of one week between subject injections. The cell dose per group is as follows: * Group 1: single intrathecal dose of 1 x 107 cells * Group 2: single intrathecal dose of 5 x 107 cells * Group 3: one intrathecal dose of 5 x 107 cells followed one month later by a second intrathecal dose of 5 x 107 cells * Group 4: single intrathecal dose of 1 x 108 cells * Group 5: one intrathecal dose of 1 x 108 cells followed one month later by a second intrathecal dose of 1 x 108 cells
Number of patients with dose-limiting toxicities
Time frame: baseline -2 years after completion of the final infusion
Number of patients with adverse events
Time frame: baseline -2 years after completion of the final infusion
Change in serum sedimentation rate
Time frame: baseline, 2 years after completion of the final infusion
Change in C-reactive protein levels
Time frame: baseline, 2 years after completion of the final infusion
Change in complete blood counts
Time frame: baseline, 2 years after completion of the final infusion
Change in total nucleated cell count in cerebrospinal fluid (CSF)
Time frame: baseline, 2 years after completion of the final infusion
Change in protein level in cerebrospinal fluid (CSF)
Time frame: baseline, 2 years after completion of the final infusion
Number of patients with presence of cancer cells in their cerebrospinal fluid (CSF)
Time frame: baseline -2 years after completion of the final infusion
Plan to share: No
This study is completed, as verified in Aug 2019. You cannot join it, but the record below documents what was studied.
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