Study Summary
This trial is examining the safety of transplanting cells that have been engineered to produce GDNF into the motor cortex of patients with ALS. The cells, called CNS10-NPC-GDNF, are a type of stem cell that can become several different types of cells in the nervous system, including astrocytes. GDNF is a protein that promotes the survival of many types of neural cells. The investigational treatment has been tested in people by delivering it to the spinal cord, but this study will test if it is safe to deliver the cells to the motor cortex of the brain.
- ALS (Amyotrophic Lateral Sclerosis)
Treatment Effectiveness
Effectiveness Progress
Study Objectives
2 Primary · 7 Secondary · Reporting Duration: 9-hole peg testing will be performed 7 times over 15 months
Trial Safety
Safety Progress
Trial Design
3 Treatment Groups
CNS10-NPC-GDNF - Group C
1 of 3
CNS10-NPC-GDNF - Group B
1 of 3
CNS10-NPC-GDNF - Group A
1 of 3
Experimental Treatment
16 Total Participants · 3 Treatment Groups
Primary Treatment: CNS10-NPC-GDNF · No Placebo Group · Phase 1
Trial Logistics
Trial Timeline
Who is running the clinical trial?
Eligibility Criteria
Age 18+ · All Participants · 12 Total Inclusion Criteria
Mark “Yes” if the following statements are true for you:Who else is applying?
What state do they live in?
| Florida | 50.0% |
| Colorado | 50.0% |
How old are they?
| 65+ | 33.3% |
| 18 - 65 | 66.7% |
What site did they apply to?
| Cedars-Sinai Medical Center | 100.0% |
What portion of applicants met pre-screening criteria?
| Met criteria | 66.7% |
| Did not meet criteria | 33.3% |
Frequently Asked Questions
Has the FDA given its stamp of approval to CNS10-NPC-GDNF?
"Due to the limited evidence available, our team assigned a rating of 1 in regards to CNS10-NPC-GDNF's safety. This is based on its standing as an early Phase 1 trial with only small amounts of data collected thus far for efficacy and security." - Anonymous Online Contributor
How many participants are being accepted into this medical research?
"Affirmative, clinicaltrials.gov has information that confirms this medical trial is actively enlisting candidates. This research project was first published on April 1st 2022 and most recently revised on March 23rd 2022. Researchers require 16 patients to be recruited from a single site." - Anonymous Online Contributor
Are investigators actively seeking participants for this experiment?
"As per the details on clinicaltrials.gov, this research initiative is currently enrolling volunteers. The trial was first posted on April 1st 2022 and recently modified on March 23rd of the same year." - Anonymous Online Contributor
What goals are this study attempting to achieve?
"This trial intends to measure safety over a year post-treatment, which will be determined by the incidence of adverse and serious adverse events. Secondary metrics that are being evaluated include pinch strength (difference from baseline), force generation via ATLIS testing (difference from baseline) and hand/wrist strength using a dynamometer (difference from baseline)." - Anonymous Online Contributor