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Stem Cell Therapy

CNS10-NPC-GDNF for Amyotrophic Lateral Sclerosis

Phase 1
Recruiting
Led By Richard Lewis, MD
Research Sponsored by Cedars-Sinai Medical Center
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Forced Vital Capacity ≥50% of predicted normal in supine
Age: 18 years or older
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 9-hole peg testing will be performed 7 times over 15 months
Awards & highlights

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.

Who is the study for?
This trial is for adults over 18 with ALS symptoms for less than 3 years, who can consent and have a caregiver. They must be able to breathe well on their own (FVC ≥50%), travel to the site, and not be on certain drugs or have used stem cells before. Women must test negative for pregnancy and use birth control.Check my eligibility
What is being tested?
The safety of CNS10-NPC-GDNF cell transplants into the brain's motor cortex is being tested. These are engineered stem cells that become neural cells producing GDNF, a protein aiding neural cell survival, previously tested in spinal cords but now in human brains.See study design
What are the potential side effects?
Potential side effects aren't specified here but may include typical risks associated with craniotomy surgery, immunosuppression therapy such as infection risk increase, reaction to foreign cells, or neurological changes due to intervention.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My lung function is at least half of what is expected for someone healthy.
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I am 18 years old or older.
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My ALS symptoms started less than 3 years ago.
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I have growing weakness in my arms, confirmed by an EMG test.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~9-hole peg testing will be performed 7 times over 15 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 9-hole peg testing will be performed 7 times over 15 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Safety, as evaluated by changes from baseline in the brain MRI
Safety, as evaluated by the incidence of Adverse Events and Serious Adverse Events and their relationship to the treatment
Secondary outcome measures
Compound Motor Action Potential (CMAP)
Force Generation via Accurate Test of Limb Isometric Strength (ATLIS) testing
Functional Hand assessments using 9-hole peg test
+4 more

Trial Design

3Treatment groups
Experimental Treatment
Group I: CNS10-NPC-GDNF - Group CExperimental Treatment1 Intervention
Unilateral Motor Cortex, 0.5x10^6 cells in 10 µL/site, 21 sites (10.5x10^6 total cells) - Motor cortex corresponding to the dominant hand
Group II: CNS10-NPC-GDNF - Group BExperimental Treatment1 Intervention
Unilateral, Motor Cortex, 0.5x10^6 cells in 10 µL/site, 21 sites (10.5x10^6 total cells) - Motor cortex corresponding to the non-dominant hand
Group III: CNS10-NPC-GDNF - Group AExperimental Treatment1 Intervention
Unilateral, Motor Cortex, 0.25x10^6 cells in 10 µL/site, 21 sites (5.25x10^6 total cells) - Motor cortex corresponding to the non-dominant hand

Find a Location

Who is running the clinical trial?

Cedars-Sinai Medical CenterLead Sponsor
500 Previous Clinical Trials
164,883 Total Patients Enrolled
6 Trials studying Amyotrophic Lateral Sclerosis
1,386 Patients Enrolled for Amyotrophic Lateral Sclerosis
California Institute for Regenerative Medicine (CIRM)OTHER
64 Previous Clinical Trials
3,141 Total Patients Enrolled
2 Trials studying Amyotrophic Lateral Sclerosis
214 Patients Enrolled for Amyotrophic Lateral Sclerosis
Richard Lewis, MDPrincipal InvestigatorCedars-Sinai Medical Center
1 Previous Clinical Trials
23 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

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."

Answered by AI

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."

Answered by AI

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."

Answered by AI

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)."

Answered by AI

Who else is applying?

What state do they live in?
Colorado
Florida
How old are they?
65+
18 - 65
What site did they apply to?
Cedars-Sinai Medical Center
What portion of applicants met pre-screening criteria?
Did not meet criteria
Met criteria
Recent research and studies
clinicaltrials.govStudy
CNS10-NPC-GDNF Delivered to the Motor Cortex for ALS
Clive Svendsen 2022. "CNS10-NPC-GDNF Delivered to the Motor Cortex for ALS". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05306457.
All > Lou Gehrigs Disease > Als
~7 spots leftby Sep 2025
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