CNS10-NPC-GDNF for Amyotrophic Lateral Sclerosis

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Cedars-Sinai Medical Center, Los Angeles, CAAmyotrophic Lateral SclerosisCNS10-NPC-GDNF - Biological
Eligibility
18+
All Sexes
What conditions do you have?
Select

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.

Eligible Conditions
  • ALS (Amyotrophic Lateral Sclerosis)

Treatment Effectiveness

Effectiveness Progress

1 of 3

Study Objectives

2 Primary · 7 Secondary · Reporting Duration: 9-hole peg testing will be performed 7 times over 15 months

12 months post-operatively
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
Month 15
Functional Hand assessments using 9-hole peg test
Month 15
Force Generation via Accurate Test of Limb Isometric Strength (ATLIS) testing
Month 15
Compound Motor Action Potential (CMAP)
Month 15
Hand/Wrist Strength
Month 15
Penn Upper Motor Neuron Score (PUMNS)
Month 15
Pinch Strength
Month 15
Hand Knob - Functional MRI (fMRI)

Trial Safety

Safety Progress

1 of 3

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

CNS10-NPC-GDNF - Group C
Biological
Experimental Group · 1 Intervention: CNS10-NPC-GDNF · Intervention Types: Biological
CNS10-NPC-GDNF - Group B
Biological
Experimental Group · 1 Intervention: CNS10-NPC-GDNF · Intervention Types: Biological
CNS10-NPC-GDNF - Group A
Biological
Experimental Group · 1 Intervention: CNS10-NPC-GDNF · Intervention Types: Biological

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: 9-hole peg testing will be performed 7 times over 15 months

Who is running the clinical trial?

California Institute for Regenerative Medicine (CIRM)OTHER
59 Previous Clinical Trials
3,244 Total Patients Enrolled
2 Trials studying Amyotrophic Lateral Sclerosis
281 Patients Enrolled for Amyotrophic Lateral Sclerosis
Cedars-Sinai Medical CenterLead Sponsor
463 Previous Clinical Trials
151,393 Total Patients Enrolled
6 Trials studying Amyotrophic Lateral Sclerosis
1,386 Patients Enrolled for Amyotrophic Lateral Sclerosis
Richard Lewis, MDPrincipal InvestigatorCedars-Sinai Medical Center
1 Previous Clinical Trials
23 Total Patients Enrolled

Eligibility Criteria

Age 18+ · All Participants · 12 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You have experienced symptoms of ALS for no longer than 3 years.
You are not currently taking riluzole and/or edaravone, or you have been taking a stable dose of these medications for at least 30 days.

Who else is applying?

What state do they live in?
Florida50.0%
Colorado50.0%
How old are they?
65+33.3%
18 - 6566.7%
What site did they apply to?
Cedars-Sinai Medical Center100.0%
What portion of applicants met pre-screening criteria?
Met criteria66.7%
Did not meet criteria33.3%

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

Unverified Answer

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

Unverified Answer

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

Unverified Answer

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

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.