Status:
ACTIVE_NOT_RECRUITING
CNS10-NPC-GDNF Delivered to the Motor Cortex for ALS
Lead Sponsor:
Cedars-Sinai Medical Center
Collaborating Sponsors:
California Institute for Regenerative Medicine (CIRM)
Conditions:
Amyotrophic Lateral Sclerosis
Eligibility:
All Genders
18+ years
Phase:
PHASE1
Brief Summary
The investigator is examining the safety of transplanting cells, that have been engineered to produce a growth factor, into the motor cortex (brain) of patients with Amyotrophic Lateral Sclerosis (ALS...
Detailed Description
This study will be the first to use a genetically modified progenitor cell line delivered to the motor cortex to treat a neurodegenerative disease. This is a Phase 1/2a, single-center, safety study of...
Eligibility Criteria
Inclusion
- Inclusion:
- Confirmed diagnosis of ALS (Possible, Lab-supported Probable, Probable or Definite El Escorial Criteria)
- Duration of ALS symptoms ≤ 36 months
- Progressive weakness in upper extremities, with EMG supported evidence of denervation in both upper extremities
- Forced Vital Capacity ≥50% of predicted normal in supine
- Age: 18 years or older
- Able to provide Informed Consent
- Be geographically accessible to the study site and able to travel to study site for required visits
- Have caregiver to assist in the transportation and care required by participation in the study
- Not taking riluzole and/or edaravone or on a stable dose for ≥ 30 day
- For women of child bearing capacity, negative pregnancy test prior to surgery and willingness to use birth control for the duration of the trial.
- Medically able to undergo craniotomy as determined by the site PI and/or investigators
- Medically able to tolerate the immunosuppression regimen as determined by the site PI
- Exclusion:
- Using invasive ventilatory assistance
- Diagnosis of another active or unstable medical illness that may interfere with study participation at discretion of PI
- Presence of any of the following conditions:
- Current drug or alcohol abuse
- Any known immunodeficiency syndrome
- Unstable medical condition
- Unstable psychiatric illness including psychosis and untreated major depression within 90 days of screening
- Persons of child bearing capacity not willing to practice birth control
- Receiving any investigational device/biologic/drug in the past 30 days or any previous exposure to stem cell therapy
- Any condition in the upper extremities that precludes serial strength or coordination testing
- Any condition that the investigators feel may pose complications for the surgery
- Any condition or ALS disease phenotype that the site PI feels may interfere with participation in the study or in the interpretation of study endpoints
- Allergy to Beta-Lactam antibiotics
- Donor Specific Antibodies (DSA) ≥ 2500MFI or CPRA ≥ 20%
- Contraindications to MRI
Exclusion
Key Trial Info
Start Date :
May 8 2022
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
October 1 2026
Estimated Enrollment :
16 Patients enrolled
Trial Details
Trial ID
NCT05306457
Start Date
May 8 2022
End Date
October 1 2026
Last Update
August 19 2025
Active Locations (1)
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1
Cedars-Sinai Medical Center
Los Angeles, California, United States, 90048