CNS10-NPC-GDNF Delivered to the Motor Cortex for ALS

Launched by CEDARS-SINAI MEDICAL CENTER · Mar 23, 2022

Keywords

ClinConnect Summary

This clinical trial, called CNS10-NPC-GDNF, is exploring a new treatment for Amyotrophic Lateral Sclerosis (ALS), a serious condition that affects the nerves and leads to muscle weakness. The researchers are testing the safety of transplanting special cells, known as neural progenitor cells, into a part of the brain called the motor cortex. These cells have been designed to produce a protein that helps protect and support nerve cells. The main goal of the study is to see if this approach is safe for people with ALS and if it can potentially improve their condition.

To participate in this trial, individuals need to be at least 18 years old and have a confirmed diagnosis of ALS, with symptoms lasting no longer than 36 months. They should also be experiencing progressive weakness in their arms and meet certain health criteria. Participants will need to travel to the study site for necessary visits and will receive care and support throughout the trial. It’s important to note that this study is currently recruiting, and interested individuals should discuss their eligibility with their healthcare provider.

Gender

ALL

Eligibility criteria

• Inclusion:
• • 1. Confirmed diagnosis of ALS (Possible, Lab-supported Probable, Probable or Definite El Escorial Criteria)
• • 2. Duration of ALS symptoms ≤ 36 months
• • 3. Progressive weakness in upper extremities, with EMG supported evidence of denervation in both upper extremities
• • 4. Forced Vital Capacity ≥50% of predicted normal in supine
• • 5. Age: 18 years or older
• • 6. Able to provide Informed Consent
• • 7. Be geographically accessible to the study site and able to travel to study site for required visits
• • 8. Have caregiver to assist in the transportation and care required by participation in the study
• • 9. Not taking riluzole and/or edaravone or on a stable dose for ≥ 30 day
• • 10. For women of child bearing capacity, negative pregnancy test prior to surgery and willingness to use birth control for the duration of the trial.
• • 11. Medically able to undergo craniotomy as determined by the site PI and/or investigators
• • 12. Medically able to tolerate the immunosuppression regimen as determined by the site PI
• Exclusion:
• • 1. Using invasive ventilatory assistance
• • 2. Diagnosis of another active or unstable medical illness that may interfere with study participation at discretion of PI
• 3. Presence of any of the following conditions:
• • 1. Current drug or alcohol abuse
• • 2. Any known immunodeficiency syndrome
• • 3. Unstable medical condition
• • 4. Unstable psychiatric illness including psychosis and untreated major depression within 90 days of screening
• • 4. Persons of child bearing capacity not willing to practice birth control
• • 5. Receiving any investigational device/biologic/drug in the past 30 days or any previous exposure to stem cell therapy
• • 6. Any condition in the upper extremities that precludes serial strength or coordination testing
• • 7. Any condition that the investigators feel may pose complications for the surgery
• • 8. 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
• • 9. Allergy to Beta-Lactam antibiotics
• • 10. Donor Specific Antibodies (DSA) ≥ 2500MFI or CPRA ≥ 20%
• • 11. Contraindications to MRI