CNS10-NPC-GDNF for the Treatment of ALS

Completed · Phase 1

Conditions studied: Amyotrophic Lateral Sclerosis

In brief

The investigator is examining the safety of transplanting cells that have been engineered to produce a growth factor into the spinal cord of patients with Amyotrophic Lateral Sclerosis (ALS). The cells are called neural progenitor cells, which are a type of stem cell that can become several different types of cells in the nervous system. These cells have been derived to specifically become astrocytes, which is a type of neuronal cell. The growth factor is called glial cell line-derived neurotrophic factor, or GDNF. GDNF is a protein that promotes the survival of many types of neuronal cells. Therefore, the cells are called "CNS10-NPC-GDNF." The investigational treatment has been tested in animals, but it has not yet been tested in people. In this study, we want to learn if CNS10-NPC-GDNF cells are safe to transplant into the spinal cords of people.

Key facts

Study ID

NCT02943850

Run by

Cedars-Sinai Medical Center

People needed

18

Starts

2017-04-01

Expected to finish

2019-10-18

Last updated by the study team

2020-07-15

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Confirmed diagnosis of ALS (Lab-supported Probable, Probable or Definite EI Escorial Criteria)
• Duration of symptoms ≤ 36 months
• Progressive weakness in lower extremities, with EMG supported evidence of denervation in both lower extremities.
• Forced Vital Capacity >60% of predicted normal in supine.
• Male/Female; Age: 18 and 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 or on a stable dose for ≥ 30 days
• For women of child bearing capacity, negative pregnancy test prior to surgery
• Medically able to undergo thoracolumbar laminectomy or laminoplasty as determined by the site PI and Neurosurgeon
• Medically able to tolerate the immunosuppression regimen as determined by the site PI

You may not qualify if…

• 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 past 30 days or any previous exposure to stem cell therapy
• Any condition in the lower extremities which precludes serial strength testing
• Any condition that the Neurosurgeon feels 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

Where it is running

• Cedars-Sinai Medical Center — Los Angeles, California, United States

Full record on ClinicalTrials.gov

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