NCT02943850

CNS10-NPC-GDNF for the Treatment of ALS

Completed Phase 1 Last updated 15 July 2020

What this trial tests

Phase 1 trial testing Stem cell (HPC) implantation in Amyotrophic Lateral Sclerosis in 18 participants. Completed in 18 October 2019.

Timeline

1 April 2017

Primary endpoint
18 October 2019

18 October 2019

Quick facts

Lead sponsor	Cedars-Sinai Medical Center
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	18
Start date	1 April 2017
Primary completion	18 October 2019
Estimated completion	18 October 2019
Sites	1 location across United States

Drugs / interventions tested

• Stem cell (HPC) implantation — full drug profile →
• Stereotactic surgical device

Conditions studied

• Amyotrophic Lateral Sclerosis — all drugs for Amyotrophic Lateral Sclerosis →

Sponsor

Cedars-Sinai Medical Center

Who can join

18 and older, any sex, with Amyotrophic Lateral Sclerosis. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Role of Neuroinflammation in Amyotrophic Lateral Sclerosis: Cellular Mechanisms and Therapeutic Implications.
   Liu J, Wang F. · · 2017 · cited 261× · PMID 28871262 · DOI 10.3389/fimmu.2017.01005
2. Protective Functions of Reactive Astrocytes Following Central Nervous System Insult.
   Linnerbauer M, Rothhammer V. · · 2020 · cited 172× · PMID 33117368 · DOI 10.3389/fimmu.2020.573256
3. Gene therapy for ALS: A review.
   Amado DA, Davidson BL. · · 2021 · cited 132× · PMID 33839324 · DOI 10.1016/j.ymthe.2021.04.008
4. Transplantation of human neural progenitor cells secreting GDNF into the spinal cord of patients with ALS: a phase 1/2a trial.
   Baloh RH, Johnson JP, Avalos P, Allred P, et al · · 2022 · cited 108× · PMID 36064599 · DOI 10.1038/s41591-022-01956-3
5. Therapeutic Plasticity of Neural Stem Cells.
   Ottoboni L, von Wunster B, Martino G. · · 2020 · cited 69× · PMID 32265815 · DOI 10.3389/fneur.2020.00148
6. Rising Stars: Astrocytes as a Therapeutic Target for ALS Disease.
   Izrael M, Slutsky SG, Revel M. · · 2020 · cited 58× · PMID 32848579 · DOI 10.3389/fnins.2020.00824
7. Glial Cells-The Strategic Targets in Amyotrophic Lateral Sclerosis Treatment.
   Filipi T, Hermanova Z, Tureckova J, Vanatko O, et al · · 2020 · cited 52× · PMID 31963681 · DOI 10.3390/jcm9010261
8. Stem cell treatments for amyotrophic lateral sclerosis: a critical overview of early phase trials.
   Goutman SA, Savelieff MG, Sakowski SA, Feldman EL. · · 2019 · cited 44× · PMID 31189354 · DOI 10.1080/13543784.2019.1627324