Intrathecal Administration of DUOC-01 in Adults With Primary Progressive Multiple Sclerosis

Summary

This study is a prospective Phase 1a open-label single- center trial. It will assess the safety of intrathecal administration of DUOC-01 cells to adults with Primary Progressive Multiple Sclerosis (PPMS). DUOC-01 is a population of cells expanded from donated human umbilical cord blood cells and is intended for treatment of neurodegenerative and demyelinating diseases. There will be approximately 20 participants enrolled. Exploratory objectives include changes in MS assessment scores, changes in brain MRI findings, and changes in blood biomarkers.

Timeline

Start

2021-01-24

Primary completion

2025-09-30

Completion

2025-09-30

Publications

• Background Kurtzberg J, Buntz S, Gentry T, Noeldner P, Ozamiz A, Rusche B, Storms RW, Wollish A, Wenger DA, Balber AE. Reprint of: Preclinical characterization of DUOC-01, a cell therapy product derived from banked umbilical cord blood for use as an adjuvant to umbilical cord blood transplantation for treatment of inherited metabolic diseases. Cytotherapy. 2015 Sep;17(9):1314-26. doi: 10.1016/j.jcyt.2015.07.014.
• Background Saha A, Buntz S, Scotland P, Xu L, Noeldner P, Patel S, Wollish A, Gunaratne A, Gentry T, Troy J, Matsushima GK, Kurtzberg J, Balber AE. A cord blood monocyte-derived cell therapy product accelerates brain remyelination. JCI Insight. 2016 Aug 18;1(13):e86667. doi: 10.1172/jci.insight.86667.
• Background Tracy ET, Zhang CY, Gentry T, Shoulars KW, Kurtzberg J. Isolation and expansion of oligodendrocyte progenitor cells from cryopreserved human umbilical cord blood. Cytotherapy. 2011 Jul;13(6):722-9. doi: 10.3109/14653249.2011.553592. Epub 2011 Feb 22.
• Background Tracy E, Aldrink J, Panosian J, Beam D, Thacker J, Reese M, Kurtzberg J. Isolation of oligodendrocyte-like cells from human umbilical cord blood. Cytotherapy. 2008;10(5):518-25. doi: 10.1080/14653240802154586.
• Background Scotland P, Buntz S, Noeldner P, Saha A, Gentry T, Kurtzberg J, Balber AE. Gene products promoting remyelination are up-regulated in a cell therapy product manufactured from banked human cord blood. Cytotherapy. 2017 Jun;19(6):771-782. doi: 10.1016/j.jcyt.2017.03.004. Epub 2017 Apr 5.
• Background Kurtzberg J, Buntz S, Gentry T, Noeldner P, Ozamiz A, Rusche B, Storms RW, Wollish A, Wenger DA, Balber AE. Preclinical characterization of DUOC-01, a cell therapy product derived from banked umbilical cord blood for use as an adjuvant to umbilical cord blood transplantation for treatment of inherited metabolic diseases. Cytotherapy. 2015 Jun;17(6):803-815. doi: 10.1016/j.jcyt.2015.02.006. Epub 2015 Mar 12.

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Indications