Extracellular Vesicles for the Treatment of Syringomyelia

Summary

This is a open-label, single-arm, dose escalation phase I clinical trial. The goal of this clinical trial is to evaluate the safety and preliminary efficacy of Intrathecal injection human umbilical cord-derived mesenchymal stromal cell-derived extracellular vesicle (hUC-MSC-sEV) in syringomyelia.

Timeline

Start

2025-12-23

Primary completion

2026-05-07

Completion

2026-12-31

Publications

• Background Yuan C, Du Y, Yao Q, Zhang C, Zhang L, Liu Z, Wang K, Duan W, Wang Z, Wang X, Zeng G, Wu H, Chen Z, Heiss JD, Guan J, Jian FZ. Natural history of Chiari I malformation-syringomyelia: longitudinal cohort study. J Neurol Neurosurg Psychiatry. 2025 Nov 13;96(12):1204-1214. doi: 10.1136/jnnp-2025-336023.
• Background Krause M, Gburek-Augustat J, Grafe D, Metzger R, Ginzel M, Griessenauer CJ, Grassner L, Weghuber D, Gradl J, Auer D, Schally T, Rund S, Kals C, Folie C, Bayer E, Gimona M, Rohde E. First-In-Human Application of Human Umbilical Cord-Derived Extracellular Vesicles in Tethered Spinal Cord Release Surgery. J Extracell Vesicles. 2025 Jun;14(6):e70104. doi: 10.1002/jev2.70104.
• Background Vaquero J, Zurita M, Rico MA, Aguayo C, Fernandez C, Rodriguez-Boto G, Marin E, Tapiador N, Sevilla M, Carballido J, Vazquez D, Garcia-Olmo D, Guadalajara H, Leon M, Valverde I; Neurological Cell Therapy Group From Puerta De Hierro-Majadahonda Hospital. Cell therapy with autologous mesenchymal stromal cells in post-traumatic syringomyelia. Cytotherapy. 2018 Jun;20(6):796-805. doi: 10.1016/j.jcyt.2018.04.006. Epub 2018 May 18.
• Background Li M, Wang X, Qi B, Cui S, Zheng T, Guan Y, Ma L, Liu S, Li Q, Chen Z, Jian F. Treatment of Syringomyelia Characterized by Focal Dilatation of the Central Canal Using Mesenchymal Stem Cells and Neural Stem Cells. Tissue Eng Regen Med. 2024 Jun;21(4):625-639. doi: 10.1007/s13770-024-00637-1. Epub 2024 Apr 5.

Drugs

Indications