Extracellular Vesicles for the Treatment of Syringomyelia

NCT07295067 | Recruiting Phase 1 DMC

• 1 other identifier: XWhUC-MSCs-EV
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Syringomyelia

Keywords

syringomyelia; spinal cord injury; mesenchymal stromal cell; extracellular vesicle

Outcome Measures

Primary Outcomes (1)

• Number of participants who experienced dose-limiting Toxicities (DLTs)

  DLTs related to hUC-MSC-sEV include adverse events of grade 3 or higher (including significant clinical laboratory findings) that are possibly, likely, or definitely related to the study drug, accompanied by clinical symptoms and requiring medical treatment within 14 days of administration. Adverse events are graded according to the Common Terminology Criteria for Adverse Events Version 5.0 (CTCAE 5.0).

  24 hours, 4±1 Weeks, 8±1 Weeks,12±1 Weeks

Secondary Outcomes (7)

• American Spinal Injury Association(ASIA) Score

  4±1 Weeks, 8±1 Weeks,12±1 Weeks, 12±1 months

• Klekamp and Sammi syringomyelia scale

  4±1 Weeks, 8±1 Weeks,12±1 Weeks, 12±1 months

• Incidence of severe adverse events

  4±1 Weeks, 8±1 Weeks,12±1 Weeks

• Time to event (death, tracheostomy, and permanent assisted mechanical ventilation)

  up to 12 months

• modified Japanese Orthopaedic Association Scores (mJOA)

  4±1 Weeks, 8±1 Weeks,12±1 Weeks, 12±1 months

Other Outcomes (6)

• Change from baseline in the blood and csf markers.

  4±1 Weeks, 8±1 Weeks,12±1 Weeks

• Change from baseline in the neuroimaging indices.

  4±1 Weeks, 8±1 Weeks,12±1 Weeks

• Change from baseline in the Electrophysiological indices.

  4±1 Weeks, 8±1 Weeks,12±1 Weeks

Study Arms (1)

Exosomes group

EXPERIMENTAL

Patients in this arm will receive exosomes derived from human umbilical cord blood mesenchymal stem cells as a Intrathecal injection, administered once a month, for a total of three months.

Drug: Exosomes group

Interventions

Exosomes group DRUG

Exosomes derived from human umbilical cord blood mesenchymal stem cells for Intrathecal injection (administered once a month, for a total of three months, based on the recommended dose during the dose-escalation phase).

Exosomes group

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients whose MR revealed that syrinx have shrunk after surgery for a year but whose clinical spinal cord symptoms have not improved.
• Patients whose MR revealed that syrinx spontaneously resolution under conservative observation, but whose clinical spinal cord symptoms have not improved.

You may not qualify if:

• \. Those with diabetes, heart disease, or impaired liver or kidney function; 2. Those with other diseases such as brainstem tumors and spinal cord tumors; 3. Infected individuals with lumbar puncture sites; 4. Those with previous or detected abnormalities in the heart; 5. Those who have autoimmune diseases and need to be treated with immunosuppressants; 6. Those who are allergic to the research drugs; 7. Have participated in other interventional clinical studies or received other cell therapies (excluding blood transfusion) within the past three months; 8. Those who are unconscious, unable to express subjective discomfort symptoms and unable to cooperate with neurological function tests, those who have been receiving drug treatment for a long time and do not cooperate with the treatment plan; 9. Pregnant women, women who are breastfeeding and those planning to become pregnant; 10. Serological tests (HBsAg, anti-HCV, anti-HIV, TP-Ab) are positive; 11. Other circumstances where the researcher deems the patient unsuitable to participate in this study (including but not limited to not meeting the treatment that benefits the patient the most, poor patient compliance, abnormal laboratory test indicators that cannot be accepted, etc.).
• Rejection Criteria:
• misdiagnosis; use of any medication that may significantly impact the assessment accuracy of hUC-MSC-sEV engraftment; absence of any evaluation outcome at any time point during the follow-up period
• Cessation Criteria:
• individual wishes of the subjects; occurrence of any hUC-MSC-sEV-associated serious adverse event (SAE) that may aggravate neurological dysfunction, or require prolongation of existing hospitalization, or need hospital readmission, or impair consciousness, or be life-threatening, or even lead to death in any subject; detection of any major mistake in the present protocol during the implementation of this clinical trial; the national administration agency requires the clinical trial to be halted

Sponsors & Collaborators

• Xuanwu Hospital, Beijing: lead
• Yisaier Medical Technology (shan xi) Co., Ltd.: collaborator

Study Sites (1)

Xuanwu Hospital

Beijing, Beijing City, 100032, China

RECRUITING

Related Publications (4)

• 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.

  PMID: 40675800 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.

  PMID: 40536443 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.

  PMID: 29784434 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.

  PMID: 38578425 BACKGROUND

MeSH Terms

Conditions

Syringomyelia; Spinal Cord Injuries

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Trauma, Nervous System; Wounds and Injuries

Central Study Contacts

fengzeng jian, md

CONTACT

+861083198899; jianfengzeng@xwh.ccmu.edu.cn

chenghua yuan

CONTACT

+861083198899; yuanchenghua@ccmu.edu.cn

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 19, 2025
• First Posted: December 19, 2025
• Study Start: December 23, 2025
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: February 6, 2026

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)