Extracellular Vesicles for the Treatment of Syringomyelia

NCT ID: NCT07295067

Last Updated: 2025-12-19

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE1
• Total Enrollment: 18 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-12-15
• Study Completion Date: 2026-12-31

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.

Detailed Description

This is a open-label, single-arm, dose escalation phase I clinical trial. The study will consist of one part: Part 1 will be a dose-escalation study.

A traditional 3+3 dose-escalation design will be implemented in Part 1. Cohort 1 will receive low-dose1×1011particles; Cohort 2 will receive middle-dose2×1011particles; and Cohort 3 will receive high-dose3×1011particles. (Cohort 1 to Cohort 3 will receive a dose of 2 ml per lumbar puncture, administered once a month, for a total of three months.) If no dose-limiting toxicities (DLTs) are observed for 2 weeks after the administration of the first Intrathecal injection, a new cohort will be enrolled at the next planned dose level. If DLTs are observed in one participant in the cohort, an additional three participants will be treated at the same dose level. Dose escalation will be stopped if DLTs are observed in more than 33% of the participants.

Conditions

Syringomyelia

Keywords

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

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Exosomes group

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.

Group Type: EXPERIMENTAL

Exosomes group

Intervention Type: 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).

Interventions

Exosomes group

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).

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• 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.
• Age: 18-60 years, inclusion of both genders;

Exclusion Criteria

• 1. 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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 60 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Yisaier Medical Technology (shan xi) Co., Ltd.

UNKNOWN

Sponsor Role: collaborator

Xuanwu Hospital, Beijing

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Xuanwu Hospital

Beijing, Beijing City, China

Site Status: RECRUITING

Countries

China

Central Contacts

fengzeng jian, md

Role: CONTACT

Phone: +861083198899

Email: jianfengzeng@xwh.ccmu.edu.cn

chenghua yuan

Role: CONTACT

Phone: +861083198899

Email: yuanchenghua@ccmu.edu.cn

Facility Contacts

fengzeng jian

Role: primary

References

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.

Reference Type: BACKGROUND

PMID: 40675800 (View on PubMed)

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.

Reference Type: BACKGROUND

PMID: 40536443 (View on PubMed)

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.

Reference Type: BACKGROUND

PMID: 29784434 (View on PubMed)

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.

Reference Type: BACKGROUND

PMID: 38578425 (View on PubMed)

Other Identifiers

XWhUC-MSCs-EV

Identifier Type: -

Identifier Source: org_study_id