Intrathecal umbilical cord MSC extracellular vesicles for syringomyelia
An Exploratory Study on the Use of Intrathecal Injection of Human Autologous Mesenchymal Stromal Cells Derived Extracellular Vesicles for the Treatment of Syringomyelia
This trial will try monthly lumbar‑puncture injections of umbilical‑cord‑derived mesenchymal stromal cell extracellular vesicles in adults with persistent symptoms after syrinx shrinkage to see if the treatment is safe and helps symptoms.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 18 (estimated) |
| Ages | 18 Years to 70 Years |
| Sex | All |
| Sponsor | Xuanwu Hospital, Beijing Academic / other |
| Locations | 1 site (Beijing, Beijing City) |
| Trial ID | NCT07295067 on ClinicalTrials.gov |
What this trial studies
This is an open‑label, single‑arm phase I dose‑escalation trial using a traditional 3+3 design to test intrathecal human umbilical cord‑derived MSC extracellular vesicles in adults with syringomyelia and ongoing spinal cord symptoms despite syrinx shrinkage or spontaneous resolution. Three sequential cohorts will receive low (1×10^11 particles), middle (2×10^11), or high (3×10^11) doses delivered as 2 mL by lumbar puncture once a month for three months, with dose‑limiting toxicity rules guiding escalation. Safety is the primary focus, with preliminary efficacy signals captured through clinical neurological assessments and imaging. Participants will be adults aged 18–70 without major comorbidities or contraindications to intrathecal injection.
Who should consider this trial
Good fit: Ideal candidates are adults 18–70 whose MRI shows syrinx shrinkage or spontaneous resolution after conservative care or surgery but who continue to have spinal cord symptoms, and who do not have major organ dysfunction or active infections.
Not a fit: Patients with significant comorbidities (uncontrolled diabetes, heart disease, liver or kidney impairment), active infections at the puncture site, tumors of the CNS, autoimmune disease requiring immunosuppression, recent cell therapy, or who are otherwise ineligible for lumbar puncture are unlikely to benefit or be eligible.
Why it matters
Potential benefit: If successful, this approach could provide a new, minimally invasive option to reduce neurological symptoms and improve function in patients whose syrinx has resolved radiographically but who still have deficits.
How similar studies have performed: Preclinical studies of MSC‑derived extracellular vesicles in spinal cord injury show promising neuroprotective and repair signals, but human clinical data are very limited and this approach remains largely experimental.
Eligibility criteria
Show full inclusion / exclusion 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: 18-70 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
Where this trial is running
Beijing, Beijing City
- Xuanwu Hospital — Beijing, Beijing City, China (Recruiting)
Study contacts
- Study coordinator: fengzeng jian, md
- Email: jianfengzeng@xwh.ccmu.edu.cn
- Phone: +861083198899
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.