Thalidomide treatment for syringomyelia

The Effect of Thalidomide in Refractory Syringomyelia（RS）: a Phase II Clinical Trial

This study is testing if thalidomide can help improve symptoms for people with syringomyelia, a condition that causes fluid-filled cavities in the spinal cord.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 30 (estimated)
• Ages: 18 Years to 80 Years
• Sex: All
• Sponsor: Xuanwu Hospital, Beijing (Academic / other)
• Locations: 1 site (Beijing, Beijing City)
• Trial ID: NCT06268093 on ClinicalTrials.gov

What this trial studies

This phase II clinical trial evaluates the therapeutic effects and safety of thalidomide in patients with refractory syringomyelia, a condition where fluid-filled cavities form within the spinal cord. The study aims to determine if thalidomide can improve neurological function as measured by the ASIA scale after 12 weeks of treatment. The hypothesis is that thalidomide may protect the blood spinal cord barrier, potentially addressing the underlying pathogenesis of syringomyelia. Participants will be monitored for both efficacy and side effects throughout the trial.

Who should consider this trial

Good fit: Ideal candidates are patients with refractory syringomyelia who cannot undergo duraplasty or shunting due to contraindications or have not had a successful response to these treatments in the past year.

Not a fit: Patients with evidence of tumor metastasis, recurrence, or invasion will not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with refractory syringomyelia who have not responded to standard surgical interventions.

How similar studies have performed: While thalidomide has been studied for various conditions, this specific application in syringomyelia is novel and has not been extensively tested in prior studies.

Eligibility criteria

Inclusion Criteria:

* Contraindication to duraplasty and shunting treatment due to history or high risk of severe adverse effects,
* or non-effective response to duraplasty and shunting treatment in 12 months prior to study entry.
* Estimated life expectancy must be greater than 12 months.
* Routine laboratory studies: bilirubin \</=1.0 \* upper limits of normal (ULN); aspartate aminotransferase (AST or SGOT) or alanine aminotransferase (ALT)\< 1.0 \* ULN; creatinine \<1.0 \* ULN; white-cell count \>/= 4,000 per cubic millimeter; neutrophils count \>/=1500 per cubic millimeter platelets \>/= 100,000 per cubic millimeter; Hb \>/=110 gram per millilitres; PT, APTT, INR in a normal range.
* Ability to understand and willingness to sign a written informed consent document, or constant caregivers who well understand and willingness to sign a written informed consent document.
* Must be able to swallow tablets

Exclusion Criteria:

* Evidence of tumor metastasis, recurrence, or invasion;
* History of psychiatric diseases ;
* History of seizures;
* History of arteriosclerotic cardiovascular diseases (ASCVD), e.g. stroke, myocardial infaction, unstable angina, within 6 months;
* New York Heart Association Grade II or greater congestive heart failure;
* Serious and inadequately controlled cardiac arrhythmia;
* Significant vascular disease, e.g. moderate or severe carotid stenosis, aortic aneurysm, history of aortic dissection;
* Severe infection;
* History of allergy to relevant drugs;
* Pregnancy, lactation, or fertility program in the following 12 months;
* History or current diagnosis of peripheral nerve disease;
* Abnormal in liver and renal function;
* Active tuberculosis;
* Transplanted organs;
* Human immunodeficiency virus;
* Participation in other experimental studies.

Where this trial is running

Beijing, Beijing City

• Fengzeng Jian — Beijing, Beijing City, China (Recruiting)

Study contacts

• Study coordinator: fengzeng jian

Conditions Syringomyelia; Thalidomide