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

XWTETS

Purpose: This phase II clinical trial aims to evaluate the indications, therapeutic effects and side effects of thalidomide in refractory syringomyelia. Primary outcome measure: The primary endpoint is the change of ASIA at week 12. The clinical efficacy is defined as ASIA increase ≥ 1 at week 12, as compared with that before thalidomide usage.

2024-02-01

2027-02-01

2028-02-01

Recruiting

Early phase I

30

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.

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 30, 'type': 'ESTIMATED'}}

2024-02-20