Syringomyelia, Thalidomide
Conditions
Brief summary
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.
Detailed description
The pathogenesis of syringomyelia is poorly understood and duraplasty or shunting is not always effective. Although it is generally thought that syringomyelia is simply an accumulation of CSF from the subarachnoid space, the pathogenesis is likely to be more complex and may involve cellular and molecular processes. The investigators supposed that blood spinal cord barrier(BSCB) might play a key role in the pathogenesis of syringomyelia, especially post-traumatic syringomyelia(PTS), and that thalidomide, as an BSCB protection-related drug, would reduce BSCB damage and protect BSCB in syringomyelia. Primary objectives: This phase II clinical trial aims to evaluate the indications, therapeutic effects and safety of thalidomide in refractory syringomyelia.
Interventions
DRUGThalidomide
Thalidomide 50 - 200 mg once at nightime
Sponsors
Xuanwu Hospital, Beijing
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| ASIA Score | 1 day before and 3 days, 3 months after drug treatment | American Spinal Injury Association(ASIA) Score for evaluating the spinal cord function， degree of the spinal cord function, motor1-100, sensory 1-224, higher scores mean a better outcome |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| The evoked electromyographic signal (eEMG) potential | 1 day before and 3 days, 3 months after drug treatment | The evoked electromyographic signal (eEMG) potential is the standard index |
| Visual Analog Scale (VAS) | 1 day before and 3 days, 3 months after drug treatment | degree of the pain, 1-10, higher scores mean a worse outcome |
| Klekamp and Sammi syringomyelia scale | 1 day before and 3 days, 3 months after drug treatment | for evaluating the spinal cord function, higher scores mean a better outcome |
| syringomyelia remission | 1 day before and 3 days, 3 months after drug treatment | syringomyelia remission is defined as ≥ 25% reduction in syringomyelia volume on T2 images at week 12, as compared with that before thalidomide usage |
| xuanwu syringomyelia scale | 1 day before and 3 days, 3 months after drug treatment | for evaluating the spinal cord function, for evaluating the spinal cord function；0-18， higher scores mean a worse outcome |
| Incidence of complications | 1 day before and 3 days, 3 months after drug treatment | Incidence of complications |
| modified Japanese Orthopaedic Association Scores (mJOA) | 1 day before and 3 days, 3 months after drug treatment | Motor function， sensory， bladder function；for evaluating the spinal cord function；0-17， higher scores mean a better outcome |
Countries
China
Contacts
Primary Contactfengzeng jian
Backup Contactchenghua yuan
Outcome results
None listed