Syringomyelia Clinical Trial
Official title:
The Effect of Thalidomide in Refractory Syringomyelia(RS): a Phase II Clinical Trial
NCT number | NCT06268093 |
Other study ID # | XWTETS |
Secondary ID | |
Status | Recruiting |
Phase | Phase 2 |
First received | |
Last updated | |
Start date | February 2024 |
Est. completion date | February 2028 |
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.
Status | Recruiting |
Enrollment | 30 |
Est. completion date | February 2028 |
Est. primary completion date | February 2027 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years to 80 Years |
Eligibility | 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. |
Country | Name | City | State |
---|---|---|---|
China | Fengzeng Jian | Beijing | Beijing City |
Lead Sponsor | Collaborator |
---|---|
Xuanwu Hospital, Beijing |
China,
Chen H, Wu S, Tang M, Zhao R, Zhang Q, Dai Z, Gao Y, Yang S, Li Z, Du Y, Yang A, Zhong L, Lu L, Xu L, Shen X, Liu S, Zhong J, Li X, Lu H, Xiong H, Shen Y, Chen H, Gong S, Xue H, Ge Z. Thalidomide for Recurrent Bleeding Due to Small-Intestinal Angiodysplasia. N Engl J Med. 2023 Nov 2;389(18):1649-1659. doi: 10.1056/NEJMoa2303706. — View Citation
Guan J, Yuan C, Yao Q, Du Y, Fang Z, Zhang L, Jia S, Zhang C, Liu Z, Wang K, Duan W, Wang Z, Wang X, Wu H, Chen Z, Jian F. A novel scoring system for assessing adult syringomyelia associated with CM I treatment outcomes. Acta Neurol Belg. 2023 Jun;123(3):807-814. doi: 10.1007/s13760-023-02264-4. Epub 2023 Apr 12. — View Citation
Guan J, Yuan C, Zhang C, Ma L, Yao Q, Cheng L, Liu Z, Wang K, Duan W, Wang X, Wu H, Chen Z, Jian F. Intradural Pathology Causing Cerebrospinal Fluid Obstruction in Syringomyelia and Effectiveness of Foramen Magnum and Foramen of Magendie Dredging Treatmen — View Citation
Heiss JD. Cerebrospinal Fluid Hydrodynamics in Chiari I Malformation and Syringomyelia: Modeling Pathophysiology. Neurosurg Clin N Am. 2023 Jan;34(1):81-90. doi: 10.1016/j.nec.2022.08.007. Epub 2022 Nov 3. — View Citation
Hemley SJ, Bilston LE, Cheng S, Stoodley MA. Aquaporin-4 expression and blood-spinal cord barrier permeability in canalicular syringomyelia. J Neurosurg Spine. 2012 Dec;17(6):602-12. doi: 10.3171/2012.9.SPINE1265. Epub 2012 Oct 19. — View Citation
Liu S, Ma L, Qi B, Li Q, Chen Z, Jian F. Suppression of TGFbetaR-Smad3 pathway alleviates the syrinx induced by syringomyelia. Cell Biosci. 2023 May 29;13(1):98. doi: 10.1186/s13578-023-01048-w. — View Citation
Yuan C, Xia P, Duan W, Wang J, Guan J, Du Y, Zhang C, Liu Z, Wang K, Wang Z, Wang X, Wu H, Chen Z, Jian F. Long-term Impairment of the Blood Spinal Cord Barrier in Patients with Posttraumatic Syringomyelia and its Effect on Prognosis. Spine (Phila Pa 1976 — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | ASIA Score | 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 | 1 day before and 3 days, 3 months after drug treatment | |
Secondary | syringomyelia remission | syringomyelia remission is defined as = 25% reduction in syringomyelia volume on T2 images at week 12, as compared with that before thalidomide usage | 1 day before and 3 days, 3 months after drug treatment | |
Secondary | The evoked electromyographic signal (eEMG) potential | The evoked electromyographic signal (eEMG) potential is the standard index | 1 day before and 3 days, 3 months after drug treatment | |
Secondary | Visual Analog Scale (VAS) | degree of the pain, 1-10, higher scores mean a worse outcome | 1 day before and 3 days, 3 months after drug treatment | |
Secondary | Klekamp and Sammi syringomyelia scale | for evaluating the spinal cord function, higher scores mean a better outcome | 1 day before and 3 days, 3 months after drug treatment | |
Secondary | modified Japanese Orthopaedic Association Scores (mJOA) | Motor function, sensory, bladder function;for evaluating the spinal cord function;0-17, higher scores mean a better outcome | 1 day before and 3 days, 3 months after drug treatment | |
Secondary | xuanwu syringomyelia scale | for evaluating the spinal cord function, for evaluating the spinal cord function;0-18, higher scores mean a worse outcome | 1 day before and 3 days, 3 months after drug treatment | |
Secondary | Incidence of complications | Incidence of complications | 1 day before and 3 days, 3 months after drug treatment |
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