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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Thalidomide | Experimental | Oral Thalidomide |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Thalidomide | Drug | Thalidomide 50 - 200 mg once at nightime |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| The evoked electromyographic signal (eEMG) potential |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| fengzeng jian | Contact | +861083198899 | jianfengzeng@xwh.ccmu.edu.cn | |
| chenghua yuan | Contact | +861083198899 | yuanchenghua@ccmu.edu.cn |
| Name | Affiliation | Role |
|---|---|---|
| fengzeng jian | Xuanwu Hospital, Beijing | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Fengzeng Jian | Recruiting | Beijing | Beijing City | 100032 | China |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37913505 | Background | 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. | |
| 37046133 |
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| ID | Term |
|---|---|
| D013595 | Syringomyelia |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
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| ID | Term |
|---|---|
| D013792 | Thalidomide |
| ID | Term |
|---|---|
| D010797 | Phthalimides |
| D010795 | Phthalic Acids |
| D000146 | Acids, Carbocyclic |
| D002264 | Carboxylic Acids |
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The evoked electromyographic signal (eEMG) potential is the standard index |
| 1 day before and 3 days, 3 months after drug treatment |
| 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 |
| 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 |
| 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 |
| 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 |
| Incidence of complications | Incidence of complications | 1 day before and 3 days, 3 months after drug treatment |
| 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. |
| 23082850 | Result | 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. |
| 38014747 | Result | 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 Post-Traumatic Syringomyelia and its Effect on Prognosis. Spine (Phila Pa 1976). 2024 Mar 15;49(6):E62-E71. doi: 10.1097/BRS.0000000000004884. Epub 2023 Nov 28. |
| 36424067 | Result | 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. |
| 32805463 | Result | 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 Treatment. World Neurosurg. 2020 Dec;144:e178-e188. doi: 10.1016/j.wneu.2020.08.068. Epub 2020 Aug 15. |
| 37248485 | Result | 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. |
| D009930 |
| Organic Chemicals |
| D010881 | Piperidones |
| D010880 | Piperidines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D054833 | Isoindoles |
| D006574 | Heterocyclic Compounds, 2-Ring |
| D000072471 | Heterocyclic Compounds, Fused-Ring |