The Therapeutic Effect of Thalidomide in Syringomyelia

NCT06268093 | Recruiting Phase 2

• 1 other identifier: XWTETS
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

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.

Conditions

Syringomyelia; Thalidomide

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (7)

• syringomyelia remission

  1 day before and 3 days, 3 months after drug treatment

• The evoked electromyographic signal (eEMG) potential

  1 day before and 3 days, 3 months after drug treatment

• Visual Analog Scale (VAS)

  1 day before and 3 days, 3 months after drug treatment

• Klekamp and Sammi syringomyelia scale

  1 day before and 3 days, 3 months after drug treatment

• modified Japanese Orthopaedic Association Scores (mJOA)

  1 day before and 3 days, 3 months after drug treatment

Study Arms (1)

Thalidomide

EXPERIMENTAL

Oral Thalidomide

Drug: Thalidomide

Interventions

Thalidomide DRUG

Thalidomide 50 - 200 mg once at nightime

Thalidomide

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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;

Sponsors & Collaborators

• Xuanwu Hospital, Beijing: lead

Study Sites (1)

Fengzeng Jian

Beijing, Beijing City, 100032, China

RECRUITING

Related Publications (7)

• 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.

  PMID: 37913505 BACKGROUND

• 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.

  PMID: 37046133 BACKGROUND

• 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.

  PMID: 23082850 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.

  PMID: 38014747 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.

  PMID: 36424067 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.

  PMID: 32805463 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.

  PMID: 37248485 RESULT

MeSH Terms

Conditions

Syringomyelia

Interventions

Thalidomide

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases

Intervention Hierarchy (Ancestors)

Phthalimides; Phthalic Acids; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Piperidones; Piperidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Isoindoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Study Officials

• fengzeng jian

  Xuanwu Hospital, Beijing

  STUDY CHAIR

Central Study Contacts

fengzeng jian

CONTACT

+861083198899; jianfengzeng@xwh.ccmu.edu.cn

chenghua yuan

CONTACT

+861083198899; yuanchenghua@ccmu.edu.cn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 7, 2024
• First Posted: February 20, 2024
• Study Start: February 1, 2024
• Primary Completion (Estimated): February 1, 2027
• Study Completion (Estimated): February 1, 2028
• Last Updated: February 20, 2024

Record last verified: 2024-02

Locations

CN (1)