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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT06308367
Other study ID # XWTEBS
Secondary ID
Status Recruiting
Phase Phase 2
First received
Last updated
Start date March 2024
Est. completion date March 2028

Study information

Verified date February 2024
Source Xuanwu Hospital, Beijing
Contact jian fengzeng
Phone +861083198899
Email jianfengzeng@xwh.ccmu.edu.cn
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

Purpose: This clinical trial aims to evaluate the indications, therapeutic effects and side effects of betaine 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 betaine usage.


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 betaine might play a key role in the pathogenesis of syringomyelia, especially post-traumatic syringomyelia(PTS), and that betaine, as an Osmotic homeostasis-related drug, would protect Osmotic homeostasis in syringomyelia. Primary objectives: This clinical trial aims to evaluate the indications, therapeutic effects and safety of betaine in refractory syringomyelia.


Recruitment information / eligibility

Status Recruiting
Enrollment 30
Est. completion date March 2028
Est. primary completion date March 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, - non-effective response to duraplasty and shunting treatment in 12 months prior to study entry. - Estimated life expectancy must be greater than 12 months. - 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: - Participants have a cardiometabolic disease for which they take prescribed medications - 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.

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
Betaine
50mg/kg for 12 weeks
Placebo
Placebo

Locations

Country Name City State
China Fengzeng Jian Beijing Beijing

Sponsors (1)

Lead Sponsor Collaborator
Xuanwu Hospital, Beijing

Country where clinical trial is conducted

China, 

References & Publications (8)

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

He R, Zhang H, Kang L, Li H, Shen M, Zhang Y, Mo R, Liu Y, Song J, Chen Z, Liu Y, Jin Y, Li M, Dong H, Zheng H, Li D, Qin J, Zhang H, Huang M, Liang D, Tian Y, Yao H, Yang Y. Analysis of 70 patients with hydrocephalus due to cobalamin C deficiency. Neurology. 2020 Dec 8;95(23):e3129-e3137. doi: 10.1212/WNL.0000000000010912. Epub 2020 Sep 17. — 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

Mohrman AE, Farrag M, Huang H, Ossowski S, Haft S, Shriver LP, Leipzig ND. Spinal Cord Transcriptomic and Metabolomic Analysis after Excitotoxic Injection Injury Model of Syringomyelia. J Neurotrauma. 2017 Feb;34(3):720-733. doi: 10.1089/neu.2015.4341. Epub 2016 Oct 13. — 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 Post-Traumatic Syringomyelia and its Effect on Prognosis. Spine (Phila Pa 197 — View Citation

Outcome

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 Electrophysiology results electromyography and evoked potential; Change of N9-13 From Baseline in Electrophysiology at postoperation 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 3 days, 3 months, after drug treatment
Secondary Incidence of complications Incidence of complications 3 days, 3 months, after drug treatment
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