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Recruiting NCT06308367

The Therapeutic Effect of Betaine in Syringomyelia

Phase II Interventional Syringomyelia

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Betaine, Placebo.
Who it may be relevant to
Registry conditions: Syringomyelia. Basic parameters: 18 years — 80 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
China
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

The Effect of Betaine in Refractory Syringomyelia（RS）

Overview

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.

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 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.

Interventions

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

Primary outcome measures

  • ASIA Score [Time frame: 1 day before and 3 days, 3 months, after drug treatment]
Secondary outcome measures (7)
  • syringomyelia remission [Time frame: 1 day before and 3 days, 3 months, after drug treatment]
  • Electrophysiology results [Time frame: 1 day before and 3 days, 3 months, after drug treatment]
  • Visual Analog Scale (VAS) [Time frame: 1 day before and 3 days, 3 months, after drug treatment]
  • Klekamp and Sammi syringomyelia scale [Time frame: 1 day before and 3 days, 3 months, after drug treatment]
  • modified Japanese Orthopaedic Association Scores (mJOA) [Time frame: 1 day before and 3 days, 3 months, after drug treatment]
  • xuanwu syringomyelia scale [Time frame: 3 days, 3 months, after drug treatment]
  • Incidence of complications [Time frame: 3 days, 3 months, after drug treatment]

Eligibility criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Parallel assignment
Masking
Quadruple blind
Primary purpose
Treatment

Study locations

China · 1 center
  • Fengzeng Jian — Beijing

Publications

  • 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
  • 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. PMID 27736311
  • 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. PMID 32943488
  • 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
  • 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
  • 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
  • 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
  • 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

Identifiers

NCT: NCT06308367 · XWTEBS

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗