NCT06308367

Brief 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.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
30

participants targeted

Target at P25-P50 for phase_2

Timeline
22mo left

Started Mar 2024

Typical duration for phase_2

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress55%
Mar 2024Mar 2028

First Submitted

Initial submission to the registry

February 17, 2024

Completed
13 days until next milestone

Study Start

First participant enrolled

March 1, 2024

Completed
12 days until next milestone

First Posted

Study publicly available on registry

March 13, 2024

Completed
3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2027

Expected
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2028

Last Updated

March 13, 2024

Status Verified

February 1, 2024

Enrollment Period

3 years

First QC Date

February 17, 2024

Last Update Submit

March 11, 2024

Conditions

Syringomyelia

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

  • Electrophysiology results

    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

  • +2 more secondary outcomes

Study Arms (2)

betaine

EXPERIMENTAL

oral betaine

Drug: Betaine

placebo

PLACEBO COMPARATOR

rice-flour as a placebo

Drug: Placebo

Interventions

BetaineDRUG

50mg/kg for 12 weeks

betaine
PlaceboDRUG

Placebo

placebo

Eligibility Criteria

Age18 Years - 80 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (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,
  • 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

You may not qualify if:

  • 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;
  • +2 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Fengzeng Jian

Beijing, Beijing Municipality, 100053, China

RECRUITING

Related 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.

    PMID: 37046133BACKGROUND

  • 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: 27736311BACKGROUND

  • 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: 32943488BACKGROUND

  • 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: 23082850RESULT

  • 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: 38014747RESULT

  • 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: 36424067RESULT

  • 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: 32805463RESULT

  • 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: 37248485RESULT

MeSH Terms

Conditions

Syringomyelia

Interventions

Betaine

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System Diseases

Intervention Hierarchy (Ancestors)

Trimethyl Ammonium CompoundsQuaternary Ammonium CompoundsAminesOrganic ChemicalsOnium Compounds

Central Study Contacts

jian fengzeng

CONTACT

+861083198899jianfengzeng@xwh.ccmu.edu.cn

chenghua yuan

CONTACT

+861083198899yuanchenghua@ccmu.edu.cn

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 17, 2024

First Posted

March 13, 2024

Study Start

March 1, 2024

Primary Completion (Estimated)

March 1, 2027

Study Completion (Estimated)

March 1, 2028

Last Updated

March 13, 2024

Record last verified: 2024-02

Locations

CN(1)