The Therapeutic Effect of Betaine in Syringomyelia

March 11, 2024 updated by: Xuanwu Hospital, Beijing

The Effect of Betaine in Refractory Syringomyelia（RS）

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Estimated)

30

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: betaine
oral betaine
Drug: Betaine
50mg/kg for 12 weeks
Placebo Comparator: placebo
rice-flour as a placebo
Drug: Placebo
Placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
ASIA Score
Time Frame: 1 day before and 3 days, 3 months, after drug treatment
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 Outcome Measures

Outcome Measure
Measure Description
Time Frame
syringomyelia remission
Time Frame: 1 day before and 3 days, 3 months, after drug treatment
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
Electrophysiology results
Time Frame: 1 day before and 3 days, 3 months, after drug treatment
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
Visual Analog Scale (VAS)
Time Frame: 1 day before and 3 days, 3 months, after drug treatment
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
Time Frame: 1 day before and 3 days, 3 months, after drug treatment
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)
Time Frame: 1 day before and 3 days, 3 months, after drug treatment
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
Time Frame: 3 days, 3 months, after drug treatment
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
Incidence of complications
Time Frame: 3 days, 3 months, after drug treatment
Incidence of complications
3 days, 3 months, after drug treatment

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Xuanwu Hospital, Beijing

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

March 1, 2024

Primary Completion (Estimated)

March 1, 2027

Study Completion (Estimated)

March 1, 2028

Study Registration Dates

First Submitted

February 17, 2024

First Submitted That Met QC Criteria

March 11, 2024

First Posted (Actual)

March 13, 2024

Study Record Updates

Last Update Posted (Actual)

March 13, 2024

Last Update Submitted That Met QC Criteria

March 11, 2024

Last Verified

February 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • XWTEBS

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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