Ignite Creation Date:
2025-12-25 @ 12:54 AM
Ignite Modification Date:
2025-12-25 @ 12:54 AM
Study NCT ID:
NCT06308367
Status:
RECRUITING
Last Update Posted:
2024-03-13
First Post:
2024-02-17
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
The Therapeutic Effect of Betaine in Syringomyelia
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D013595', 'term': 'Syringomyelia'}], 'ancestors': [{'id': 'D013118', 'term': 'Spinal Cord Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D001622', 'term': 'Betaine'}], 'ancestors': [{'id': 'D050337', 'term': 'Trimethyl Ammonium Compounds'}, {'id': 'D000644', 'term': 'Quaternary Ammonium Compounds'}, {'id': 'D000588', 'term': 'Amines'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D009861', 'term': 'Onium Compounds'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 30}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2024-03', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-02', 'completionDateStruct': {'date': '2028-03', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2024-03-11', 'studyFirstSubmitDate': '2024-02-17', 'studyFirstSubmitQcDate': '2024-03-11', 'lastUpdatePostDateStruct': {'date': '2024-03-13', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2024-03-13', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2027-03', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'ASIA Score', 'timeFrame': '1 day before and 3 days, 3 months, after drug treatment', 'description': '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'}], 'secondaryOutcomes': [{'measure': 'syringomyelia remission', 'timeFrame': '1 day before and 3 days, 3 months, after drug treatment', 'description': 'syringomyelia remission is defined as ≥ 25% reduction in syringomyelia volume on T2 images at week 12, as compared with that before thalidomide usage'}, {'measure': 'Electrophysiology results', 'timeFrame': '1 day before and 3 days, 3 months, after drug treatment', 'description': 'electromyography and evoked potential; Change of N9-13 From Baseline in Electrophysiology at postoperation'}, {'measure': 'Visual Analog Scale (VAS)', 'timeFrame': '1 day before and 3 days, 3 months, after drug treatment', 'description': 'degree of the pain, 1-10, higher scores mean a worse outcome'}, {'measure': 'Klekamp and Sammi syringomyelia scale', 'timeFrame': '1 day before and 3 days, 3 months, after drug treatment', 'description': 'for evaluating the spinal cord function, higher scores mean a better outcome'}, {'measure': 'modified Japanese Orthopaedic Association Scores (mJOA)', 'timeFrame': '1 day before and 3 days, 3 months, after drug treatment', 'description': 'Motor function， sensory， bladder function；for evaluating the spinal cord function；0-17， higher scores mean a better outcome'}, {'measure': 'xuanwu syringomyelia scale', 'timeFrame': '3 days, 3 months, after drug treatment', 'description': 'for evaluating the spinal cord function, for evaluating the spinal cord function；0-18， higher scores mean a worse outcome'}, {'measure': 'Incidence of complications', 'timeFrame': '3 days, 3 months, after drug treatment', 'description': 'Incidence of complications'}]}, 'oversightModule': {'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Syringomyelia']}, 'referencesModule': {'references': [{'pmid': '37046133', 'type': 'BACKGROUND', 'citation': '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': '27736311', 'type': 'BACKGROUND', '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.'}, {'pmid': '32943488', 'type': 'BACKGROUND', '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.'}, {'pmid': '23082850', 'type': 'RESULT', '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.'}, {'pmid': '38014747', 'type': 'RESULT', '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 1976). 2024 Mar 15;49(6):E62-E71. doi: 10.1097/BRS.0000000000004884. Epub 2023 Nov 28.'}, {'pmid': '36424067', 'type': 'RESULT', '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.'}, {'pmid': '32805463', 'type': 'RESULT', '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 Treatment. World Neurosurg. 2020 Dec;144:e178-e188. doi: 10.1016/j.wneu.2020.08.068. Epub 2020 Aug 15.'}, {'pmid': '37248485', 'type': 'RESULT', '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.'}]}, 'descriptionModule': {'briefSummary': 'Purpose: This clinical trial aims to evaluate the indications, therapeutic effects and side effects of betaine in refractory syringomyelia.\n\nPrimary 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.', 'detailedDescription': '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.\n\nThe 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.\n\nPrimary objectives: This clinical trial aims to evaluate the indications, therapeutic effects and safety of betaine in refractory syringomyelia.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '80 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Contraindication to duraplasty and shunting treatment due to history or high risk of severe adverse effects,\n* non-effective response to duraplasty and shunting treatment in 12 months prior to study entry.\n* Estimated life expectancy must be greater than 12 months.\n* 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.\n* Must be able to swallow tablets\n\nExclusion Criteria:\n\n* Participants have a cardiometabolic disease for which they take prescribed medications\n* Evidence of tumor metastasis, recurrence, or invasion;\n* History of psychiatric diseases ;\n* History of seizures;\n* History of arteriosclerotic cardiovascular diseases (ASCVD), e.g. stroke, myocardial infaction, unstable angina, within 6 months;\n* New York Heart Association Grade II or greater congestive heart failure;\n* Serious and inadequately controlled cardiac arrhythmia;\n* Significant vascular disease, e.g. moderate or severe carotid stenosis, aortic aneurysm, -history of aortic dissection;\n* Severe infection;\n* History of allergy to relevant drugs;\n* Pregnancy, lactation, or fertility program in the following 12 months;\n* History or current diagnosis of peripheral nerve disease;\n* Abnormal in liver and renal function;\n* Active tuberculosis;\n* Transplanted organs;\n* Human immunodeficiency virus;\n* Participation in other experimental studies.'}, 'identificationModule': {'nctId': 'NCT06308367', 'briefTitle': 'The Therapeutic Effect of Betaine in Syringomyelia', 'organization': {'class': 'OTHER', 'fullName': 'Xuanwu Hospital, Beijing'}, 'officialTitle': 'The Effect of Betaine in Refractory Syringomyelia（RS）', 'orgStudyIdInfo': {'id': 'XWTEBS'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'betaine', 'description': 'oral betaine', 'interventionNames': ['Drug: Betaine']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'placebo', 'description': 'rice-flour as a placebo', 'interventionNames': ['Drug: Placebo']}], 'interventions': [{'name': 'Betaine', 'type': 'DRUG', 'description': '50mg/kg for 12 weeks', 'armGroupLabels': ['betaine']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Placebo', 'armGroupLabels': ['placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '100053', 'city': 'Beijing', 'state': 'Beijing Municipality', 'status': 'RECRUITING', 'country': 'China', 'contacts': [{'name': 'Yuan Chenghua', 'role': 'CONTACT', 'email': 'yuanchenghua@ccmu.edu.cn'}, {'name': 'Jian Fengzeng', 'role': 'CONTACT', 'email': 'jianfengzeng@xwh.ccmu.edu.cn'}, {'name': 'jian guan, dr', 'role': 'SUB_INVESTIGATOR'}], 'facility': 'Fengzeng Jian', 'geoPoint': {'lat': 39.9075, 'lon': 116.39723}}], 'centralContacts': [{'name': 'jian fengzeng', 'role': 'CONTACT', 'email': 'jianfengzeng@xwh.ccmu.edu.cn', 'phone': '+861083198899'}, {'name': 'chenghua yuan', 'role': 'CONTACT', 'email': 'yuanchenghua@ccmu.edu.cn', 'phone': '+861083198899'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'UNDECIDED'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Xuanwu Hospital, Beijing', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}