Ignite Creation Date:
2025-12-25 @ 1:10 AM
Ignite Modification Date:
2025-12-25 @ 11:21 PM
Study NCT ID:
NCT06268093
Status:
RECRUITING
Last Update Posted:
2024-02-20
First Post:
2024-02-07
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
The Therapeutic Effect of Thalidomide 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': 'D013792', 'term': 'Thalidomide'}], 'ancestors': [{'id': 'D010797', 'term': 'Phthalimides'}, {'id': 'D010795', 'term': 'Phthalic Acids'}, {'id': 'D000146', 'term': 'Acids, Carbocyclic'}, {'id': 'D002264', 'term': 'Carboxylic Acids'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D010881', 'term': 'Piperidones'}, {'id': 'D010880', 'term': 'Piperidines'}, {'id': 'D006573', 'term': 'Heterocyclic Compounds, 1-Ring'}, {'id': 'D006571', 'term': 'Heterocyclic Compounds'}, {'id': 'D054833', 'term': 'Isoindoles'}, {'id': 'D006574', 'term': 'Heterocyclic Compounds, 2-Ring'}, {'id': 'D000072471', 'term': 'Heterocyclic Compounds, Fused-Ring'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 30}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2024-02', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-02', 'completionDateStruct': {'date': '2028-02', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2024-02-19', 'studyFirstSubmitDate': '2024-02-07', 'studyFirstSubmitQcDate': '2024-02-19', 'lastUpdatePostDateStruct': {'date': '2024-02-20', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2024-02-20', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2027-02', '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': 'The evoked electromyographic signal (eEMG) potential', 'timeFrame': '1 day before and 3 days, 3 months after drug treatment', 'description': 'The evoked electromyographic signal (eEMG) potential is the standard index'}, {'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': '1 day before and 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': '1 day before and 3 days, 3 months after drug treatment', 'description': 'Incidence of complications'}]}, 'oversightModule': {'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Syringomyelia', 'Thalidomide']}, 'referencesModule': {'references': [{'pmid': '37913505', 'type': 'BACKGROUND', 'citation': 'Chen H, Wu S, Tang M, Zhao R, Zhang Q, Dai Z, Gao Y, Yang S, Li Z, Du Y, Yang A, Zhong L, Lu L, Xu L, Shen X, Liu S, Zhong J, Li X, Lu H, Xiong H, Shen Y, Chen H, Gong S, Xue H, Ge Z. Thalidomide for Recurrent Bleeding Due to Small-Intestinal Angiodysplasia. N Engl J Med. 2023 Nov 2;389(18):1649-1659. doi: 10.1056/NEJMoa2303706.'}, {'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': '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 phase II clinical trial aims to evaluate the indications, therapeutic effects and side effects of thalidomide 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 thalidomide 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 blood spinal cord barrier(BSCB) might play a key role in the pathogenesis of syringomyelia, especially post-traumatic syringomyelia(PTS), and that thalidomide, as an BSCB protection-related drug, would reduce BSCB damage and protect BSCB in syringomyelia.\n\nPrimary objectives: This phase II clinical trial aims to evaluate the indications, therapeutic effects and safety of thalidomide 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* or 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* Routine laboratory studies: bilirubin \\</=1.0 \\* upper limits of normal (ULN); aspartate aminotransferase (AST or SGOT) or alanine aminotransferase (ALT)\\< 1.0 \\* ULN; creatinine \\<1.0 \\* ULN; white-cell count \\>/= 4,000 per cubic millimeter; neutrophils count \\>/=1500 per cubic millimeter platelets \\>/= 100,000 per cubic millimeter; Hb \\>/=110 gram per millilitres; PT, APTT, INR in a normal range.\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* 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': 'NCT06268093', 'briefTitle': 'The Therapeutic Effect of Thalidomide in Syringomyelia', 'organization': {'class': 'OTHER', 'fullName': 'Xuanwu Hospital, Beijing'}, 'officialTitle': 'The Effect of Thalidomide in Refractory Syringomyelia（RS）: a Phase II Clinical Trial', 'orgStudyIdInfo': {'id': 'XWTETS'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Thalidomide', 'description': 'Oral Thalidomide', 'interventionNames': ['Drug: Thalidomide']}], 'interventions': [{'name': 'Thalidomide', 'type': 'DRUG', 'description': 'Thalidomide 50 - 200 mg once at nightime', 'armGroupLabels': ['Thalidomide']}]}, 'contactsLocationsModule': {'locations': [{'zip': '100032', 'city': 'Beijing', 'state': 'Beijing City', 'status': 'RECRUITING', 'country': 'China', 'contacts': [{'name': 'fengzeng jian, md', 'role': 'CONTACT', 'email': 'jianfengzeng@xwh.ccmu.edu.cn'}], 'facility': 'Fengzeng Jian', 'geoPoint': {'lat': 39.9075, 'lon': 116.39723}}], 'centralContacts': [{'name': 'fengzeng jian', 'role': 'CONTACT', 'email': 'jianfengzeng@xwh.ccmu.edu.cn', 'phone': '+861083198899'}, {'name': 'chenghua yuan', 'role': 'CONTACT', 'email': 'yuanchenghua@ccmu.edu.cn', 'phone': '+861083198899'}], 'overallOfficials': [{'name': 'fengzeng jian', 'role': 'STUDY_CHAIR', 'affiliation': 'Xuanwu Hospital, Beijing'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'UNDECIDED'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Xuanwu Hospital, Beijing', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}