Ignite Creation Date:
2025-12-25 @ 12:45 AM
Ignite Modification Date:
2026-02-24 @ 10:56 AM
Study NCT ID:
NCT07295067
Status:
RECRUITING
Last Update Posted:
2025-12-19
First Post:
2025-11-19
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Extracellular Vesicles for the Treatment of Syringomyelia
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D013595', 'term': 'Syringomyelia'}, {'id': 'D013119', 'term': 'Spinal Cord Injuries'}], 'ancestors': [{'id': 'D013118', 'term': 'Spinal Cord Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D020196', 'term': 'Trauma, Nervous System'}, {'id': 'D014947', 'term': 'Wounds and Injuries'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 18}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2025-12-15', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-12', 'completionDateStruct': {'date': '2026-12-31', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-12-15', 'studyFirstSubmitDate': '2025-11-19', 'studyFirstSubmitQcDate': '2025-12-15', 'lastUpdatePostDateStruct': {'date': '2025-12-19', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2025-12-19', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2026-12-31', 'type': 'ESTIMATED'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Change from baseline in the blood and csf markers.', 'timeFrame': '4±1 Weeks, 8±1 Weeks,12±1 Weeks', 'description': 'A number of blood and csf markers will be examined.'}, {'measure': 'Change from baseline in the neuroimaging indices.', 'timeFrame': '4±1 Weeks, 8±1 Weeks,12±1 Weeks', 'description': 'Neuroimaging indices include DTI, DTI-ALPS.'}, {'measure': 'Change from baseline in the Electrophysiological indices.', 'timeFrame': '4±1 Weeks, 8±1 Weeks,12±1 Weeks', 'description': 'Electrophysiological indices include EMG.'}, {'measure': 'Change from baseline in the neuroimaging indices.', 'timeFrame': '4±1 Weeks, 8±1 Weeks,12±1 Weeks', 'description': 'Neuroimaging indices include QSM.'}, {'measure': 'Change from baseline in the Electrophysiological indices.', 'timeFrame': '4±1 Weeks, 8±1 Weeks,12±1 Weeks', 'description': 'Electrophysiological indices include Somatosensory evoked potential .'}, {'measure': 'Change from baseline in the Electrophysiological indices.', 'timeFrame': '4±1 Weeks, 8±1 Weeks,12±1 Weeks', 'description': 'Electrophysiological indices include Motor Evoked Potential.'}], 'primaryOutcomes': [{'measure': 'Number of participants who experienced dose-limiting Toxicities (DLTs)', 'timeFrame': '24 hours, 4±1 Weeks, 8±1 Weeks,12±1 Weeks', 'description': 'DLTs related to hUC-MSC-sEV include adverse events of grade 3 or higher (including significant clinical laboratory findings) that are possibly, likely, or definitely related to the study drug, accompanied by clinical symptoms and requiring medical treatment within 14 days of administration. Adverse events are graded according to the Common Terminology Criteria for Adverse Events Version 5.0 (CTCAE 5.0).'}], 'secondaryOutcomes': [{'measure': 'American Spinal Injury Association(ASIA) Score', 'timeFrame': '4±1 Weeks, 8±1 Weeks,12±1 Weeks, 12±1 months', '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'}, {'measure': 'Klekamp and Sammi syringomyelia scale', 'timeFrame': '4±1 Weeks, 8±1 Weeks,12±1 Weeks, 12±1 months', 'description': 'for evaluating the spinal cord function, each score 1-5, higher scores mean a better outcome'}, {'measure': 'Incidence of severe adverse events', 'timeFrame': '4±1 Weeks, 8±1 Weeks,12±1 Weeks', 'description': 'The proportion of patients who experienced severe adverse events.'}, {'measure': 'Time to event (death, tracheostomy, and permanent assisted mechanical ventilation)', 'timeFrame': 'up to 12 months', 'description': 'The number of time-to-event outcomes.'}, {'measure': 'modified Japanese Orthopaedic Association Scores (mJOA)', 'timeFrame': '4±1 Weeks, 8±1 Weeks,12±1 Weeks, 12±1 months', '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': '4±1 Weeks, 8±1 Weeks,12±1 Weeks, 12±1 months', 'description': 'for evaluating the spinal cord function, for evaluating the spinal cord function；0-18， higher scores mean a worse outcome'}, {'measure': 'improvement or resolution of the syrinx', 'timeFrame': '4±1 Weeks, 8±1 Weeks,12±1 Weeks, 12±1 months', 'description': 'improvement in size.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['syringomyelia', 'spinal cord injury', 'mesenchymal stromal cell', 'extracellular vesicle'], 'conditions': ['Syringomyelia']}, 'referencesModule': {'references': [{'pmid': '40675800', 'type': 'BACKGROUND', 'citation': 'Yuan C, Du Y, Yao Q, Zhang C, Zhang L, Liu Z, Wang K, Duan W, Wang Z, Wang X, Zeng G, Wu H, Chen Z, Heiss JD, Guan J, Jian FZ. Natural history of Chiari I malformation-syringomyelia: longitudinal cohort study. J Neurol Neurosurg Psychiatry. 2025 Nov 13;96(12):1204-1214. doi: 10.1136/jnnp-2025-336023.'}, {'pmid': '40536443', 'type': 'BACKGROUND', 'citation': 'Krause M, Gburek-Augustat J, Grafe D, Metzger R, Ginzel M, Griessenauer CJ, Grassner L, Weghuber D, Gradl J, Auer D, Schally T, Rund S, Kals C, Folie C, Bayer E, Gimona M, Rohde E. First-In-Human Application of Human Umbilical Cord-Derived Extracellular Vesicles in Tethered Spinal Cord Release Surgery. J Extracell Vesicles. 2025 Jun;14(6):e70104. doi: 10.1002/jev2.70104.'}, {'pmid': '29784434', 'type': 'BACKGROUND', 'citation': 'Vaquero J, Zurita M, Rico MA, Aguayo C, Fernandez C, Rodriguez-Boto G, Marin E, Tapiador N, Sevilla M, Carballido J, Vazquez D, Garcia-Olmo D, Guadalajara H, Leon M, Valverde I; Neurological Cell Therapy Group From Puerta De Hierro-Majadahonda Hospital. Cell therapy with autologous mesenchymal stromal cells in post-traumatic syringomyelia. Cytotherapy. 2018 Jun;20(6):796-805. doi: 10.1016/j.jcyt.2018.04.006. Epub 2018 May 18.'}, {'pmid': '38578425', 'type': 'BACKGROUND', 'citation': 'Li M, Wang X, Qi B, Cui S, Zheng T, Guan Y, Ma L, Liu S, Li Q, Chen Z, Jian F. Treatment of Syringomyelia Characterized by Focal Dilatation of the Central Canal Using Mesenchymal Stem Cells and Neural Stem Cells. Tissue Eng Regen Med. 2024 Jun;21(4):625-639. doi: 10.1007/s13770-024-00637-1. Epub 2024 Apr 5.'}]}, 'descriptionModule': {'briefSummary': 'This is a open-label, single-arm, dose escalation phase I clinical trial. The goal of this clinical trial is to evaluate the safety and preliminary efficacy of Intrathecal injection human umbilical cord-derived mesenchymal stromal cell-derived extracellular vesicle (hUC-MSC-sEV) in syringomyelia.', 'detailedDescription': 'This is a open-label, single-arm, dose escalation phase I clinical trial. The study will consist of one part: Part 1 will be a dose-escalation study.\n\nA traditional 3+3 dose-escalation design will be implemented in Part 1. Cohort 1 will receive low-dose1×1011particles; Cohort 2 will receive middle-dose2×1011particles; and Cohort 3 will receive high-dose3×1011particles. (Cohort 1 to Cohort 3 will receive a dose of 2 ml per lumbar puncture, administered once a month, for a total of three months.) If no dose-limiting toxicities (DLTs) are observed for 2 weeks after the administration of the first Intrathecal injection, a new cohort will be enrolled at the next planned dose level. If DLTs are observed in one participant in the cohort, an additional three participants will be treated at the same dose level. Dose escalation will be stopped if DLTs are observed in more than 33% of the participants.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT'], 'maximumAge': '60 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Patients whose MR revealed that syrinx have shrunk after surgery for a year but whose clinical spinal cord symptoms have not improved.\n* Patients whose MR revealed that syrinx spontaneously resolution under conservative observation, but whose clinical spinal cord symptoms have not improved.\n* Age: 18-60 years, inclusion of both genders;\n\nExclusion Criteria:\n\n* 1\\. Those with diabetes, heart disease, or impaired liver or kidney function; 2. Those with other diseases such as brainstem tumors and spinal cord tumors; 3. Infected individuals with lumbar puncture sites; 4. Those with previous or detected abnormalities in the heart; 5. Those who have autoimmune diseases and need to be treated with immunosuppressants; 6. Those who are allergic to the research drugs; 7. Have participated in other interventional clinical studies or received other cell therapies (excluding blood transfusion) within the past three months; 8. Those who are unconscious, unable to express subjective discomfort symptoms and unable to cooperate with neurological function tests, those who have been receiving drug treatment for a long time and do not cooperate with the treatment plan; 9. Pregnant women, women who are breastfeeding and those planning to become pregnant; 10. Serological tests (HBsAg, anti-HCV, anti-HIV, TP-Ab) are positive; 11. Other circumstances where the researcher deems the patient unsuitable to participate in this study (including but not limited to not meeting the treatment that benefits the patient the most, poor patient compliance, abnormal laboratory test indicators that cannot be accepted, etc.).\n\nRejection Criteria:\n\nmisdiagnosis; use of any medication that may significantly impact the assessment accuracy of hUC-MSC-sEV engraftment; absence of any evaluation outcome at any time point during the follow-up period\n\nCessation Criteria:\n\nindividual wishes of the subjects; occurrence of any hUC-MSC-sEV-associated serious adverse event (SAE) that may aggravate neurological dysfunction, or require prolongation of existing hospitalization, or need hospital readmission, or impair consciousness, or be life-threatening, or even lead to death in any subject; detection of any major mistake in the present protocol during the implementation of this clinical trial; the national administration agency requires the clinical trial to be halted'}, 'identificationModule': {'nctId': 'NCT07295067', 'briefTitle': 'Extracellular Vesicles for the Treatment of Syringomyelia', 'organization': {'class': 'OTHER', 'fullName': 'Xuanwu Hospital, Beijing'}, 'officialTitle': 'An Exploratory Study on the Use of Intrathecal Injection of Human Autologous Mesenchymal Stromal Cells Derived Extracellular Vesicles for the Treatment of Syringomyelia', 'orgStudyIdInfo': {'id': 'XWhUC-MSCs-EV'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Exosomes group', 'description': 'Patients in this arm will receive exosomes derived from human umbilical cord blood mesenchymal stem cells as a Intrathecal injection, administered once a month, for a total of three months.', 'interventionNames': ['Drug: Exosomes group']}], 'interventions': [{'name': 'Exosomes group', 'type': 'DRUG', 'description': 'Exosomes derived from human umbilical cord blood mesenchymal stem cells for Intrathecal injection (administered once a month, for a total of three months, based on the recommended dose during the dose-escalation phase).', 'armGroupLabels': ['Exosomes group']}]}, 'contactsLocationsModule': {'locations': [{'zip': '100032', 'city': 'Beijing', 'state': 'Beijing City', 'status': 'RECRUITING', 'country': 'China', 'contacts': [{'name': 'fengzeng jian', 'role': 'CONTACT', 'email': 'jianfengzeng@xwh.ccmu.edu.cn', 'phone': '01083198899'}], 'facility': 'Xuanwu Hospital', 'geoPoint': {'lat': 39.9075, 'lon': 116.39723}}], 'centralContacts': [{'name': 'fengzeng jian, md', 'role': 'CONTACT', 'email': 'jianfengzeng@xwh.ccmu.edu.cn', 'phone': '+861083198899'}, {'name': 'chenghua yuan', 'role': 'CONTACT', 'email': 'yuanchenghua@ccmu.edu.cn', 'phone': '+861083198899'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO', 'description': 'The study will not share individual participant data to other researchers.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Xuanwu Hospital, Beijing', 'class': 'OTHER'}, 'collaborators': [{'name': 'Yisaier Medical Technology (shan xi) Co., Ltd.', 'class': 'UNKNOWN'}], 'responsibleParty': {'type': 'SPONSOR'}}}}