Ignite Creation Date:
2025-12-24 @ 10:53 PM
Ignite Modification Date:
2026-02-21 @ 2:54 PM
Study NCT ID:
NCT03676569
Status:
COMPLETED
Last Update Posted:
2020-04-15
First Post:
2018-09-14
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Intrathecal Autologous ADRC Treatment of Autoimmune Refractory Epilepsy
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000069279', 'term': 'Drug Resistant Epilepsy'}], 'ancestors': [{'id': 'D004827', 'term': 'Epilepsy'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': 'Experimental clinical treatment'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 6}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2015-11-15', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2020-04', 'completionDateStruct': {'date': '2019-04-14', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-04-14', 'studyFirstSubmitDate': '2018-09-14', 'studyFirstSubmitQcDate': '2018-09-17', 'lastUpdatePostDateStruct': {'date': '2020-04-15', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2018-09-18', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2019-04-14', 'type': 'ACTUAL'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Radiological assesment', 'timeFrame': '12 months', 'description': 'MRI'}, {'measure': 'The level of antineural antibodies', 'timeFrame': '3 months', 'description': 'protein level, oligoclonal bands, IgG index as well as GluR3, VGKC complex/LGI1, GM1, NT-3, GAD, NMDAR antibodies from cerebrospinal fluid for evaluation the origin'}], 'primaryOutcomes': [{'measure': 'Rate of epileptic seizures', 'timeFrame': '3 months', 'description': 'recording of epileptic seizures frequency, EEG'}], 'secondaryOutcomes': [{'measure': 'Intelligence test', 'timeFrame': '3 months', 'description': 'The Leiter contains 10 subtests organized into four domains: Fluid Intelligence Visualization, Memory, Attention'}, {'measure': 'School progress', 'timeFrame': '3 months', 'description': 'teacher references'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['autoimmune epilepsy', 'refractory epilepsy', 'ADRC', 'mezenchymal stem cells', 'anti-neuronal antibody', 'Rasmussen encephality', 'adipose derived regenerative stem cells'], 'conditions': ['Refractory Epilepsy']}, 'referencesModule': {'references': [{'pmid': '32190059', 'type': 'RESULT', 'citation': 'Szczepanik E, Mierzewska H, Antczak-Marach D, Figiel-Dabrowska A, Terczynska I, Tryfon J, Krzesniak N, Noszczyk BH, Sawicka E, Domanska-Janik K, Sarnowska A. Intrathecal Infusion of Autologous Adipose-Derived Regenerative Cells in Autoimmune Refractory Epilepsy: Evaluation of Safety and Efficacy. Stem Cells Int. 2020 Jan 3;2020:7104243. doi: 10.1155/2020/7104243. eCollection 2020.'}]}, 'descriptionModule': {'briefSummary': 'Refractory epilepsies caused by an autoimmune mechanisms lead in children to progressive neurodegeneration. Immunomodulation therapy is effective only in a half of such cases. New approaches are undertaken. It was found that ADRC (adipose derived regenerative cells) isolated from adipose tissue consist mesenchymal stem cells that act as tissue repair cells. The purpose of this experimental study is to evaluate the possibility and safety of the use of multipotent mesenchymal adipose derived regenerative cells (ADRC) in patients diagnosed with an autoimmune determined refractory epilepsy.\n\nStudy protocol:\n\nIntrathecal infusions of autologous ADRC obtained after liposuction followed by isolation by Cytori system will be performed. Procedure will be repeated 3 times every 3 months in each patient. Neurological status, brain MRI, cognitive function and antiepileptic effect will be supervised during 24 months.', 'detailedDescription': 'Epilepsy is a chronic neurological disorder diagnosed in about 1% of the population, or c. 400,000 patients in Poland. Autoimmune refractory epilepsy is rare but most disabled epilepsy leading to developmental regression. The belief in a positive effect of ADRCs in drug-resistant epilepsy comes from test results showing the ability of mesenchymal cells to concentrate in damaged tissues, as well as their strong immunomodulating, especially anti-inflammatory, properties. Patients with drug resistant epilepsy show decreased numbers of neurons within the epilepsy zone, with most of these being hyperactive and malfunctioning. Additionally, an inflammatory active reaction in affected brain tissue is visible. ADRC-induced activation of endogenous neurogenesis may increase chances that the epilepsy zone will be reduced, and give a positive impact as regards the neuropsychiatric disorders commonly present in patients with epilepsy. Another common process accompanying recurring epilepsy attacks is active inflammation. Per the literature, ADRCs show strong neuroprotective, immunomodulating and antiapoptotic qualities and they may potentially reduce the frequency of epilepsy attacks. Thus, a clinical trial with ADRCs, addressed to drug-resistant epilepsy sufferers would be promising in controlling the epileptic seizures and coexisting behavioral / psychiatric symptoms. The goal is to improve the quality of life of the patients and their caregivers.\n\nIn patients who were diagnosed with autoimmune drug-resistant epilepsy and have met the set criteria and qualified to take part in the examination, after a formal written consent of their parents at the Clinic of Child Neurology IMC neurological examination, routine laboratory tests, EEG and neuropsychological assessment will be performed. ADRC will be obtained after liposuction and isolation with Cytori system.\n\nBefore intrathecal transplantation of ADRC suspension ( 5 ml), cerebrospinal fluid will be collected for evaluation of origin protein level, oligoclonal bands, the IgG index as well as GluR3, VGKC complex/LGI1, GM1, NT-3, GAD, and NMDAR antibodies. At the same time there will be an intake of 5ml of serum to evaluate levels of immunoglobulins IgA, IgM and IgG.\n\nProcedure will be repeated 3 times every 3 months in each patient. Neurological status, brain MRI, cognitive function and antiepileptic effect will be supervised during 24 months.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Drug-resistant epilepsy confirmed in anamnesis and diagnostic tests (EEG, MR)\n* Children between 3 and 18 years old\n* Presence of antineuronal antibody in serum or CSF\n* Rasmussen encephalitis (proven cellular immunity pathogenesis)\n* Probable autoimmune pathogenesis (autoimmune diseases in family, febrile infection associated refractory epilepsy)\n* Willing and able to provide written informed consent (patient or legal guardian)\n\nExclusion Criteria:\n\n* Refractory epilepsy with proven genetic or metabolic ethiology\n* Neoplastic disease\n* Active inflammatory process at the time of recruitment\n* Coagulation disorder\n* Status epilepticus\n* Participation in another clinical trial\n* Lack of willingness and ability to provide written informed consent'}, 'identificationModule': {'nctId': 'NCT03676569', 'acronym': 'EPIMSC', 'briefTitle': 'Intrathecal Autologous ADRC Treatment of Autoimmune Refractory Epilepsy', 'organization': {'class': 'OTHER', 'fullName': 'Mossakowski Medical Research Centre Polish Academy of Sciences'}, 'officialTitle': 'Intrathecal Autologous Adipose Derived Regenerative Cells Treatment of Autoimmune Determined Refractory Epilepsy - Evaluation of Safety and Efficacy', 'orgStudyIdInfo': {'id': '17/2014 IMiD'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Experimental group', 'description': 'Autologous ADRC transplantation in autoimmune refractory epilepsy', 'interventionNames': ['Biological: ADRC transplantation in autoimmune refractory epilepsy']}], 'interventions': [{'name': 'ADRC transplantation in autoimmune refractory epilepsy', 'type': 'BIOLOGICAL', 'armGroupLabels': ['Experimental group']}]}, 'contactsLocationsModule': {'overallOfficials': [{'name': 'Dorota Antczak-Marach, M.D.', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Institute of Mother and Child'}, {'name': 'Ewa Sawicka, Prof.', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Institute of Mother and Child'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Mossakowski Medical Research Centre Polish Academy of Sciences', 'class': 'OTHER'}, 'collaborators': [{'name': 'Institute of Mother and Child, Warsaw, Poland', 'class': 'OTHER'}], 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'M.D., Ph.D.; Head of Translative Platform for Regenerative Medicine MRC Mossakowski PAS', 'investigatorFullName': 'Anna Sarnowska', 'investigatorAffiliation': 'Mossakowski Medical Research Centre Polish Academy of Sciences'}}}}