Ignite Creation Date:
2025-12-24 @ 11:00 PM
Ignite Modification Date:
2026-01-01 @ 5:33 PM
Study NCT ID:
NCT03351569
Status:
UNKNOWN
Last Update Posted:
2017-11-27
First Post:
2017-10-27
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Intravenous Immunoglobulin for Unverricht-Lundborg Disease.
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D020194', 'term': 'Unverricht-Lundborg Syndrome'}, {'id': 'D020191', 'term': 'Myoclonic Epilepsies, Progressive'}], 'ancestors': [{'id': 'D004831', 'term': 'Epilepsies, Myoclonic'}, {'id': 'D004829', 'term': 'Epilepsy, Generalized'}, {'id': 'D004827', 'term': 'Epilepsy'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D000073376', 'term': 'Epileptic Syndromes'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D016756', 'term': 'Immunoglobulins, Intravenous'}], 'ancestors': [{'id': 'D007074', 'term': 'Immunoglobulin G'}, {'id': 'D007132', 'term': 'Immunoglobulin Isotypes'}, {'id': 'D000906', 'term': 'Antibodies'}, {'id': 'D007136', 'term': 'Immunoglobulins'}, {'id': 'D007162', 'term': 'Immunoproteins'}, {'id': 'D001798', 'term': 'Blood Proteins'}, {'id': 'D011506', 'term': 'Proteins'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}, {'id': 'D012712', 'term': 'Serum Globulins'}, {'id': 'D005916', 'term': 'Globulins'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR'], 'maskingDescription': 'Drug and placebo had the same appearance and are wrapped in foil paper before being shown to the patient.\n\nOutcome evaluation is carried out by personnel not involved in the treatment at a distant site, one month after treatment.'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'CROSSOVER', 'interventionModelDescription': 'sigle patient trial'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 1}}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2015-12-09', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2017-11', 'completionDateStruct': {'date': '2017-12-30', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2017-11-22', 'studyFirstSubmitDate': '2017-10-27', 'studyFirstSubmitQcDate': '2017-11-21', 'lastUpdatePostDateStruct': {'date': '2017-11-27', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2017-11-24', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2016-12-06', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Improvement of at least 20% of the action myoclonus at one year, measured with section 4 (Action Myoclonus) of the Unified Myoclonus Rating Scale.', 'timeFrame': 'monthly for one year', 'description': 'The range for Action Myoclonus Score is 0 (best) - 160 (worst, , i.e. more severe involuntary movements). Percent change = 100 X (Placebo UMRS4 - Treatment UMRS4) / Placebo UMRS4).'}], 'secondaryOutcomes': [{'measure': 'Unified Myoclonus Rating Scale (UMRS) overall score improvement.', 'timeFrame': 'monthly for one year', 'description': 'The total value of the UMRS (range from 0 - best - to 365 - worst) is composed of the sum of 6 sections: (1) Patient Questionnaire (range 0-48), (2) Myoclonus at rest (range 0-108), (3) Stimulus Sensitivity (range 0-17), (4) Myoclonus with Action (range 0-160), (5) Functional Tests (0-28), (6) Global Disability Score (range 0-4).'}, {'measure': "Patient's preference", 'timeFrame': 'one year', 'description': "The program was to discuss the patient's analysis data with the patient himself and to let him decide in three possible ways: (1) to continue the trial, (2) to continue treatment with immunoglobulins, (3) to suspend the treatment.The choice number 2 is considered a favorable outcome."}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Unverricht-Lundborg disease', 'progressive myoclonus epilepsy', 'pharmacoresistant epilepsy'], 'conditions': ['Unverricht-Lundborg Disease']}, 'referencesModule': {'references': [{'pmid': '21695041', 'type': 'BACKGROUND', 'citation': 'Lillie EO, Patay B, Diamant J, Issell B, Topol EJ, Schork NJ. The n-of-1 clinical trial: the ultimate strategy for individualizing medicine? Per Med. 2011 Mar;8(2):161-173. doi: 10.2217/pme.11.7.'}, {'pmid': '2384914', 'type': 'BACKGROUND', 'citation': 'Guatelli JC, Gingeras TR, Richman DD. Alternative splice acceptor utilization during human immunodeficiency virus type 1 infection of cultured cells. J Virol. 1990 Sep;64(9):4093-8. doi: 10.1128/JVI.64.9.4093-4098.1990.'}]}, 'descriptionModule': {'briefSummary': 'Single patient randomized double blind trial to assess whether intravenous immunoglobulin can improve the clinical outcome of a case suffering from Unverricht-Lundborg disease.', 'detailedDescription': "Single patient randomized double blind trial to assess whether intravenous immunoglobulin can improve the clinical outcome of a case suffering from Unverricht-Lundborg disease (clinical and genetic diagnosis).\n\nThe patient was randomized to be treated with intravenous immunoglobulin or placebo 1:1 (crossover) once a month for at least one year.\n\nMain objective: improvement of the action myoclonus. Secondary objectives: Improvement in the overall score and in individual sections of the Unified Myoclonus Rating Scale at one year; patient preferences based on results at the end of the trial.\n\nThe first analyst was scheduled at one year from the start of the trial. The program was to discuss the patient's analysis data and to let the patient decide in three possible ways: to continue the trial, to continue treatment with immunoglobulins, to suspend the treatment. Depending on the decision, it was planned to follow the patient throughout the year after the analysis, at least for one year."}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['ADULT'], 'maximumAge': '25 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Malattia di Unverricht-Lundborg (genetic diagnosis)\n\nExclusion Criteria:\n\n* Contraindications to intravenous immunoglobulin'}, 'identificationModule': {'nctId': 'NCT03351569', 'briefTitle': 'Intravenous Immunoglobulin for Unverricht-Lundborg Disease.', 'organization': {'class': 'OTHER', 'fullName': 'Azienda Socio Sanitaria Territoriale di Mantova'}, 'officialTitle': 'Intravenous Immunoglobulin for Unverricht-Lundborg Disease: Single-patient Trial.', 'orgStudyIdInfo': {'id': '200200'}, 'secondaryIdInfos': [{'id': '2017-002147-15', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Immunoglobulin', 'description': 'Intravenous immunoglobulin 25 grams (five 100 ml bottles, 5g/100ml), in 3 hours, once a month for one year.', 'interventionNames': ['Drug: Intravenous immunoglobulin']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Saline solution', 'description': 'Intravenous saline solution 500 ml (five 100 ml bottles), in 3 hours, once a month for one year.', 'interventionNames': ['Drug: Intravenous immunoglobulin']}], 'interventions': [{'name': 'Intravenous immunoglobulin', 'type': 'DRUG', 'otherNames': ['Venital'], 'description': 'Intravenous drip.', 'armGroupLabels': ['Immunoglobulin', 'Saline solution']}]}, 'ipdSharingStatementModule': {'ipdSharing': 'YES', 'description': 'We planned to publish the results of this single patient trial.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Azienda Socio Sanitaria Territoriale di Mantova', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'MD', 'investigatorFullName': 'Alfonso Ciccone', 'investigatorAffiliation': 'Azienda Socio Sanitaria Territoriale di Mantova'}}}}