Ignite Creation Date:
2025-12-24 @ 6:36 PM
Ignite Modification Date:
2025-12-28 @ 7:00 AM
Study NCT ID:
NCT01441557
Status:
COMPLETED
Last Update Posted:
2025-09-19
First Post:
2011-09-26
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Pilot Study on the Usefulness of 3,4-diaminopyridine in the Treatment of Botulism
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D001906', 'term': 'Botulism'}], 'ancestors': [{'id': 'D003015', 'term': 'Clostridium Infections'}, {'id': 'D016908', 'term': 'Gram-Positive Bacterial Infections'}, {'id': 'D001424', 'term': 'Bacterial Infections'}, {'id': 'D001423', 'term': 'Bacterial Infections and Mycoses'}, {'id': 'D007239', 'term': 'Infections'}, {'id': 'D020511', 'term': 'Neuromuscular Junction Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D020258', 'term': 'Neurotoxicity Syndromes'}, {'id': 'D005517', 'term': 'Foodborne Diseases'}, {'id': 'D011041', 'term': 'Poisoning'}, {'id': 'D064419', 'term': 'Chemically-Induced Disorders'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D000077770', 'term': 'Amifampridine'}], 'ancestors': [{'id': 'D015761', 'term': '4-Aminopyridine'}, {'id': 'D000631', 'term': 'Aminopyridines'}, {'id': 'D000588', 'term': 'Amines'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D011725', 'term': 'Pyridines'}, {'id': 'D006573', 'term': 'Heterocyclic Compounds, 1-Ring'}, {'id': 'D006571', 'term': 'Heterocyclic Compounds'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2', 'PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'OTHER', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 3}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2011-09'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-09', 'completionDateStruct': {'date': '2012-03', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-09-16', 'studyFirstSubmitDate': '2011-09-26', 'studyFirstSubmitQcDate': '2011-09-26', 'lastUpdatePostDateStruct': {'date': '2025-09-19', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2011-09-27', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2012-02', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'electrophysiological', 'timeFrame': '90 min', 'description': 'Primary endpoint:\n\nClinical, electrophysiological and respiratory before and after administration of 3,4-diaminopyridine.'}]}, 'conditionsModule': {'keywords': ['mechanical ventilation'], 'conditions': ['Botulism']}, 'referencesModule': {'references': [{'pmid': '24007658', 'type': 'RESULT', 'citation': 'Friggeri A, Marcon F, Marciniak S, Lemaire-Hurtel AS, Seydi A, Ammenouche N, Levrard M, Mahjoub Y, Airapetian N, Tinturier F, Petitjean M, Dupont H. 3,4-Diaminopyridine may improve neuromuscular block during botulism. Crit Care. 2013 Sep 5;17(5):449. doi: 10.1186/cc12880. No abstract available.'}]}, 'descriptionModule': {'briefSummary': 'Main objectives:\n\nEvaluate the effectiveness of an administration of 3,4-diaminopyridine (FIRDAPSE ®) in severe botulinic poisoning in measuring the effect on electrophysiological and respiratory parameters\n\nSecondary Objective:\n\nStudy the natural history of electrophysiological and respiratory parameters during the botulinic intoxication\n\nPrimary endpoint:\n\nClinical, electrophysiological and respiratory before and after administration of 3,4-diaminopyridine.\n\nStudy Design:\n\nPilot study, prospective, interventional.\n\nStudy population:\n\nCase series (n = 8 patients) suffering from botulinic type A, respiratory failure, but with no other organ failure\n\nExperimental treatment :\n\n3,4-diaminopyridine, FIRDAPSE ® (BioMarin) The dosage will be gradually increased according to a predetermined scheme and will not exceed 60 mg / day and 20 mg / dose.\n\nStatistics:\n\nIntra-individual comparison of physiological parameters measured before and after administration of 3,4-diaminopyridine. Electromyographic and respiratory parameters will be measured for each patient. Then a dose of 10 mg of 3,4-diaminopyridine will be administrated. If this dose is well tolerated and provides a relative improvement of 10% for at least one of the parameters studied, the dose will be maintained at 10 mg for 48 hours 3 times a day then increased to 20 mg.\n\nThe primary endpoint is the change in the amplitude of muscle response evaluated by the subtraction of amplitude at T1.5 and T0.', 'detailedDescription': 'Main objectives:\n\nEvaluate the effectiveness of an administration of 3,4-diaminopyridine (FIRDAPSE ®) in severe botulinic poisoning in measuring the effect on electrophysiological and respiratory parameters\n\nSecondary Objective:\n\nStudy the natural history of electrophysiological and respiratory parameters during the botulinic intoxication\n\nPrimary endpoint:\n\nClinical, electrophysiological and respiratory before and after administration of 3,4-diaminopyridine.\n\nStudy Design:\n\nPilot study, prospective, interventional.\n\nStudy population:\n\nCase series (n = 8 patients) suffering from botulinic type A, respiratory failure, but with no other organ failure\n\nExperimental treatment :\n\n3,4-diaminopyridine, FIRDAPSE ® (BioMarin) The dosage will be gradually increased according to a predetermined scheme and will not exceed 60 mg / day and 20 mg / dose.\n\nStatistics:\n\nIntra-individual comparison of physiological parameters measured before and after administration of 3,4-diaminopyridine. Electromyographic and respiratory parameters will be measured for each patient. Then a dose of 10 mg of 3,4-diaminopyridine will be administrated. If this dose is well tolerated and provides a relative improvement of 10% for at least one of the parameters studied, the dose will be maintained at 10 mg for 48 hours 3 times a day then increased to 20 mg.\n\nThe primary endpoint is the change in the amplitude of muscle response evaluated by the subtraction of amplitude at T1.5 and T0.\n\nQuantitative variables are expressed as mean ± standard deviation or median (minimum - maximum) and qualitative variables as percentages.\n\nThe null hypothesis (the evolution is identical between the two doses) will be rejected in favour of the alternative hypothesis (the evolution is different) using the mixed model analysis of variance on the threshold of 5% for the first kind with adjusting the amplitude at T0, age, the time between patient admission and the first administration of 3,4-diaminopyridine. The evolution of latency and respiratory parameters will be analyzed with the same mixed ANOVA without risk adjustment alpha.\n\nFor the secondary objective, the evolution curves of the amplitude of muscle response on the one hand and respiratory function on the other hand, will be built for each patient.\n\nThe mean (or median) of amplitude and respiratory function at weaning will be calculated with a confidence level of 95% (or range). The average time between the first 3,4-diaminopyridine administration and withdrawal of mechanical ventilation will also be calculated.\n\nFor each dose, the average evolution of the amplitude will be calculated with a confidence level of 95%.\n\nStatistical analysis will be carried out using SAS software version 9.2.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* botulism\n* ventilation\n\nExclusion Criteria:\n\n* renal failure\n* cardiac failure\n* pregnancy\n* under age 18\n* hepatic failure'}, 'identificationModule': {'nctId': 'NCT01441557', 'briefTitle': 'Pilot Study on the Usefulness of 3,4-diaminopyridine in the Treatment of Botulism', 'organization': {'class': 'OTHER', 'fullName': 'Centre Hospitalier Universitaire, Amiens'}, 'officialTitle': 'Pilot Study on the Usefulness of 3,4-diaminopyridine in the Treatment of Botulism', 'orgStudyIdInfo': {'id': 'PI11-PR-DUPONT'}}, 'armsInterventionsModule': {'interventions': [{'name': '3,4-diaminopyridine', 'type': 'DRUG', 'description': 'test dose 10 mg then 20 mg, then depending on tolerance and efficacy full-dose administration(from 10mg /8h to 20 mg /8h)'}]}, 'contactsLocationsModule': {'locations': [{'zip': '80000', 'city': 'Amiens', 'country': 'France', 'facility': 'CHU', 'geoPoint': {'lat': 49.9, 'lon': 2.3}}], 'overallOfficials': [{'name': 'Herve DUPONT, Md, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'CHU Amiens'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Centre Hospitalier Universitaire, Amiens', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}