{ "FullStudy":{ "Rank":218158, "Study":{ "ProtocolSection":{ "IdentificationModule":{ "NCTId":"NCT01511978", "OrgStudyIdInfo":{ "OrgStudyId":"JPC 3,4-DAPPER" }, "Organization":{ "OrgFullName":"Jacobus Pharmaceutical", "OrgClass":"INDUSTRY" }, "BriefTitle":"Effectiveness of 3,4-Diaminopyridine in Lambert-Eaton Myasthenic Syndrome", "OfficialTitle":"Inpatient Double-Blind Placebo-Controlled Withdrawal Study of 3,4-Diaminopyridine Base (3,4-DAP) in Subjects With Known Lambert-Eaton Myasthenic Syndrome", "Acronym":"DAPPER" }, "StatusModule":{ "StatusVerifiedDate":"July 2017", "OverallStatus":"Completed", "StartDateStruct":{ "StartDate":"January 2012" }, "PrimaryCompletionDateStruct":{ "PrimaryCompletionDate":"February 2014", "PrimaryCompletionDateType":"Actual" }, "CompletionDateStruct":{ "CompletionDate":"July 2015", "CompletionDateType":"Actual" }, "StudyFirstSubmitDate":"December 24, 2011", "StudyFirstSubmitQCDate":"January 13, 2012", "StudyFirstPostDateStruct":{ "StudyFirstPostDate":"January 19, 2012", "StudyFirstPostDateType":"Estimate" }, "ResultsFirstSubmitDate":"June 7, 2017", "ResultsFirstSubmitQCDate":"July 7, 2017", "ResultsFirstPostDateStruct":{ "ResultsFirstPostDate":"July 11, 2017", "ResultsFirstPostDateType":"Actual" }, "LastUpdateSubmitDate":"July 7, 2017", "LastUpdatePostDateStruct":{ "LastUpdatePostDate":"July 11, 2017", "LastUpdatePostDateType":"Actual" } }, "SponsorCollaboratorsModule":{ "ResponsibleParty":{ "ResponsiblePartyType":"Sponsor" }, "LeadSponsor":{ "LeadSponsorName":"Jacobus Pharmaceutical", "LeadSponsorClass":"INDUSTRY" } }, "OversightModule":{ "OversightHasDMC":"No", "IsFDARegulatedDrug":"Yes", "IsFDARegulatedDevice":"No" }, "DescriptionModule":{ "BriefSummary":"Hypothesis: 3,4-Diaminopyridine base (3,4-DAP) improves Lambert-Eaton Myasthenic Syndrome (LEMS)-related weakness.", "DetailedDescription":"The objectives of the study were to confirm the safety and to test the efficacy of 3,4-DAP in the treatment of LEMS-related weakness.\n\nThis was a phase 2 randomized double-blind placebo-controlled withdrawal study in subjects with known clinically active LEMS who had been on a chronic stable dose of compassionate distribution Jacobus 3,4-DAP provided through FDA-approved individual investigator-held INDs." }, "ConditionsModule":{ "ConditionList":{ "Condition":[ "Lambert-Eaton Myasthenic Syndrome", "Eaton-Lambert Myasthenic Syndrome" ] }, "KeywordList":{ "Keyword":[ "Lambert-Eaton", "Eaton-Lambert", "myasthenia", "myasthenic", "LEMS", "LES", "DAP", "diaminopyridine", "3,4-diaminopyridine", "3,4-DAP" ] } }, "DesignModule":{ "StudyType":"Interventional", "PhaseList":{ "Phase":[ "Phase 2" ] }, "DesignInfo":{ "DesignAllocation":"Randomized", "DesignInterventionModel":"Parallel Assignment", "DesignInterventionModelDescription":"This was a multicenter, randomized, double-blind, placebo-controlled withdrawal study to assess the safety and efficacy of 3,4-DAP in subjects on a stable regimen of all LEMS-related treatments, including 3,4-DAP, for a minimum of 3 months prior to study entry. Subjects who met all study entry criteria were randomized in a 1:1 ratio to continue their current treatment regimen (Group A, continuous 3,4-DAP) or tapered withdrawal from 3,4-DAP (Group B, taper to placebo).", "DesignPrimaryPurpose":"Treatment", "DesignMaskingInfo":{ "DesignMasking":"Quadruple", "DesignWhoMaskedList":{ "DesignWhoMasked":[ "Participant", "Care Provider", "Investigator", "Outcomes Assessor" ] } } }, "EnrollmentInfo":{ "EnrollmentCount":"32", "EnrollmentType":"Actual" } }, "ArmsInterventionsModule":{ "ArmGroupList":{ "ArmGroup":[ { "ArmGroupLabel":"Continuous 3,4-DAP", "ArmGroupType":"Active Comparator", "ArmGroupDescription":"Subjects continued taking their usual individualized regimen of 3,4-DAP base, 30 to 100 mg daily divided into at least 3 doses.", "ArmGroupInterventionList":{ "ArmGroupInterventionName":[ "Drug: Continuous 3,4-DAP" ] } },{ "ArmGroupLabel":"Taper 3,4-DAP to Placebo", "ArmGroupType":"Placebo Comparator", "ArmGroupDescription":"Subjects were tapered over 3 days from their usual individualized regimen of 3,4-DAP base (30 to 100 mg daily divided into at least 3 doses) to placebo with up to an additional 16 hours of placebo before resuming their usual pre-study regimen of 3,4-DAP base", "ArmGroupInterventionList":{ "ArmGroupInterventionName":[ "Drug: Taper 3,4-DAP to Placebo" ] } } ] }, "InterventionList":{ "Intervention":[ { "InterventionType":"Drug", "InterventionName":"Continuous 3,4-DAP", "InterventionDescription":"Subjects were maintained on their usual personal dose and schedule of 3,4-DAP base", "InterventionArmGroupLabelList":{ "InterventionArmGroupLabel":[ "Continuous 3,4-DAP" ] }, "InterventionOtherNameList":{ "InterventionOtherName":[ "3,4-Diaminopyridine", "3,4-Pyridinediamine", "Diamino-3,4-pyridine" ] } },{ "InterventionType":"Drug", "InterventionName":"Taper 3,4-DAP to Placebo", "InterventionDescription":"Subjects were tapered over 3 days from their usual regimen of 3,4-DAP base to placebo with up to an additional 16 hours of placebo before resuming their usual pre-study regimen of 3,4-DAP base", "InterventionArmGroupLabelList":{ "InterventionArmGroupLabel":[ "Taper 3,4-DAP to Placebo" ] }, "InterventionOtherNameList":{ "InterventionOtherName":[ "3,4-Diaminopyridine", "3,4-Pyridinediamine", "Diamino-3,4-pyridine", "Placebo" ] } } ] } }, "OutcomesModule":{ "PrimaryOutcomeList":{ "PrimaryOutcome":[ { "PrimaryOutcomeMeasure":"Number of Participants With 30% or More Deterioration in Triple Timed Up & Go (3TUG) Test, Compared to Time-matched Baseline", "PrimaryOutcomeDescription":"The 3TUG time obtained 2 hours after the last dose of the withdrawal period (i.e., at time of theoretical \"peak drug effect\") was compared to the average time-matched 3TUG tests performed during 2 days of baseline observation prior to randomization.\n\nThe study endpoint was a change of more than 30% in the final post-dose 3TUG during the withdrawal period and was based on blinded readings of video recordings of 3TUG tests.", "PrimaryOutcomeTimeFrame":"Baseline period (days 0, 1, 2); Randomized treatment period (starting with last dose of day 2, and days 3, 4, 5, and ending with first dose on day 6 when pre-randomization regimen was resumed, or rescue, if indicated sooner)" } ] }, "SecondaryOutcomeList":{ "SecondaryOutcome":[ { "SecondaryOutcomeMeasure":"Self-assessment of LEMS-related Weakness, W-SAS", "SecondaryOutcomeDescription":"The last post-dose self-assessment of LEMS-related weakness from the withdrawal period with categories of much much weaker (-3), much weaker (-2), somewhat weaker (-1), about the same (0), somewhat stronger (1), much stronger (2), and much much stronger (3).", "SecondaryOutcomeTimeFrame":"Participants were followed for up to 7 days" } ] } }, "EligibilityModule":{ "EligibilityCriteria":"Inclusion Criteria:\n\nAge 18 or over\nAmbulatory while taking 3,4-DAP, i.e. the patient was able to perform the timed up and go (TUG), either with or without an assistive device\nEstablished diagnosis of LEMS, with documentation provided\nContinuous use of Jacobus 3,4-DAP for at least 3 months\nMinimum of 3 doses per day with no single dose less than 10 mg of 3,4-DAP\nThe patient needed to wait about 15 to 30 minutes to experience an unequivocal improvement in a LEMS-induced dysfunction after they take their first dose of 3,4-DAP in the morning [a patient who remains in bed past this point by choice may still be eligible]\nStable regimen of all LEMS-related treatments for at least 3 months\nStable daily regimen of other medications (prescription and over-the-counter) for a minimum of 1 month\nWilling to chance being tapered off of 3,4-DAP\nFluency in English\nIf applicable, agreed to use birth control during heterosexual intercourse until at least 2 weeks after completion of study\nA signed informed consent by the study subject\n\nExclusion Criteria:\n\nLast monoclonal antibody treatment (e.g. rituximab) was less than 6 months ago (i.e., recent treatment is an exclusion)\nClinically significant or poorly controlled condition that in the opinion of the study personnel might pose an unacceptable risk to the patient if entered into the study\nRespiratory failure requiring intubation while on 3,4-DAP with no precipitating event or medication\nUse of any investigational drug other than 3,4-DAP within the last 30 days\nPregnant or lactating\nCurrent use of other aminopyridines (e.g.4-AP) or guanidine\nDid not display a sufficiently large response to 3,4-DAP during the baseline observation period in the CRU to detect a decline during withdrawal of 3,4-DAP", "HealthyVolunteers":"No", "Gender":"All", "MinimumAge":"18 Years", "StdAgeList":{ "StdAge":[ "Adult", "Older Adult" ] } }, "ContactsLocationsModule":{ "OverallOfficialList":{ "OverallOfficial":[ { "OverallOfficialName":"Kathy L Aleš, MD", "OverallOfficialAffiliation":"Jacobus Pharmaceutical", "OverallOfficialRole":"Study Director" } ] }, "LocationList":{ "Location":[ { "LocationFacility":"University of California at Davis", "LocationCity":"Sacramento", "LocationState":"California", "LocationZip":"95817", "LocationCountry":"United States" },{ "LocationFacility":"Indiana University", "LocationCity":"Indianapolis", "LocationState":"Indiana", "LocationZip":"46202", "LocationCountry":"United States" },{ "LocationFacility":"Duke University", "LocationCity":"Durham", "LocationState":"North Carolina", "LocationZip":"27710", "LocationCountry":"United States" },{ "LocationFacility":"Oregon Health & Science University", "LocationCity":"Portland", "LocationState":"Oregon", "LocationZip":"97239", "LocationCountry":"United States" },{ "LocationFacility":"Vanderbilt University Medical Center", "LocationCity":"Nashville", "LocationState":"Tennessee", "LocationZip":"37232", "LocationCountry":"United States" },{ "LocationFacility":"Baylor College of Medicine", "LocationCity":"Houston", "LocationState":"Texas", "LocationZip":"77030", "LocationCountry":"United States" },{ "LocationFacility":"University of Utah", "LocationCity":"Salt Lake City", "LocationState":"Utah", "LocationZip":"84132", "LocationCountry":"United States" } ] } }, "IPDSharingStatementModule":{ "IPDSharing":"Undecided" } }, "ResultsSection":{ "ParticipantFlowModule":{ "FlowPreAssignmentDetails":"52 patients were assessed for eligibility. 20 were ineligible. 32 were randomized and completed the study.", "FlowRecruitmentDetails":"Participants were referred by active Investigational New Drug (IND) holders involved with the Jacobus Pharmaceutical Company's 3,4-diaminopyridine (3,4-DAP) free base compassionate distribution program.", "FlowGroupList":{ "FlowGroup":[ { "FlowGroupId":"FG000", "FlowGroupTitle":"Continuous 3,4-DAP", "FlowGroupDescription":"Subjects were administered their usual dosage on their regular personalized schedule" },{ "FlowGroupId":"FG001", "FlowGroupTitle":"Taper 3,4-DAP to Placebo", "FlowGroupDescription":"Subjects were administered decreasing amounts of 3,4-DAP on their regular personalized schedule" } ] }, "FlowPeriodList":{ "FlowPeriod":[ { "FlowPeriodTitle":"Overall Study", "FlowMilestoneList":{ "FlowMilestone":[ { "FlowMilestoneType":"STARTED", "FlowAchievementList":{ "FlowAchievement":[ { "FlowAchievementGroupId":"FG000", "FlowAchievementNumSubjects":"14" },{ "FlowAchievementGroupId":"FG001", "FlowAchievementNumSubjects":"18" } ] } },{ "FlowMilestoneType":"COMPLETED", "FlowAchievementList":{ "FlowAchievement":[ { "FlowAchievementGroupId":"FG000", "FlowAchievementNumSubjects":"14" },{ "FlowAchievementGroupId":"FG001", "FlowAchievementNumSubjects":"18" } ] } },{ "FlowMilestoneType":"NOT COMPLETED", "FlowAchievementList":{ "FlowAchievement":[ { "FlowAchievementGroupId":"FG000", "FlowAchievementNumSubjects":"0" },{ "FlowAchievementGroupId":"FG001", "FlowAchievementNumSubjects":"0" } ] } } ] } } ] } }, "BaselineCharacteristicsModule":{ "BaselineGroupList":{ "BaselineGroup":[ { "BaselineGroupId":"BG000", "BaselineGroupTitle":"Continuous 3,4-Diaminopyridine (3,4-DAP)", "BaselineGroupDescription":"Subjects were administered their usual dosage on their regular personalized schedule" },{ "BaselineGroupId":"BG001", "BaselineGroupTitle":"3,4-DAP Taper to Placebo", "BaselineGroupDescription":"Subjects were administered decreasing amounts of 3,4-DAP on their regular personalized schedule" },{ "BaselineGroupId":"BG002", "BaselineGroupTitle":"Total", "BaselineGroupDescription":"Total of all reporting groups" } ] }, "BaselineDenomList":{ "BaselineDenom":[ { "BaselineDenomUnits":"Participants", "BaselineDenomCountList":{ "BaselineDenomCount":[ { "BaselineDenomCountGroupId":"BG000", "BaselineDenomCountValue":"14" },{ "BaselineDenomCountGroupId":"BG001", "BaselineDenomCountValue":"18" },{ "BaselineDenomCountGroupId":"BG002", "BaselineDenomCountValue":"32" } ] } } ] }, "BaselineMeasureList":{ "BaselineMeasure":[ { "BaselineMeasureTitle":"Age, Continuous", "BaselineMeasureParamType":"Mean", "BaselineMeasureDispersionType":"Standard Deviation", "BaselineMeasureUnitOfMeasure":"years", "BaselineClassList":{ "BaselineClass":[ { "BaselineCategoryList":{ "BaselineCategory":[ { "BaselineMeasurementList":{ "BaselineMeasurement":[ { "BaselineMeasurementGroupId":"BG000", "BaselineMeasurementValue":"50.7", "BaselineMeasurementSpread":"15.97" },{ "BaselineMeasurementGroupId":"BG001", "BaselineMeasurementValue":"59.3", "BaselineMeasurementSpread":"14.99" },{ "BaselineMeasurementGroupId":"BG002", "BaselineMeasurementValue":"55.5", "BaselineMeasurementSpread":"15.77" } ] } } ] } } ] } },{ "BaselineMeasureTitle":"Sex: Female, Male", "BaselineMeasureParamType":"Count of Participants", "BaselineMeasureUnitOfMeasure":"Participants", "BaselineClassList":{ "BaselineClass":[ { "BaselineCategoryList":{ "BaselineCategory":[ { "BaselineCategoryTitle":"Female", "BaselineMeasurementList":{ "BaselineMeasurement":[ { "BaselineMeasurementGroupId":"BG000", "BaselineMeasurementValue":"10" },{ "BaselineMeasurementGroupId":"BG001", "BaselineMeasurementValue":"11" },{ "BaselineMeasurementGroupId":"BG002", "BaselineMeasurementValue":"21" } ] } },{ "BaselineCategoryTitle":"Male", "BaselineMeasurementList":{ "BaselineMeasurement":[ { "BaselineMeasurementGroupId":"BG000", "BaselineMeasurementValue":"4" },{ "BaselineMeasurementGroupId":"BG001", "BaselineMeasurementValue":"7" },{ "BaselineMeasurementGroupId":"BG002", "BaselineMeasurementValue":"11" } ] } } ] } } ] } },{ "BaselineMeasureTitle":"Ethnicity (NIH/OMB)", "BaselineMeasureParamType":"Count of Participants", "BaselineMeasureUnitOfMeasure":"Participants", "BaselineClassList":{ "BaselineClass":[ { "BaselineCategoryList":{ "BaselineCategory":[ { "BaselineCategoryTitle":"Hispanic or Latino", "BaselineMeasurementList":{ "BaselineMeasurement":[ { "BaselineMeasurementGroupId":"BG000", "BaselineMeasurementValue":"0" },{ "BaselineMeasurementGroupId":"BG001", "BaselineMeasurementValue":"1" },{ "BaselineMeasurementGroupId":"BG002", "BaselineMeasurementValue":"1" } ] } },{ "BaselineCategoryTitle":"Not Hispanic or Latino", "BaselineMeasurementList":{ "BaselineMeasurement":[ { "BaselineMeasurementGroupId":"BG000", "BaselineMeasurementValue":"14" },{ "BaselineMeasurementGroupId":"BG001", "BaselineMeasurementValue":"17" },{ "BaselineMeasurementGroupId":"BG002", "BaselineMeasurementValue":"31" } ] } },{ "BaselineCategoryTitle":"Unknown or Not Reported", "BaselineMeasurementList":{ "BaselineMeasurement":[ { "BaselineMeasurementGroupId":"BG000", "BaselineMeasurementValue":"0" },{ "BaselineMeasurementGroupId":"BG001", "BaselineMeasurementValue":"0" },{ "BaselineMeasurementGroupId":"BG002", "BaselineMeasurementValue":"0" } ] } } ] } } ] } },{ "BaselineMeasureTitle":"Race (NIH/OMB)", "BaselineMeasureParamType":"Count of Participants", "BaselineMeasureUnitOfMeasure":"Participants", "BaselineClassList":{ "BaselineClass":[ { "BaselineCategoryList":{ "BaselineCategory":[ { "BaselineCategoryTitle":"American Indian or Alaska Native", "BaselineMeasurementList":{ "BaselineMeasurement":[ { "BaselineMeasurementGroupId":"BG000", "BaselineMeasurementValue":"0" },{ "BaselineMeasurementGroupId":"BG001", "BaselineMeasurementValue":"0" },{ "BaselineMeasurementGroupId":"BG002", "BaselineMeasurementValue":"0" } ] } },{ "BaselineCategoryTitle":"Asian", "BaselineMeasurementList":{ "BaselineMeasurement":[ { "BaselineMeasurementGroupId":"BG000", "BaselineMeasurementValue":"0" },{ "BaselineMeasurementGroupId":"BG001", "BaselineMeasurementValue":"0" },{ "BaselineMeasurementGroupId":"BG002", "BaselineMeasurementValue":"0" } ] } },{ "BaselineCategoryTitle":"Native Hawaiian or Other Pacific Islander", "BaselineMeasurementList":{ "BaselineMeasurement":[ { "BaselineMeasurementGroupId":"BG000", "BaselineMeasurementValue":"0" },{ "BaselineMeasurementGroupId":"BG001", "BaselineMeasurementValue":"0" },{ "BaselineMeasurementGroupId":"BG002", "BaselineMeasurementValue":"0" } ] } },{ "BaselineCategoryTitle":"Black or African American", "BaselineMeasurementList":{ "BaselineMeasurement":[ { "BaselineMeasurementGroupId":"BG000", "BaselineMeasurementValue":"3" },{ "BaselineMeasurementGroupId":"BG001", "BaselineMeasurementValue":"0" },{ "BaselineMeasurementGroupId":"BG002", "BaselineMeasurementValue":"3" } ] } },{ "BaselineCategoryTitle":"White", "BaselineMeasurementList":{ "BaselineMeasurement":[ { "BaselineMeasurementGroupId":"BG000", "BaselineMeasurementValue":"11" },{ "BaselineMeasurementGroupId":"BG001", "BaselineMeasurementValue":"18" },{ "BaselineMeasurementGroupId":"BG002", "BaselineMeasurementValue":"29" } ] } },{ "BaselineCategoryTitle":"More than one race", "BaselineMeasurementList":{ "BaselineMeasurement":[ { "BaselineMeasurementGroupId":"BG000", "BaselineMeasurementValue":"0" },{ "BaselineMeasurementGroupId":"BG001", "BaselineMeasurementValue":"0" },{ "BaselineMeasurementGroupId":"BG002", "BaselineMeasurementValue":"0" } ] } },{ "BaselineCategoryTitle":"Unknown or Not Reported", "BaselineMeasurementList":{ "BaselineMeasurement":[ { "BaselineMeasurementGroupId":"BG000", "BaselineMeasurementValue":"0" },{ "BaselineMeasurementGroupId":"BG001", "BaselineMeasurementValue":"0" },{ "BaselineMeasurementGroupId":"BG002", "BaselineMeasurementValue":"0" } ] } } ] } } ] } },{ "BaselineMeasureTitle":"Height", "BaselineMeasureParamType":"Mean", "BaselineMeasureDispersionType":"Standard Deviation", "BaselineMeasureUnitOfMeasure":"cm", "BaselineClassList":{ "BaselineClass":[ { "BaselineCategoryList":{ "BaselineCategory":[ { "BaselineMeasurementList":{ "BaselineMeasurement":[ { "BaselineMeasurementGroupId":"BG000", "BaselineMeasurementValue":"168.5", "BaselineMeasurementSpread":"5.67" },{ "BaselineMeasurementGroupId":"BG001", "BaselineMeasurementValue":"169.6", "BaselineMeasurementSpread":"8.98" },{ "BaselineMeasurementGroupId":"BG002", "BaselineMeasurementValue":"169.2", "BaselineMeasurementSpread":"7.67" } ] } } ] } } ] } },{ "BaselineMeasureTitle":"Weight", "BaselineMeasureParamType":"Mean", "BaselineMeasureDispersionType":"Standard Deviation", "BaselineMeasureUnitOfMeasure":"kg", "BaselineClassList":{ "BaselineClass":[ { "BaselineCategoryList":{ "BaselineCategory":[ { "BaselineMeasurementList":{ "BaselineMeasurement":[ { "BaselineMeasurementGroupId":"BG000", "BaselineMeasurementValue":"78.0", "BaselineMeasurementSpread":"19.11" },{ "BaselineMeasurementGroupId":"BG001", "BaselineMeasurementValue":"80.3", "BaselineMeasurementSpread":"18.95" },{ "BaselineMeasurementGroupId":"BG002", "BaselineMeasurementValue":"79.3", "BaselineMeasurementSpread":"18.73" } ] } } ] } } ] } },{ "BaselineMeasureTitle":"Body Mass Index (BMI)", "BaselineMeasureParamType":"Mean", "BaselineMeasureDispersionType":"Standard Deviation", "BaselineMeasureUnitOfMeasure":"kg/m2", "BaselineClassList":{ "BaselineClass":[ { "BaselineCategoryList":{ "BaselineCategory":[ { "BaselineMeasurementList":{ "BaselineMeasurement":[ { "BaselineMeasurementGroupId":"BG000", "BaselineMeasurementValue":"27.3", "BaselineMeasurementSpread":"5.92" },{ "BaselineMeasurementGroupId":"BG001", "BaselineMeasurementValue":"27.7", "BaselineMeasurementSpread":"5.14" },{ "BaselineMeasurementGroupId":"BG002", "BaselineMeasurementValue":"27.5", "BaselineMeasurementSpread":"5.39" } ] } } ] } } ] } },{ "BaselineMeasureTitle":"Age at diagnosis", "BaselineMeasureParamType":"Mean", "BaselineMeasureDispersionType":"Standard Deviation", "BaselineMeasureUnitOfMeasure":"years", "BaselineClassList":{ "BaselineClass":[ { "BaselineCategoryList":{ "BaselineCategory":[ { "BaselineMeasurementList":{ "BaselineMeasurement":[ { "BaselineMeasurementGroupId":"BG000", "BaselineMeasurementValue":"44.1", "BaselineMeasurementSpread":"13.79" },{ "BaselineMeasurementGroupId":"BG001", "BaselineMeasurementValue":"52.7", "BaselineMeasurementSpread":"14.76" },{ "BaselineMeasurementGroupId":"BG002", "BaselineMeasurementValue":"48.9", "BaselineMeasurementSpread":"14.76" } ] } } ] } } ] } },{ "BaselineMeasureTitle":"Time between symptom onset and diagnosis", "BaselineMeasureParamType":"Mean", "BaselineMeasureDispersionType":"Standard Deviation", "BaselineMeasureUnitOfMeasure":"years", "BaselineClassList":{ "BaselineClass":[ { "BaselineCategoryList":{ "BaselineCategory":[ { "BaselineMeasurementList":{ "BaselineMeasurement":[ { "BaselineMeasurementGroupId":"BG000", "BaselineMeasurementValue":"0.9", "BaselineMeasurementSpread":"0.62" },{ "BaselineMeasurementGroupId":"BG001", "BaselineMeasurementValue":"2.2", "BaselineMeasurementSpread":"3.00" },{ "BaselineMeasurementGroupId":"BG002", "BaselineMeasurementValue":"1.7", "BaselineMeasurementSpread":"2.35" } ] } } ] } } ] } },{ "BaselineMeasureTitle":"Duration of diagnosis prior to randomization", "BaselineMeasureParamType":"Mean", "BaselineMeasureDispersionType":"Standard Deviation", "BaselineMeasureUnitOfMeasure":"years", "BaselineClassList":{ "BaselineClass":[ { "BaselineCategoryList":{ "BaselineCategory":[ { "BaselineMeasurementList":{ "BaselineMeasurement":[ { "BaselineMeasurementGroupId":"BG000", "BaselineMeasurementValue":"6.7", "BaselineMeasurementSpread":"5.70" },{ "BaselineMeasurementGroupId":"BG001", "BaselineMeasurementValue":"6.7", "BaselineMeasurementSpread":"6.08" },{ "BaselineMeasurementGroupId":"BG002", "BaselineMeasurementValue":"6.7", "BaselineMeasurementSpread":"5.82" } ] } } ] } } ] } },{ "BaselineMeasureTitle":"Positive P/Q type voltage-gated calcium channel (VGCC) antibodies at screening", "BaselineMeasureParamType":"Count of Participants", "BaselineMeasureUnitOfMeasure":"Participants", "BaselineClassList":{ "BaselineClass":[ { "BaselineCategoryList":{ "BaselineCategory":[ { "BaselineMeasurementList":{ "BaselineMeasurement":[ { "BaselineMeasurementGroupId":"BG000", "BaselineMeasurementValue":"12" },{ "BaselineMeasurementGroupId":"BG001", "BaselineMeasurementValue":"17" },{ "BaselineMeasurementGroupId":"BG002", "BaselineMeasurementValue":"29" } ] } } ] } } ] } },{ "BaselineMeasureTitle":"Compound Muscle Action Potential (CMAP) consistent with Lambert-Eaton Myasthenia (LEM) at screening", "BaselineMeasureParamType":"Count of Participants", "BaselineMeasureUnitOfMeasure":"Participants", "BaselineClassList":{ "BaselineClass":[ { "BaselineCategoryList":{ "BaselineCategory":[ { "BaselineMeasurementList":{ "BaselineMeasurement":[ { "BaselineMeasurementGroupId":"BG000", "BaselineMeasurementValue":"7" },{ "BaselineMeasurementGroupId":"BG001", "BaselineMeasurementValue":"10" },{ "BaselineMeasurementGroupId":"BG002", "BaselineMeasurementValue":"17" } ] } } ] } } ] } },{ "BaselineMeasureTitle":"Total Daily Dose of 3,4-DAP at randomization", "BaselineMeasureParamType":"Median", "BaselineMeasureDispersionType":"Full Range", "BaselineMeasureUnitOfMeasure":"mg", "BaselineClassList":{ "BaselineClass":[ { "BaselineCategoryList":{ "BaselineCategory":[ { "BaselineMeasurementList":{ "BaselineMeasurement":[ { "BaselineMeasurementGroupId":"BG000", "BaselineMeasurementValue":"80.0", "BaselineMeasurementLowerLimit":"35", "BaselineMeasurementUpperLimit":"100" },{ "BaselineMeasurementGroupId":"BG001", "BaselineMeasurementValue":"80.0", "BaselineMeasurementLowerLimit":"30", "BaselineMeasurementUpperLimit":"100" },{ "BaselineMeasurementGroupId":"BG002", "BaselineMeasurementValue":"80", "BaselineMeasurementLowerLimit":"30", "BaselineMeasurementUpperLimit":"100" } ] } } ] } } ] } },{ "BaselineMeasureTitle":"Number of 3,4-DAP individual daily doses at randomization", "BaselineMeasureParamType":"Median", "BaselineMeasureDispersionType":"Full Range", "BaselineMeasureUnitOfMeasure":"number of daily doses", "BaselineClassList":{ "BaselineClass":[ { "BaselineCategoryList":{ "BaselineCategory":[ { "BaselineMeasurementList":{ "BaselineMeasurement":[ { "BaselineMeasurementGroupId":"BG000", "BaselineMeasurementValue":"4.5", "BaselineMeasurementLowerLimit":"3", "BaselineMeasurementUpperLimit":"7" },{ "BaselineMeasurementGroupId":"BG001", "BaselineMeasurementValue":"5.0", "BaselineMeasurementLowerLimit":"3", "BaselineMeasurementUpperLimit":"6" },{ "BaselineMeasurementGroupId":"BG002", "BaselineMeasurementValue":"5.0", "BaselineMeasurementLowerLimit":"3", "BaselineMeasurementUpperLimit":"7" } ] } } ] } } ] } } ] } }, "OutcomeMeasuresModule":{ "OutcomeMeasureList":{ "OutcomeMeasure":[ { "OutcomeMeasureType":"Primary", "OutcomeMeasureTitle":"Number of Participants With 30% or More Deterioration in Triple Timed Up & Go (3TUG) Test, Compared to Time-matched Baseline", "OutcomeMeasureDescription":"The 3TUG time obtained 2 hours after the last dose of the withdrawal period (i.e., at time of theoretical \"peak drug effect\") was compared to the average time-matched 3TUG tests performed during 2 days of baseline observation prior to randomization.\n\nThe study endpoint was a change of more than 30% in the final post-dose 3TUG during the withdrawal period and was based on blinded readings of video recordings of 3TUG tests.", "OutcomeMeasureReportingStatus":"Posted", "OutcomeMeasureParamType":"Count of Participants", "OutcomeMeasureUnitOfMeasure":"Participants", "OutcomeMeasureTimeFrame":"Baseline period (days 0, 1, 2); Randomized treatment period (starting with last dose of day 2, and days 3, 4, 5, and ending with first dose on day 6 when pre-randomization regimen was resumed, or rescue, if indicated sooner)", "OutcomeGroupList":{ "OutcomeGroup":[ { "OutcomeGroupId":"OG000", "OutcomeGroupTitle":"Continuous 3,4-Diaminopyridine (3,4-DAP)", "OutcomeGroupDescription":"Subjects were continued on their usual pre-randomization 3,4-DAP dosing regimen." },{ "OutcomeGroupId":"OG001", "OutcomeGroupTitle":"3,4-DAP Taper to Placebo", "OutcomeGroupDescription":"Subjects were tapered off of their pre-randomization 3,4-DAP dosing regimen over 3 days with up to an additional 16 hours of placebo." } ] }, "OutcomeDenomList":{ "OutcomeDenom":[ { "OutcomeDenomUnits":"Participants", "OutcomeDenomCountList":{ "OutcomeDenomCount":[ { "OutcomeDenomCountGroupId":"OG000", "OutcomeDenomCountValue":"14" },{ "OutcomeDenomCountGroupId":"OG001", "OutcomeDenomCountValue":"18" } ] } } ] }, "OutcomeClassList":{ "OutcomeClass":[ { "OutcomeCategoryList":{ "OutcomeCategory":[ { "OutcomeMeasurementList":{ "OutcomeMeasurement":[ { "OutcomeMeasurementGroupId":"OG000", "OutcomeMeasurementValue":"0" },{ "OutcomeMeasurementGroupId":"OG001", "OutcomeMeasurementValue":"13" } ] } } ] } } ] }, "OutcomeAnalysisList":{ "OutcomeAnalysis":[ { "OutcomeAnalysisGroupIdList":{ "OutcomeAnalysisGroupId":[ "OG000", "OG001" ] }, "OutcomeAnalysisNonInferiorityType":"Superiority", "OutcomeAnalysisPValue":"<0.0001", "OutcomeAnalysisStatisticalMethod":"Fisher Exact" } ] } },{ "OutcomeMeasureType":"Secondary", "OutcomeMeasureTitle":"Self-assessment of LEMS-related Weakness, W-SAS", "OutcomeMeasureDescription":"The last post-dose self-assessment of LEMS-related weakness from the withdrawal period with categories of much much weaker (-3), much weaker (-2), somewhat weaker (-1), about the same (0), somewhat stronger (1), much stronger (2), and much much stronger (3).", "OutcomeMeasureReportingStatus":"Posted", "OutcomeMeasureParamType":"Mean", "OutcomeMeasureDispersionType":"Standard Deviation", "OutcomeMeasureUnitOfMeasure":"units on a scale", "OutcomeMeasureTimeFrame":"Participants were followed for up to 7 days", "OutcomeGroupList":{ "OutcomeGroup":[ { "OutcomeGroupId":"OG000", "OutcomeGroupTitle":"3,4-DAP", "OutcomeGroupDescription":"Subjects were administered their usual dosage on their regular personalized schedule." },{ "OutcomeGroupId":"OG001", "OutcomeGroupTitle":"3,4-DAP Taper to Placebo", "OutcomeGroupDescription":"Subjects were administered decreasing amounts of 3,4-DAP on their regular personalized schedule" } ] }, "OutcomeDenomList":{ "OutcomeDenom":[ { "OutcomeDenomUnits":"Participants", "OutcomeDenomCountList":{ "OutcomeDenomCount":[ { "OutcomeDenomCountGroupId":"OG000", "OutcomeDenomCountValue":"14" },{ "OutcomeDenomCountGroupId":"OG001", "OutcomeDenomCountValue":"18" } ] } } ] }, "OutcomeClassList":{ "OutcomeClass":[ { "OutcomeCategoryList":{ "OutcomeCategory":[ { "OutcomeMeasurementList":{ "OutcomeMeasurement":[ { "OutcomeMeasurementGroupId":"OG000", "OutcomeMeasurementValue":"-0.2", "OutcomeMeasurementSpread":"1.24" },{ "OutcomeMeasurementGroupId":"OG001", "OutcomeMeasurementValue":"-2.4", "OutcomeMeasurementSpread":"0.85" } ] } } ] } } ] }, "OutcomeAnalysisList":{ "OutcomeAnalysis":[ { "OutcomeAnalysisGroupIdList":{ "OutcomeAnalysisGroupId":[ "OG000", "OG001" ] }, "OutcomeAnalysisNonInferiorityType":"Superiority", "OutcomeAnalysisPValue":"0.0001", "OutcomeAnalysisStatisticalMethod":"t-test, 2 sided" } ] } } ] } }, "AdverseEventsModule":{ "EventsFrequencyThreshold":"0", "EventsTimeFrame":"Adverse events were collected during the inpatient stay of up to 7 days on the clinical research unit and then weekly with standard telephone follow-up and diary entries for one month following discharge from the clinical research unit.", "EventsDescription":"The return of Lambert-Eaton Myasthenic Syndrome (LEMS)-related signs and symptoms in a withdrawal design was anticipated and therefore adverse events were categorized as (1) treatment emergent adverse events, (2) treatment emergent adverse events excluding LEMS-related signs and symptoms, and (3) LEMS-related signs and symptoms adverse events", "EventGroupList":{ "EventGroup":[ { "EventGroupId":"EG000", "EventGroupTitle":"Continuous 3,4-Diaminopyridine (3,4-DAP)", "EventGroupDescription":"Subjects were administered their usual dosage on their regular personalized schedule", "EventGroupDeathsNumAffected":"0", "EventGroupDeathsNumAtRisk":"14", "EventGroupSeriousNumAffected":"0", "EventGroupSeriousNumAtRisk":"14", "EventGroupOtherNumAffected":"5", "EventGroupOtherNumAtRisk":"14" },{ "EventGroupId":"EG001", "EventGroupTitle":"3,4-DAP Taper to Placebo", "EventGroupDescription":"Subjects were administered decreasing amounts of 3,4-DAP on their regular personalized schedule", "EventGroupDeathsNumAffected":"0", "EventGroupDeathsNumAtRisk":"18", "EventGroupSeriousNumAffected":"1", "EventGroupSeriousNumAtRisk":"18", "EventGroupOtherNumAffected":"12", "EventGroupOtherNumAtRisk":"18" } ] }, "SeriousEventList":{ "SeriousEvent":[ { "SeriousEventTerm":"pneumonia", "SeriousEventOrganSystem":"Infections and infestations", "SeriousEventSourceVocabulary":"MedDRA 16.0", "SeriousEventAssessmentType":"Systematic Assessment", "SeriousEventNotes":"Hospitalization for pneumonia occurred one month following discharge from the clinical research unit and was consider not related", "SeriousEventStatsList":{ "SeriousEventStats":[ { "SeriousEventStatsGroupId":"EG000", "SeriousEventStatsNumEvents":"0", "SeriousEventStatsNumAffected":"0", "SeriousEventStatsNumAtRisk":"14" },{ "SeriousEventStatsGroupId":"EG001", "SeriousEventStatsNumEvents":"1", "SeriousEventStatsNumAffected":"1", "SeriousEventStatsNumAtRisk":"18" } ] } } ] }, "OtherEventList":{ "OtherEvent":[ { "OtherEventTerm":"Abdominal discomfort", "OtherEventOrganSystem":"Gastrointestinal disorders", "OtherEventSourceVocabulary":"MedDRA 16.0", "OtherEventAssessmentType":"Systematic Assessment", "OtherEventStatsList":{ "OtherEventStats":[ { "OtherEventStatsGroupId":"EG000", "OtherEventStatsNumAffected":"0", "OtherEventStatsNumAtRisk":"14" },{ "OtherEventStatsGroupId":"EG001", "OtherEventStatsNumAffected":"2", "OtherEventStatsNumAtRisk":"18" } ] } },{ "OtherEventTerm":"Respiratory tract infection", "OtherEventOrganSystem":"Infections and infestations", "OtherEventSourceVocabulary":"MedDRA 16.0", "OtherEventAssessmentType":"Systematic Assessment", "OtherEventStatsList":{ "OtherEventStats":[ { "OtherEventStatsGroupId":"EG000", "OtherEventStatsNumAffected":"0", "OtherEventStatsNumAtRisk":"14" },{ "OtherEventStatsGroupId":"EG001", "OtherEventStatsNumAffected":"2", "OtherEventStatsNumAtRisk":"18" } ] } },{ "OtherEventTerm":"Back pain", "OtherEventOrganSystem":"Musculoskeletal and connective tissue disorders", "OtherEventSourceVocabulary":"MedDRA 16.0", "OtherEventAssessmentType":"Systematic Assessment", "OtherEventStatsList":{ "OtherEventStats":[ { "OtherEventStatsGroupId":"EG000", "OtherEventStatsNumAffected":"1", "OtherEventStatsNumAtRisk":"14" },{ "OtherEventStatsGroupId":"EG001", "OtherEventStatsNumAffected":"1", "OtherEventStatsNumAtRisk":"18" } ] } },{ "OtherEventTerm":"Headache", "OtherEventOrganSystem":"Nervous system disorders", "OtherEventSourceVocabulary":"MedDRA 16.0", "OtherEventAssessmentType":"Systematic Assessment", "OtherEventStatsList":{ "OtherEventStats":[ { "OtherEventStatsGroupId":"EG000", "OtherEventStatsNumAffected":"1", "OtherEventStatsNumAtRisk":"14" },{ "OtherEventStatsGroupId":"EG001", "OtherEventStatsNumAffected":"1", "OtherEventStatsNumAtRisk":"18" } ] } },{ "OtherEventTerm":"Nasopharyngitis", "OtherEventOrganSystem":"Infections and infestations", "OtherEventSourceVocabulary":"MedDRA 16.0", "OtherEventAssessmentType":"Systematic Assessment", "OtherEventStatsList":{ "OtherEventStats":[ { "OtherEventStatsGroupId":"EG000", "OtherEventStatsNumAffected":"1", "OtherEventStatsNumAtRisk":"14" },{ "OtherEventStatsGroupId":"EG001", "OtherEventStatsNumAffected":"1", "OtherEventStatsNumAtRisk":"18" } ] } },{ "OtherEventTerm":"Oropharyngeal pain", "OtherEventOrganSystem":"Respiratory, thoracic and mediastinal disorders", "OtherEventSourceVocabulary":"MedDRA 16.0", "OtherEventAssessmentType":"Systematic Assessment", "OtherEventStatsList":{ "OtherEventStats":[ { "OtherEventStatsGroupId":"EG000", "OtherEventStatsNumAffected":"1", "OtherEventStatsNumAtRisk":"14" },{ "OtherEventStatsGroupId":"EG001", "OtherEventStatsNumAffected":"1", "OtherEventStatsNumAtRisk":"18" } ] } },{ "OtherEventTerm":"Abdominal pain lower", "OtherEventOrganSystem":"Gastrointestinal disorders", "OtherEventSourceVocabulary":"MedDRA 16.0", "OtherEventAssessmentType":"Systematic Assessment", "OtherEventStatsList":{ "OtherEventStats":[ { "OtherEventStatsGroupId":"EG000", "OtherEventStatsNumAffected":"0", "OtherEventStatsNumAtRisk":"14" },{ "OtherEventStatsGroupId":"EG001", "OtherEventStatsNumAffected":"1", "OtherEventStatsNumAtRisk":"18" } ] } },{ "OtherEventTerm":"Blood glucose increased", "OtherEventOrganSystem":"Investigations", "OtherEventSourceVocabulary":"MedDRA 16.0", "OtherEventAssessmentType":"Systematic Assessment", "OtherEventStatsList":{ "OtherEventStats":[ { "OtherEventStatsGroupId":"EG000", "OtherEventStatsNumAffected":"0", "OtherEventStatsNumAtRisk":"14" },{ "OtherEventStatsGroupId":"EG001", "OtherEventStatsNumAffected":"1", "OtherEventStatsNumAtRisk":"18" } ] } },{ "OtherEventTerm":"Cheilitis", "OtherEventOrganSystem":"Gastrointestinal disorders", "OtherEventSourceVocabulary":"MedDRA 16.0", "OtherEventAssessmentType":"Systematic Assessment", "OtherEventStatsList":{ "OtherEventStats":[ { "OtherEventStatsGroupId":"EG000", "OtherEventStatsNumAffected":"0", "OtherEventStatsNumAtRisk":"14" },{ "OtherEventStatsGroupId":"EG001", "OtherEventStatsNumAffected":"1", "OtherEventStatsNumAtRisk":"18" } ] } },{ "OtherEventTerm":"Dizziness", "OtherEventOrganSystem":"Nervous system disorders", "OtherEventSourceVocabulary":"MedDRA 16.0", "OtherEventAssessmentType":"Systematic Assessment", "OtherEventStatsList":{ "OtherEventStats":[ { "OtherEventStatsGroupId":"EG000", "OtherEventStatsNumAffected":"0", "OtherEventStatsNumAtRisk":"14" },{ "OtherEventStatsGroupId":"EG001", "OtherEventStatsNumAffected":"1", "OtherEventStatsNumAtRisk":"18" } ] } },{ "OtherEventTerm":"Electrocardiogram QT prolonged", "OtherEventOrganSystem":"Investigations", "OtherEventSourceVocabulary":"MedDRA 16.0", "OtherEventAssessmentType":"Systematic Assessment", "OtherEventStatsList":{ "OtherEventStats":[ { "OtherEventStatsGroupId":"EG000", "OtherEventStatsNumAffected":"0", "OtherEventStatsNumAtRisk":"14" },{ "OtherEventStatsGroupId":"EG001", "OtherEventStatsNumAffected":"1", "OtherEventStatsNumAtRisk":"18" } ] } },{ "OtherEventTerm":"Gout", "OtherEventOrganSystem":"Metabolism and nutrition disorders", "OtherEventSourceVocabulary":"MedDRA 16.0", "OtherEventAssessmentType":"Systematic Assessment", "OtherEventStatsList":{ "OtherEventStats":[ { "OtherEventStatsGroupId":"EG000", "OtherEventStatsNumAffected":"0", "OtherEventStatsNumAtRisk":"14" },{ "OtherEventStatsGroupId":"EG001", "OtherEventStatsNumAffected":"1", "OtherEventStatsNumAtRisk":"18" } ] } },{ "OtherEventTerm":"Nausea", "OtherEventOrganSystem":"Gastrointestinal disorders", "OtherEventSourceVocabulary":"MedDRA 16.0", "OtherEventAssessmentType":"Systematic Assessment", "OtherEventStatsList":{ "OtherEventStats":[ { "OtherEventStatsGroupId":"EG000", "OtherEventStatsNumAffected":"0", "OtherEventStatsNumAtRisk":"14" },{ "OtherEventStatsGroupId":"EG001", "OtherEventStatsNumAffected":"1", "OtherEventStatsNumAtRisk":"18" } ] } },{ "OtherEventTerm":"Paresthesia", "OtherEventOrganSystem":"Nervous system disorders", "OtherEventSourceVocabulary":"MedDRA 16.0", "OtherEventAssessmentType":"Systematic Assessment", "OtherEventStatsList":{ "OtherEventStats":[ { "OtherEventStatsGroupId":"EG000", "OtherEventStatsNumAffected":"0", "OtherEventStatsNumAtRisk":"14" },{ "OtherEventStatsGroupId":"EG001", "OtherEventStatsNumAffected":"1", "OtherEventStatsNumAtRisk":"18" } ] } },{ "OtherEventTerm":"Pyrexia", "OtherEventOrganSystem":"General disorders", "OtherEventSourceVocabulary":"MedDRA 16.0", "OtherEventAssessmentType":"Systematic Assessment", "OtherEventStatsList":{ "OtherEventStats":[ { "OtherEventStatsGroupId":"EG000", "OtherEventStatsNumAffected":"0", "OtherEventStatsNumAtRisk":"14" },{ "OtherEventStatsGroupId":"EG001", "OtherEventStatsNumAffected":"1", "OtherEventStatsNumAtRisk":"18" } ] } },{ "OtherEventTerm":"Rash", "OtherEventOrganSystem":"Skin and subcutaneous tissue disorders", "OtherEventSourceVocabulary":"MedDRA 16.0", "OtherEventAssessmentType":"Systematic Assessment", "OtherEventStatsList":{ "OtherEventStats":[ { "OtherEventStatsGroupId":"EG000", "OtherEventStatsNumAffected":"0", "OtherEventStatsNumAtRisk":"14" },{ "OtherEventStatsGroupId":"EG001", "OtherEventStatsNumAffected":"1", "OtherEventStatsNumAtRisk":"18" } ] } },{ "OtherEventTerm":"Seasonal allergy", "OtherEventOrganSystem":"Immune system disorders", "OtherEventSourceVocabulary":"MedDRA 16.0", "OtherEventAssessmentType":"Systematic Assessment", "OtherEventStatsList":{ "OtherEventStats":[ { "OtherEventStatsGroupId":"EG000", "OtherEventStatsNumAffected":"0", "OtherEventStatsNumAtRisk":"14" },{ "OtherEventStatsGroupId":"EG001", "OtherEventStatsNumAffected":"1", "OtherEventStatsNumAtRisk":"18" } ] } },{ "OtherEventTerm":"Sinus headache", "OtherEventOrganSystem":"Nervous system disorders", "OtherEventSourceVocabulary":"MedDRA 16.0", "OtherEventAssessmentType":"Systematic Assessment", "OtherEventStatsList":{ "OtherEventStats":[ { "OtherEventStatsGroupId":"EG000", "OtherEventStatsNumAffected":"0", "OtherEventStatsNumAtRisk":"14" },{ "OtherEventStatsGroupId":"EG001", "OtherEventStatsNumAffected":"1", "OtherEventStatsNumAtRisk":"18" } ] } },{ "OtherEventTerm":"Sinusitis", "OtherEventOrganSystem":"Infections and infestations", "OtherEventSourceVocabulary":"MedDRA 16.0", "OtherEventAssessmentType":"Systematic Assessment", "OtherEventStatsList":{ "OtherEventStats":[ { "OtherEventStatsGroupId":"EG000", "OtherEventStatsNumAffected":"0", "OtherEventStatsNumAtRisk":"14" },{ "OtherEventStatsGroupId":"EG001", "OtherEventStatsNumAffected":"1", "OtherEventStatsNumAtRisk":"18" } ] } },{ "OtherEventTerm":"Sjogren", "OtherEventOrganSystem":"Musculoskeletal and connective tissue disorders", "OtherEventSourceVocabulary":"MedDRA 16.0", "OtherEventAssessmentType":"Systematic Assessment", "OtherEventStatsList":{ "OtherEventStats":[ { "OtherEventStatsGroupId":"EG000", "OtherEventStatsNumAffected":"0", "OtherEventStatsNumAtRisk":"14" },{ "OtherEventStatsGroupId":"EG001", "OtherEventStatsNumAffected":"1", "OtherEventStatsNumAtRisk":"18" } ] } },{ "OtherEventTerm":"Gastritis", "OtherEventOrganSystem":"Gastrointestinal disorders", "OtherEventSourceVocabulary":"MedDRA 16.0", "OtherEventAssessmentType":"Systematic Assessment", "OtherEventStatsList":{ "OtherEventStats":[ { "OtherEventStatsGroupId":"EG000", "OtherEventStatsNumAffected":"1", "OtherEventStatsNumAtRisk":"14" },{ "OtherEventStatsGroupId":"EG001", "OtherEventStatsNumAffected":"0", "OtherEventStatsNumAtRisk":"18" } ] } },{ "OtherEventTerm":"Injection site bruising", "OtherEventOrganSystem":"General disorders", "OtherEventSourceVocabulary":"MedDRA 16.0", "OtherEventAssessmentType":"Systematic Assessment", "OtherEventStatsList":{ "OtherEventStats":[ { "OtherEventStatsGroupId":"EG000", "OtherEventStatsNumAffected":"1", "OtherEventStatsNumAtRisk":"14" },{ "OtherEventStatsGroupId":"EG001", "OtherEventStatsNumAffected":"0", "OtherEventStatsNumAtRisk":"18" } ] } },{ "OtherEventTerm":"Instillation site irritation", "OtherEventOrganSystem":"General disorders", "OtherEventSourceVocabulary":"MedDRA 16.0", "OtherEventAssessmentType":"Systematic Assessment", "OtherEventStatsList":{ "OtherEventStats":[ { "OtherEventStatsGroupId":"EG000", "OtherEventStatsNumAffected":"1", "OtherEventStatsNumAtRisk":"14" },{ "OtherEventStatsGroupId":"EG001", "OtherEventStatsNumAffected":"0", "OtherEventStatsNumAtRisk":"18" } ] } },{ "OtherEventTerm":"Musculoskeletal stiffness", "OtherEventOrganSystem":"Musculoskeletal and connective tissue disorders", "OtherEventSourceVocabulary":"MedDRA 16.0", "OtherEventAssessmentType":"Systematic Assessment", "OtherEventStatsList":{ "OtherEventStats":[ { "OtherEventStatsGroupId":"EG000", "OtherEventStatsNumAffected":"1", "OtherEventStatsNumAtRisk":"14" },{ "OtherEventStatsGroupId":"EG001", "OtherEventStatsNumAffected":"0", "OtherEventStatsNumAtRisk":"18" } ] } },{ "OtherEventTerm":"Oral herpes", "OtherEventOrganSystem":"Infections and infestations", "OtherEventSourceVocabulary":"MedDRA 16.0", "OtherEventAssessmentType":"Systematic Assessment", "OtherEventStatsList":{ "OtherEventStats":[ { "OtherEventStatsGroupId":"EG000", "OtherEventStatsNumAffected":"1", "OtherEventStatsNumAtRisk":"14" },{ "OtherEventStatsGroupId":"EG001", "OtherEventStatsNumAffected":"0", "OtherEventStatsNumAtRisk":"18" } ] } } ] } }, "MoreInfoModule":{ "CertainAgreement":{ "AgreementPISponsorEmployee":"No", "AgreementRestrictionType":"OTHER", "AgreementRestrictiveAgreement":"Yes", "AgreementOtherDetails":"The first publication shall be a multicenter publication submitted after conclusion of the Study at all sites. If a multicenter publication is not submitted within 12 months, PIs may use results.\n\nThe only other disclosure restriction on the PI is that the Sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period of 30 days for confidential review. This period may be extended by 60 days at Sponsor’s request." }, "PointOfContact":{ "PointOfContactTitle":"Dr. Kathy Aleš, Medical Director", "PointOfContactOrganization":"Jacobus Pharmaceutical Company, Inc.", "PointOfContactEMail":"kathy.ales@jacobus-pharmaceutical.com", "PointOfContactPhone":"6099217447", "PointOfContactPhoneExt":"231" } } }, "DerivedSection":{ "MiscInfoModule":{ "VersionHolder":"April 22, 2020" }, "InterventionBrowseModule":{ "InterventionMeshList":{ "InterventionMesh":[ { "InterventionMeshId":"D000077770", "InterventionMeshTerm":"Amifampridine" } ] }, "InterventionAncestorList":{ "InterventionAncestor":[ { "InterventionAncestorId":"D000009465", "InterventionAncestorTerm":"Neuromuscular Agents" },{ "InterventionAncestorId":"D000018373", "InterventionAncestorTerm":"Peripheral Nervous System Agents" },{ "InterventionAncestorId":"D000045505", "InterventionAncestorTerm":"Physiological Effects of Drugs" },{ "InterventionAncestorId":"D000026902", "InterventionAncestorTerm":"Potassium Channel Blockers" },{ "InterventionAncestorId":"D000049990", "InterventionAncestorTerm":"Membrane Transport Modulators" },{ "InterventionAncestorId":"D000045504", "InterventionAncestorTerm":"Molecular Mechanisms of Pharmacological Action" } ] }, "InterventionBrowseLeafList":{ "InterventionBrowseLeaf":[ { "InterventionBrowseLeafId":"M1902", "InterventionBrowseLeafName":"Amifampridine", "InterventionBrowseLeafAsFound":"3,4-diaminopyridine", "InterventionBrowseLeafRelevance":"high" },{ "InterventionBrowseLeafId":"M21755", "InterventionBrowseLeafName":"Potassium Channel Blockers", "InterventionBrowseLeafRelevance":"low" } ] }, "InterventionBrowseBranchList":{ "InterventionBrowseBranch":[ { "InterventionBrowseBranchAbbrev":"ChanBlk", "InterventionBrowseBranchName":"Channel Blockers" },{ "InterventionBrowseBranchAbbrev":"All", "InterventionBrowseBranchName":"All Drugs and Chemicals" } ] } }, "ConditionBrowseModule":{ "ConditionMeshList":{ "ConditionMesh":[ { "ConditionMeshId":"D000015624", "ConditionMeshTerm":"Lambert-Eaton Myasthenic Syndrome" },{ "ConditionMeshId":"D000013577", "ConditionMeshTerm":"Syndrome" } ] }, "ConditionAncestorList":{ "ConditionAncestor":[ { "ConditionAncestorId":"D000004194", "ConditionAncestorTerm":"Disease" },{ "ConditionAncestorId":"D000010335", "ConditionAncestorTerm":"Pathologic Processes" },{ "ConditionAncestorId":"D000020361", "ConditionAncestorTerm":"Paraneoplastic Syndromes, Nervous System" },{ "ConditionAncestorId":"D000009423", "ConditionAncestorTerm":"Nervous System Neoplasms" },{ "ConditionAncestorId":"D000009371", "ConditionAncestorTerm":"Neoplasms by Site" },{ "ConditionAncestorId":"D000009369", "ConditionAncestorTerm":"Neoplasms" },{ "ConditionAncestorId":"D000010257", "ConditionAncestorTerm":"Paraneoplastic Syndromes" },{ "ConditionAncestorId":"D000020274", "ConditionAncestorTerm":"Autoimmune Diseases of the Nervous System" },{ "ConditionAncestorId":"D000009422", "ConditionAncestorTerm":"Nervous System Diseases" },{ "ConditionAncestorId":"D000019636", "ConditionAncestorTerm":"Neurodegenerative Diseases" },{ "ConditionAncestorId":"D000020511", "ConditionAncestorTerm":"Neuromuscular Junction Diseases" },{ "ConditionAncestorId":"D000009468", "ConditionAncestorTerm":"Neuromuscular Diseases" },{ "ConditionAncestorId":"D000001327", "ConditionAncestorTerm":"Autoimmune Diseases" },{ "ConditionAncestorId":"D000007154", "ConditionAncestorTerm":"Immune System Diseases" } ] }, "ConditionBrowseLeafList":{ "ConditionBrowseLeaf":[ { "ConditionBrowseLeafId":"M14938", "ConditionBrowseLeafName":"Syndrome", "ConditionBrowseLeafAsFound":"Syndrome", "ConditionBrowseLeafRelevance":"high" },{ "ConditionBrowseLeafId":"M16807", "ConditionBrowseLeafName":"Lambert-Eaton Myasthenic Syndrome", "ConditionBrowseLeafAsFound":"Lambert-Eaton Myasthenic Syndrome", "ConditionBrowseLeafRelevance":"high" },{ "ConditionBrowseLeafId":"M11753", 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} } } } }