Nctid:
NCT06225882
Payload:
{"hasResults"=>false, "derivedSection"=>{"miscInfoModule"=>{"versionHolder"=>"2024-10-01"}, "conditionBrowseModule"=>{"meshes"=>[{"id"=>"D000006960", "term"=>"Hyperoxaluria, Primary"}], "ancestors"=>[{"id"=>"D000006959", "term"=>"Hyperoxaluria"}, {"id"=>"D000007674", "term"=>"Kidney Diseases"}, {"id"=>"D000014570", "term"=>"Urologic Diseases"}, {"id"=>"D000052776", "term"=>"Female Urogenital Diseases"}, {"id"=>"D000005261", "term"=>"Female Urogenital Diseases and Pregnancy Complications"}, {"id"=>"D000091642", "term"=>"Urogenital Diseases"}, {"id"=>"D000052801", "term"=>"Male Urogenital Diseases"}, {"id"=>"D000002239", "term"=>"Carbohydrate Metabolism, Inborn Errors"}, {"id"=>"D000008661", "term"=>"Metabolism, Inborn Errors"}, {"id"=>"D000030342", "term"=>"Genetic Diseases, Inborn"}, {"id"=>"D000008659", "term"=>"Metabolic Diseases"}], "browseLeaves"=>[{"id"=>"M10011", "name"=>"Hyperoxaluria, Primary", "asFound"=>"Primary Hyperoxaluria", "relevance"=>"HIGH"}, {"id"=>"M10010", "name"=>"Hyperoxaluria", "relevance"=>"LOW"}, {"id"=>"M10698", "name"=>"Kidney Diseases", "relevance"=>"LOW"}, {"id"=>"M17319", "name"=>"Urologic Diseases", "relevance"=>"LOW"}, {"id"=>"M2875", "name"=>"Urogenital Diseases", "relevance"=>"LOW"}, {"id"=>"M27093", "name"=>"Female Urogenital Diseases", "relevance"=>"LOW"}, {"id"=>"M14127", "name"=>"Pregnancy Complications", "relevance"=>"LOW"}, {"id"=>"M8399", "name"=>"Female Urogenital Diseases and Pregnancy Complications", "relevance"=>"LOW"}, {"id"=>"M27095", "name"=>"Male Urogenital Diseases", "relevance"=>"LOW"}, {"id"=>"M11641", "name"=>"Metabolism, Inborn Errors", "relevance"=>"LOW"}, {"id"=>"M5498", "name"=>"Carbohydrate Metabolism, Inborn Errors", "relevance"=>"LOW"}, {"id"=>"M23686", "name"=>"Genetic Diseases, Inborn", "relevance"=>"LOW"}, {"id"=>"M11639", "name"=>"Metabolic Diseases", "relevance"=>"LOW"}, {"id"=>"T4693", "name"=>"Primary Hyperoxaluria Type 1", "asFound"=>"Primary Hyperoxaluria Type 1", "relevance"=>"HIGH"}], "browseBranches"=>[{"name"=>"Urinary Tract, Sexual Organs, and Pregnancy Conditions", "abbrev"=>"BXS"}, {"name"=>"Diseases and Abnormalities at or Before Birth", "abbrev"=>"BC16"}, {"name"=>"Nutritional and Metabolic Diseases", "abbrev"=>"BC18"}, {"name"=>"All Conditions", "abbrev"=>"All"}, {"name"=>"Rare Diseases", "abbrev"=>"Rare"}]}, "interventionBrowseModule"=>{"browseLeaves"=>[{"id"=>"M346884", "name"=>"Lumasiran", "relevance"=>"LOW"}], "browseBranches"=>[{"name"=>"Renal Agents", "abbrev"=>"Rena"}, {"name"=>"All Drugs and Chemicals", "abbrev"=>"All"}]}}, "protocolSection"=>{"designModule"=>{"studyType"=>"OBSERVATIONAL", "designInfo"=>{"timePerspective"=>"CROSS_SECTIONAL", "observationalModel"=>"COHORT"}, "enrollmentInfo"=>{"type"=>"ESTIMATED", "count"=>100}, "patientRegistry"=>false}, "statusModule"=>{"overallStatus"=>"RECRUITING", "startDateStruct"=>{"date"=>"2023-01-01", "type"=>"ACTUAL"}, "expandedAccessInfo"=>{"hasExpandedAccess"=>false}, "statusVerifiedDate"=>"2024-01", "completionDateStruct"=>{"date"=>"2026-12-31", "type"=>"ESTIMATED"}, "lastUpdateSubmitDate"=>"2024-01-17", "studyFirstSubmitDate"=>"2024-01-17", "studyFirstSubmitQcDate"=>"2024-01-17", "lastUpdatePostDateStruct"=>{"date"=>"2024-01-26", "type"=>"ACTUAL"}, "studyFirstPostDateStruct"=>{"date"=>"2024-01-26", "type"=>"ACTUAL"}, "primaryCompletionDateStruct"=>{"date"=>"2024-01-01", "type"=>"ACTUAL"}}, "outcomesModule"=>{"primaryOutcomes"=>[{"measure"=>"Evolution of oxaluria.", "timeFrame"=>"At baseline, At 1 month from the baseline, At 2 months from baseline, At 3 months from baseline, At 6 months from baseline, At 9 months from baseline, At 12 months from baseline, At 18 months from baseline, And 2 times a year until 5 year", "description"=>"The evolution of oxaluria is followed by urinary biological analysis."}]}, "oversightModule"=>{"oversightHasDmc"=>false, "isFdaRegulatedDrug"=>false, "isFdaRegulatedDevice"=>false}, "conditionsModule"=>{"keywords"=>["lumarisan", "Primary hyperoxaluria type 1 (PH1)", "retrospective and prospective data."], "conditions"=>["Patients With PH1 Treated With Lumasiran in France"]}, "descriptionModule"=>{"briefSummary"=>"Primary hyperoxaluria type 1 (PH1) is a rare genetic disease caused by mutation in the AGXT gene encoding the hepatic peroxisomal enzyme AGT. Reduced AGT activity results in increased glyoxylate and oxalate production, causing the formation of kidney stones, nephrocalcinosis and renal failure. Clinical trials of Lumasiran have provided information on the efficacy and safety of Lumasiran in the treatment of primary hyperoxaluria type 1. However, they do not provide data on long-term efficacy, safety and patient management. As part of the post-marketing follow-up of Lumasiran, in agreement with the authorities, this study proposes a retrospective and prospective follow-up over 5 years of pediatrics and adults patients treated in France with a standardized clinical, biological and radiological follow-up. The main objective is to monitor the evolution of PH1 parameters and particularly oxaluria before and after treatment."}, "eligibilityModule"=>{"sex"=>"ALL", "stdAges"=>["CHILD", "ADULT", "OLDER_ADULT"], "maximumAge"=>"99 years", "minimumAge"=>"0 years", "samplingMethod"=>"NON_PROBABILITY_SAMPLE", "studyPopulation"=>"Patient with primary hyperoxaluria type 1 who has been treated with Lumasiran in France.", "eligibilityCriteria"=>"Inclusion Criteria:\n\n* Patient with primary hyperoxaluria type 1 who has been treated with Lumasiran, since the beginning of the ATU (temporary authorization for use) and in post-marketing.\n\nExclusion Criteria:\n\n* Opposition of the patient or his legal representatives for minors.\n* Not covered by social security."}, "identificationModule"=>{"nctId"=>"NCT06225882", "acronym"=>"DAILY-LUMA", "briefTitle"=>"Retrospective and Prospective Follow-up of Patients With Primary Hyperoxaluria Type 1 Treated With Lumasiran in France.", "organization"=>{"class"=>"OTHER", "fullName"=>"Hospices Civils de Lyon"}, "officialTitle"=>"Retrospective and Prospective Follow-up of Patients With Primary Hyperoxaluria Type 1 Treated With Lumasiran in France - DAILY-LUMA", "orgStudyIdInfo"=>{"id"=>"69HCL22_0535"}}, "armsInterventionsModule"=>{"armGroups"=>[{"label"=>"Lumasiran", "description"=>"Patient with primary hyperoxaluria type 1 who has been treated with Lumasiran, since the beginning of the ATU (temporary authorization for use) and in post-marketing.", "interventionNames"=>["Drug: Oxaluria evolution."]}], "interventions"=>[{"name"=>"Oxaluria evolution.", "type"=>"DRUG", "description"=>"To collect real data from the specific French experience by collecting data from patients treated throughout the country and to monitor in particular the evolution of oxaluria before and after treatment.", "armGroupLabels"=>["Lumasiran"]}]}, "contactsLocationsModule"=>{"locations"=>[{"zip"=>"75015", "city"=>"Paris", "state"=>"Ile De France", "status"=>"RECRUITING", "country"=>"France", "contacts"=>[{"name"=>"Bertrand KNEBELMANN, Pr", "role"=>"CONTACT", "email"=>"bertrand.knebelmann@nck.aphp.fr", "phone"=>"(0)144 495 241", "phoneExt"=>"+33"}], "facility"=>"Hôpital Necker, APHP Paris, Service de néphrologie-dialyse, 149 rue de Sèvres", "geoPoint"=>{"lat"=>48.85341, "lon"=>2.3488}}, {"zip"=>"25030", "city"=>"Besançon", "status"=>"RECRUITING", "country"=>"France", "contacts"=>[{"name"=>"Francois NOBILI, MD,PhD", "role"=>"CONTACT", "email"=>"fnobili@chu-besancon.fr", "phone"=>"03 81 21 88 15", "phoneExt"=>"+33"}, {"name"=>"Francois NOBILI, MD,PhD", "role"=>"PRINCIPAL_INVESTIGATOR"}], "facility"=>"CHU de Besançon", "geoPoint"=>{"lat"=>47.24878, "lon"=>6.01815}}, {"zip"=>"69500", "city"=>"Bron", "status"=>"RECRUITING", "country"=>"France", "contacts"=>[{"name"=>"Justine BACCHETTA, MD", "role"=>"CONTACT", "email"=>"justine.bacchetta@chu-lyon.fr"}, {"name"=>"Justine BACCHETTA, MD", "role"=>"PRINCIPAL_INVESTIGATOR"}], "facility"=>"Centre de Référence des Maladies Rénales Rares - Hospices Civils de Lyon - Service de Néphrologie et Rhumatologie Pédiatriques - Hôpital Femme Mère Enfant", "geoPoint"=>{"lat"=>45.73333, "lon"=>4.91667}}, {"zip"=>"69003", "city"=>"Lyon", "status"=>"RECRUITING", "country"=>"France", "contacts"=>[{"name"=>"Sandrine LEMOINE, PU-PH", "role"=>"CONTACT", "email"=>"sandrine.lemoine01@chu-lyon.fr", "phone"=>"04 72 11 02 44"}, {"name"=>"Sandrine LEMOINE, PU-PH", "role"=>"PRINCIPAL_INVESTIGATOR"}], "facility"=>"Hopital Edouard Herriot", "geoPoint"=>{"lat"=>45.74848, "lon"=>4.84669}}, {"zip"=>"13385", "city"=>"Marseille", "status"=>"RECRUITING", "country"=>"France", "contacts"=>[{"name"=>"Caroline ROUSSET-ROUVIERE, MD PHD", "role"=>"CONTACT", "email"=>"caroline.rousset-rouviere@ap-hm.fr", "phone"=>"04 91 38 80 50", "phoneExt"=>"+33"}, {"name"=>"Caroline ROUSSET-ROUVIERE, MD PHD", "role"=>"PRINCIPAL_INVESTIGATOR"}], "facility"=>"AP-HM - Timone Enfants", "geoPoint"=>{"lat"=>43.29551, "lon"=>5.38958}}, {"zip"=>"75015", "city"=>"Paris", "status"=>"RECRUITING", "country"=>"France", "contacts"=>[{"name"=>"Alexandre KARRAS, Pr", "role"=>"CONTACT", "email"=>"alexandre.karras@egp.aphp.fr", "phone"=>"1 56 09 37 60", "phoneExt"=>"+33"}, {"name"=>"Alexandre KARRAS, Pr", "role"=>"PRINCIPAL_INVESTIGATOR"}], "facility"=>"Hôpital Européen G. Pompidou", "geoPoint"=>{"lat"=>48.85341, "lon"=>2.3488}}, {"zip"=>"75743", "city"=>"Paris", "status"=>"RECRUITING", "country"=>"France", "contacts"=>[{"name"=>"Aude SERVAIS, PU PH", "role"=>"CONTACT", "email"=>"aude.servais@aphp.fr", "phone"=>"01 44 49 54 16", "phoneExt"=>"+33"}, {"name"=>"Olivia BOYER, MD PHD", "role"=>"CONTACT", "email"=>"olivia.boyer@aphp.fr", "phone"=>"01 44 49 54 16", "phoneExt"=>"+33"}, {"name"=>"Aude SERVAIS, PU PH", "role"=>"PRINCIPAL_INVESTIGATOR"}, {"name"=>"Olivia BOYER, MD PHD", "role"=>"SUB_INVESTIGATOR"}], "facility"=>"CHU Paris - Hôpital Necker-Enfants Malades", "geoPoint"=>{"lat"=>48.85341, "lon"=>2.3488}}], "centralContacts"=>[{"name"=>"Mélissa CLOAREC, Clinical Research Associate", "role"=>"CONTACT", "email"=>"melissa.cloarec@chu-lyon.fr", "phone"=>"04 27 85 51 54"}, {"name"=>"Sacha FLAMMIER, Project Manager", "role"=>"CONTACT", "email"=>"sacha.flammier@chu-lyon.fr", "phone"=>"04 72 68 13 49"}]}, "sponsorCollaboratorsModule"=>{"leadSponsor"=>{"name"=>"Hospices Civils de Lyon", "class"=>"OTHER"}, "responsibleParty"=>{"type"=>"SPONSOR"}}}}
