Ignite Creation Date:
2025-12-26 @ 11:49 AM
Ignite Modification Date:
2025-12-26 @ 11:49 AM
Study NCT ID:
NCT04923516
Status:
UNKNOWN
Last Update Posted:
2021-06-16
First Post:
2021-06-08
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Prevalence of Acute Hepatic Porphyria
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D046349', 'term': 'Coproporphyria, Hereditary'}], 'ancestors': [{'id': 'D017094', 'term': 'Porphyrias, Hepatic'}, {'id': 'D008107', 'term': 'Liver Diseases'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}, {'id': 'D012873', 'term': 'Skin Diseases, Genetic'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D012871', 'term': 'Skin Diseases'}, {'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}, {'id': 'D011164', 'term': 'Porphyrias'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D011162', 'term': 'Porphobilinogen'}], 'ancestors': [{'id': 'D011758', 'term': 'Pyrroles'}, {'id': 'D001393', 'term': 'Azoles'}, {'id': 'D006573', 'term': 'Heterocyclic Compounds, 1-Ring'}, {'id': 'D006571', 'term': 'Heterocyclic Compounds'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'CROSS_SECTIONAL', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 500}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'NOT_YET_RECRUITING', 'startDateStruct': {'date': '2021-06', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-06', 'completionDateStruct': {'date': '2022-04', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2021-06-11', 'studyFirstSubmitDate': '2021-06-08', 'studyFirstSubmitQcDate': '2021-06-08', 'lastUpdatePostDateStruct': {'date': '2021-06-16', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2021-06-11', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2022-04', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Binary criterion corresponding to the diagnosis of acute hepatic porphyria (acute hepatic porphyria + / acute hepatic porphyria -) via physiological parameter.', 'timeFrame': '1 day', 'description': 'The diagnosis of acute hepatic porphyria is based on urine testing of the neurotoxic precursors of haem: Delta-aminolevulinic acid and Porphobilinogen. A patient will be considered to have acute hepatic porphyria (acute hepatic porphyria +) if:\n\n* Delta-aminolevulinic acid ≥ 3 µmol / mol Cr And or\n* Porphobilinogen ≥ 1 µmol / mol Cr Otherwise, the patient will be considered to be acute hepatic porphyria free (acute hepatic porphyria -).'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Acute Hepatic Porphyria']}, 'descriptionModule': {'briefSummary': 'To determine the proportion of patients suffering from acute hepatic porphyria (AHP) from different hospital departments and referred to an internist referent for a suggestive clinical picture with a first negative etiological assessment.', 'detailedDescription': 'In this context, this study aims to assess the prevalence of PHA in a population of patients with a suggestive clinical picture. A better knowledge of the pathology will make it possible to better guide patients and prevent them from diagnostic wandering fraught with physical and psychological consequences.\n\nThis is an observational, ambispective, transversal, multicenter study carried out in France. The goal is to recruit a cohort that will reflect current practice. 500 patients will be recruted.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT'], 'maximumAge': '60 Years', 'minimumAge': '18 Years', 'samplingMethod': 'PROBABILITY_SAMPLE', 'studyPopulation': 'The target population is made up of patients presenting signs suggestive of acute hepatic porphyria or recently diagnosed, from consultations with gastroenterologists, emergency physicians, neurologists, gynecologists, internists or other specialties, referred to an acute hepatic porphyria referent internist participating in the study, within the framework of of the etiological exploration of their clinical picture.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Man or woman aged 18 to 60;\n* Presenting severe abdominal pain, afebrile and diffuse over several days, evolving for less than 5 years;\n* With at least one of the following symptoms:\n\n * Peripheral neurological symptoms: decreased driving force and pain in the limbs (roots of the thighs), etc. ;\n * Central neurological symptoms: psychiatric (depression, anxiety, insomnia), disturbances of consciousness, etc. ;\n * Autonomous neurological symptoms: tachycardia, nausea, vomiting, constipation, etc.\n* With a first negative etiological assessment requested by an emergency physician, gastroenterologist, gynecologist, neurologist, internist or another specialty, or with a diagnosis of acute hepatic porphyria less than 6 months;\n* Patient able to understand the information related to the study and having indicated his non-objection to the collection of data concerning him;\n\nExclusion Criteria:\n\n* Patient with a diagnosis of acute hepatic porphyria for more than 6 months;\n* Patient unfit to participate in the study, due to cognitive or linguistic difficulties;\n* Protected patient (under legal protection, or deprived of liberty by judicial or administrative decision).'}, 'identificationModule': {'nctId': 'NCT04923516', 'acronym': 'PHA', 'briefTitle': 'Prevalence of Acute Hepatic Porphyria', 'organization': {'class': 'NETWORK', 'fullName': 'Association pour la Recherche en Medecine Interne'}, 'officialTitle': 'Prevalence of Acute Hepatic Porphyria in Population With Suggestive Clinical Picture', 'orgStudyIdInfo': {'id': '2021-A00566-35'}}, 'armsInterventionsModule': {'interventions': [{'name': 'Dosage of Delta-aminolevulinic acid and Porphobilinogen', 'type': 'OTHER', 'description': 'Results of dosages of Delta-aminolevulinic acid and Porphobilinogen which are performed in accordance with the current practice will be recorded'}]}, 'contactsLocationsModule': {'centralContacts': [{'name': 'Patrice CACOUB, MD', 'role': 'CONTACT', 'email': 'patrice.cacoub@aphp.fr', 'phone': '01 42 17 80 27'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Association pour la Recherche en Medecine Interne', 'class': 'NETWORK'}, 'responsibleParty': {'type': 'SPONSOR'}}}}