Prevalence of Acute Hepatic Porphyria

Acute Hepatic Porphyria Clinical Trial

— PHA  

Official title:

Prevalence of Acute Hepatic Porphyria in Population With Suggestive Clinical Picture

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT04923516
• Other study ID #: 2021-A00566-35
• Status: Not yet recruiting
• Start date: June 2021
• Est. completion date: April 2022

Study information

• Verified date: June 2021
• Source: Association pour la Recherche en Medecine Interne
• Contact: Patrice CACOUB, MD
• Phone: 01 42 17 80 27
• Email: patrice.cacoub[@]aphp.fr
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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.

Description:

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.

This 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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 500
• Est. completion date: April 2022
• Est. primary completion date: April 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 60 Years

Eligibility

Inclusion Criteria:

• Man or woman aged 18 to 60;

• Presenting severe abdominal pain, afebrile and diffuse over several days, evolving for less than 5 years;

• With at least one of the following symptoms:

• Peripheral neurological symptoms: decreased driving force and pain in the limbs (roots of the thighs), etc. ;

• Central neurological symptoms: psychiatric (depression, anxiety, insomnia), disturbances of consciousness, etc. ;

• Autonomous neurological symptoms: tachycardia, nausea, vomiting, constipation, etc.

• 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;

• Patient able to understand the information related to the study and having indicated his non-objection to the collection of data concerning him;

Exclusion Criteria:

• Patient with a diagnosis of acute hepatic porphyria for more than 6 months;

• Patient unfit to participate in the study, due to cognitive or linguistic difficulties;

• Protected patient (under legal protection, or deprived of liberty by judicial or administrative decision).

Related Conditions & MeSH terms

• Acute Hepatic Porphyria
• Porphyria, Erythropoietic
• Porphyrias

Intervention

Other:
• Dosage of Delta-aminolevulinic acid and Porphobilinogen
Results of dosages of Delta-aminolevulinic acid and Porphobilinogen which are performed in accordance with the current practice will be recorded

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Association pour la Recherche en Medecine Interne

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Binary criterion corresponding to the diagnosis of acute hepatic porphyria (acute hepatic porphyria + / acute hepatic porphyria -) via physiological parameter.	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: Delta-aminolevulinic acid = 3 µmol / mol Cr And or; Porphobilinogen = 1 µmol / mol Cr Otherwise, the patient will be considered to be acute hepatic porphyria free (acute hepatic porphyria -).	1 day