Acute Hepatic PorphyriaPHANCT04923516
Prevalence of Acute Hepatic Porphyria
An observational study of Acute Hepatic Porphyria, run by Association pour la Recherche en Medecine Interne at 1 site in France.
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. Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Acute Hepatic Porphyria
- What is being tested?
- Dosage of Delta-aminolevulinic acid and Porphobilinogen
- How many people
- 150 participants
- Where
- 1 siteFrance
- Study dates
- 23 Aug 2021to 29 Dec 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 60 years
- All sexes
- No healthy volunteers
- Adult
Study population: 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.
Who may be able to join
- 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;
Who cannot join
- 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).
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Prevalence of Acute Hepatic Porphyria in Population With Suggestive Clinical Picture
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.
Read the detailed 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.
- Conditions
- Acute Hepatic Porphyria
- Being tested
- Dosage of Delta-aminolevulinic acid and Porphobilinogen
- Medical subject headings
- Coproporphyria, Hereditary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Dosage of Delta-aminolevulinic acid and PorphobilinogenOther
Results of dosages of Delta-aminolevulinic acid and Porphobilinogen which are performed in accordance with the current practice will be recorded
What is being measured
The primary outcome is the main question the study is designed to answer.
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 -).
Measured: 1 day
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jun 2021
- Study start23 Aug 2021
- Primary completion29 Dec 2023
- Study completion29 Dec 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Hepatic PorphyriaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Enrolment
- 150 participants (actual)
Sponsor and investigators
- Lead sponsor
- Association pour la Recherche en Medecine Interne
- Sponsor type
- Research network
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04923516
- Sponsor's protocol ID
- 2021-A00566-35
- Acronym
- PHA
- Status last verified
- Jan 2026
- First submitted
- 8 Jun 2021
- Last updated
- 28 Jan 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prevalence of Acute Hepatic Porphyria in Population With Suggestive Clinical Picture
- ClinicalTrials.gov ID
- NCT04923516
- Acronym / sponsor ID
- PHA / 2021-A00566-35
- Conditions
- Acute Hepatic Porphyria
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 150 participants (actual)
- Lead sponsor
- Association pour la Recherche en Medecine InterneResearch network
- Study start
- 23 Aug 2021 (actual)
- Primary completion
- 29 Dec 2023 (actual)
- Study completion
- 29 Dec 2023 (actual)
- First posted
- 11 Jun 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed 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.
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
- Study population
- 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.
Key inclusion criteria (8 listed)
- 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:
- 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;
Key exclusion criteria (3 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Dosage of Delta-aminolevulinic acid and PorphobilinogenResults of dosages of Delta-aminolevulinic acid and Porphobilinogen which are performed in accordance with the current practice will be recorded | Other | – |
Primary outcome
Binary criterion corresponding to the diagnosis of acute hepatic porphyria (acute hepatic porphyria + / acute hepatic porphyria -) via physiological parameter.
Time frame: 1 day
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 -).
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Jan 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Acute Hepatic Porphyria, run by Association pour la Recherche en Medecine Interne at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 28 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Binary criterion corresponding to the diagnosis of acute hepatic porphyria (acute hepatic porphyria + / acute hepatic porphyria -) via physiological parameter. (time frame: 1 day). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Jan 2026.