Autoimmune HepatitisNCT04247490
Autoimmune Hepatitis in Pediatric Patients
An observational study of Autoimmune Hepatitis, run by University Hospital of Ferrara at 1 site in Italy.
Trial summary
Autoimmune hepatitis (AIH) is an immune-mediated and inflammatory liver disorder. It is currently divided into types 1 and 2, differentiated and defined by the presence of specific autoantibodies. Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Autoimmune Hepatitis
- What is being tested?
- pediatric AIH
- How many people
- 109 participants
- Where
- 1 siteItaly
- Study dates
- 17 Dec 2019to 31 Dec 2019
- 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 Up to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: The study population is represented by all the patients who presented the onset of Autoimmune hepatitis type 1 and type 2 from 01/01/1989 to 31/12/2019 and were followed at the Pediatric Hepatology Centers of Ferrara (Hospital Saint Anna of Cona), Naples (Federico II University Hospital) and Palermo (Mediterranean Institute for Transplants and Highly Specialized Therapies).
Who may be able to join
- Age ≤ 18 years at the time of diagnosis
- Diagnosis of autoimmune hepatitis type 1 or 2 according to the Mieli-Vergani score: a pre-treatment score> 15 or post-treatment> 17 indicates a definite diagnosis of Autoimmune Hepatitis; a pre-treatment score between 10 and 15 or post-treatment between 12 and 17 indicates a probable diagnosis.
Who cannot join
- Age> 18 years at the time of diagnosis
- Presence of other causes of liver disease
- Presence of overlap syndrome (hepatito-cholangitis)
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Multicentric Study on the Long-term Follow-up of Autoimmune Hepatitis in the Pediatric Patients
Autoimmune hepatitis (AIH) is an immune-mediated and inflammatory liver disorder. It is currently divided into types 1 and 2, differentiated and defined by the presence of specific autoantibodies. The objectives are to describe the prevalence and incidence of type 1 and 2 autoimmune hepatitis and to analyze the clinics, biochemical and histopathological profiles at diagnosis and follow-up, initial therapy, response to therapy and long-term follow-up in three Italian centers of patients with type 1 and type 2 AIH.
Read the detailed description
Background: Autoimmune hepatitis (AIH) is an immune-mediated and inflammatory liver disorder with typical onset in pediatric age. It is currently divided into types 1 and 2, differentiated and defined by the presence of specific autoantibodies. AIH type 1 (AIH-1) is characterised by smooth muscle and/or antinuclear autoantibodies (SMA/ANA) while anti-liver kidney microsomal (anti-LKM) or liver cytosol type 1 (anti-LC1) identified AIH type 2 (AIH-2). The conventional treatment is based on corticosteroids and azathioprine as first line therapy. The optimal duration is not clear definied, however it should be extended for at least five years due to the frequent relapses.
Aims of the study: The primary objective is to describe the prevalence and incidence of type 1 and 2 autoimmune hepatitis on a selected italian pediatric population. Secondary objectives are to analyze the clinical, biochemical and histopathological profiles at diagnosis and follow-up, type of treatment received, response to therapy and long-term follow-up.
Methods: The investigators conducted a retrospective and prospective cohort study on patients aged ≤ 18 years who the diagnosis of AIH 1 and 2 between 01/01/1989 and 31/12/2019 and were followed in three Italian pediatric centers.
- Conditions
- Autoimmune Hepatitis
- Being tested
- pediatric AIH
- Keywords
- Autoimmune hepatitis, incidence, prevalence, immunosuppressive treatmen, liver transplantation
- Medical subject headings
- Hepatitis, Autoimmune
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
pediatric AIHDiagnostic test
pediatric patients with clinical, laboratory (transaminase and IgG increase), instrumental (ultrasound, liver biopsy) characteristics
| Study group | Role | What they receive |
|---|---|---|
| Pediatric AIH | Patients diagnosed with hepatitis type 1 and type 2 formulated between the ages of 0 and 18. Receives: Diagnostic Test: pediatric AIH |
What is being measured
The primary outcome is the main question the study is designed to answer.
Prevalence and incidence
The primary objective was to calculate the proportion of AIH type 1 and 2 patients present in the Italian pediatric population at a given time (prevalence) and the proportion of new AIH type 1 and 2 patients in the Italian pediatric population (0-18 years) in a given period of time (incidence).
Measured: from 01/01/1989 to 31/12/2019
Secondary outcomes (1)
Characteristics of AIH in pediatric patients
Describe the baseline characteristics (age of onset, clinical-laboratory, radiological and histological characteristics), the response to therapy (medical and / or the possible use of liver transplantation) and the follow-up of patients with autoimmune hepatitis type 1 and 2.
Measured: from 01/01/1989 to 31/12/2019
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jan 2020
- Study start17 Dec 2019
- Primary completion31 Dec 2019
- Study completion31 Dec 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (6)Papers the sponsor has linked to this study.
- Other trials for Autoimmune HepatitisStudies 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
- Retrospective
- Enrolment
- 109 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital of Ferrara
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Giuseppe Maggiore, University Hospital of Ferrara
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04247490
- Sponsor's protocol ID
- 637/2019/Oss/AOUFe
- Status last verified
- Jan 2020
- First submitted
- 20 Jan 2020
- Last updated
- 30 Jan 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Jimenez-Rivera C, Ling SC, Ahmed N, Yap J, Aglipay M, Barrowman N, Graitson S, Critch J, Rashid M, Ng VL, Roberts EA, Brill H, Dowhaniuk JK, Bruce G, Bax K, Deneau M, Guttman OR, Schreiber RA, Martin S, Alvarez F. Incidence and Characteristics of Autoimmune Hepatitis. Pediatrics. 2015 Nov;136(5):e1237-48. doi: 10.1542/peds.2015-0578. Epub 2015 Oct 19.
PubMed 26482664Verma S, Torbenson M, Thuluvath PJ. The impact of ethnicity on the natural history of autoimmune hepatitis. Hepatology. 2007 Dec;46(6):1828-35. doi: 10.1002/hep.21884.
PubMed 17705297Nastasio S, Sciveres M, Matarazzo L, Maggiore G. Old and New Treatments for Pediatric Autoimmune Hepatitis. Curr Pediatr Rev. 2018;14(3):187-195. doi: 10.2174/1573396314666180516130314.
PubMed 29766815Maggiore G, Porta G, Bernard O, Hadchouel M, Alvarez F, Homberg JC, Alagille D. Autoimmune hepatitis with initial presentation as acute hepatic failure in young children. J Pediatr. 1990 Feb;116(2):280-2. doi: 10.1016/s0022-3476(05)82892-6. No abstract available.
PubMed 2299503Alvarez F, Berg PA, Bianchi FB, Bianchi L, Burroughs AK, Cancado EL, Chapman RW, Cooksley WG, Czaja AJ, Desmet VJ, Donaldson PT, Eddleston AL, Fainboim L, Heathcote J, Homberg JC, Hoofnagle JH, Kakumu S, Krawitt EL, Mackay IR, MacSween RN, Maddrey WC, Manns MP, McFarlane IG, Meyer zum Buschenfelde KH, Zeniya M, et al. International Autoimmune Hepatitis Group Report: review of criteria for diagnosis of autoimmune hepatitis. J Hepatol. 1999 Nov;31(5):929-38. doi: 10.1016/s0168-8278(99)80297-9. No abstract available.
PubMed 10580593Mieli-Vergani G, Vergani D, Baumann U, Czubkowski P, Debray D, Dezsofi A, Fischler B, Gupte G, Hierro L, Indolfi G, Jahnel J, Smets F, Verkade HJ, Hadzic N. Diagnosis and Management of Pediatric Autoimmune Liver Disease: ESPGHAN Hepatology Committee Position Statement. J Pediatr Gastroenterol Nutr. 2018 Feb;66(2):345-360. doi: 10.1097/MPG.0000000000001801.
PubMed 29356770
Study at a glance
From the registry record, last updated by the sponsor on 30 Jan 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicentric Study on the Long-term Follow-up of Autoimmune Hepatitis in the Pediatric Patients
- ClinicalTrials.gov ID
- NCT04247490
- Acronym / sponsor ID
- 637/2019/Oss/AOUFe
- Conditions
- Autoimmune Hepatitis
- Keywords
- Autoimmune hepatitis, incidence, prevalence, immunosuppressive treatmen, liver transplantation
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 109 participants (actual)
- Lead sponsor
- University Hospital of FerraraUniversity, hospital or other
- Study start
- 17 Dec 2019 (actual)
- Primary completion
- 31 Dec 2019 (actual)
- Study completion
- 31 Dec 2019 (actual)
- First posted
- 30 Jan 2020 (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.
Autoimmune hepatitis (AIH) is an immune-mediated and inflammatory liver disorder. It is currently divided into types 1 and 2, differentiated and defined by the presence of specific autoantibodies. The objectives are to describe the prevalence and incidence of type 1 and 2 autoimmune hepatitis and to analyze the clinics, biochemical and histopathological profiles at diagnosis and follow-up, initial therapy, response to therapy and long-term follow-up in three Italian centers of patients with type 1 and type 2 AIH.
Detailed description
Background: Autoimmune hepatitis (AIH) is an immune-mediated and inflammatory liver disorder with typical onset in pediatric age. It is currently divided into types 1 and 2, differentiated and defined by the presence of specific autoantibodies. AIH type 1 (AIH-1) is characterised by smooth muscle and/or antinuclear autoantibodies (SMA/ANA) while anti-liver kidney microsomal (anti-LKM) or liver cytosol type 1 (anti-LC1) identified AIH type 2 (AIH-2). The conventional treatment is based on corticosteroids and azathioprine as first line therapy. The optimal duration is not clear definied, however it should be extended for at least five years due to the frequent relapses.
Aims of the study: The primary objective is to describe the prevalence and incidence of type 1 and 2 autoimmune hepatitis on a selected italian pediatric population. Secondary objectives are to analyze the clinical, biochemical and histopathological profiles at diagnosis and follow-up, type of treatment received, response to therapy and long-term follow-up.
Methods: The investigators conducted a retrospective and prospective cohort study on patients aged ≤ 18 years who the diagnosis of AIH 1 and 2 between 01/01/1989 and 31/12/2019 and were followed in three Italian pediatric centers.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pediatric AIH | – | Diagnostic Test: pediatric AIHPatients diagnosed with hepatitis type 1 and type 2 formulated between the ages of 0 and 18. |
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- The study population is represented by all the patients who presented the onset of Autoimmune hepatitis type 1 and type 2 from 01/01/1989 to 31/12/2019 and were followed at the Pediatric Hepatology Centers of Ferrara (Hospital Saint Anna of Cona), Naples (Federico II University Hospital) and Palermo (Mediterranean Institute for Transplants and Highly Specialized Therapies).
Key inclusion criteria (2 listed)
- Age ≤ 18 years at the time of diagnosis
- Diagnosis of autoimmune hepatitis type 1 or 2 according to the Mieli-Vergani score: a pre-treatment score> 15 or post-treatment> 17 indicates a definite diagnosis of Autoimmune Hepatitis; a pre-treatment score between 10 and 15 or post-treatment between 12 and 17 indicates a probable diagnosis.
Key exclusion criteria (3 listed)
- Age> 18 years at the time of diagnosis
- Presence of other causes of liver disease
- Presence of overlap syndrome (hepatito-cholangitis)
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 |
|---|---|---|
| pediatric AIHpediatric patients with clinical, laboratory (transaminase and IgG increase), instrumental (ultrasound, liver biopsy) characteristics | Diagnostic test | Pediatric AIH |
Primary outcome
Prevalence and incidence
Time frame: from 01/01/1989 to 31/12/2019
The primary objective was to calculate the proportion of AIH type 1 and 2 patients present in the Italian pediatric population at a given time (prevalence) and the proportion of new AIH type 1 and 2 patients in the Italian pediatric population (0-18 years) in a given period of time (incidence).
1 secondary outcome
Characteristics of AIH in pediatric patients
Time frame: from 01/01/1989 to 31/12/2019
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 30 Jan 2020.
- Background: PubMed 26482664Jimenez-Rivera C, Ling SC, Ahmed N, Yap J, Aglipay M, Barrowman N, Graitson S, Critch J, Rashid M, Ng VL, Roberts EA, Brill H, Dowhaniuk JK, Bruce G, Bax K, Deneau M, Guttman OR, Schreiber RA, Martin S, Alvarez F. Incidence and Characteristics of Autoimmune Hepatitis. Pediatrics. 2015 Nov;136(5):e1237-48. doi: 10.1542/peds.2015-0578. Epub 2015 Oct 19.
- Results publication: PubMed 17705297Verma S, Torbenson M, Thuluvath PJ. The impact of ethnicity on the natural history of autoimmune hepatitis. Hepatology. 2007 Dec;46(6):1828-35. doi: 10.1002/hep.21884.
- Results publication: PubMed 29766815Nastasio S, Sciveres M, Matarazzo L, Maggiore G. Old and New Treatments for Pediatric Autoimmune Hepatitis. Curr Pediatr Rev. 2018;14(3):187-195. doi: 10.2174/1573396314666180516130314.
- Results publication: PubMed 2299503Maggiore G, Porta G, Bernard O, Hadchouel M, Alvarez F, Homberg JC, Alagille D. Autoimmune hepatitis with initial presentation as acute hepatic failure in young children. J Pediatr. 1990 Feb;116(2):280-2. doi: 10.1016/s0022-3476(05)82892-6. No abstract available.
- Results publication: PubMed 10580593Alvarez F, Berg PA, Bianchi FB, Bianchi L, Burroughs AK, Cancado EL, Chapman RW, Cooksley WG, Czaja AJ, Desmet VJ, Donaldson PT, Eddleston AL, Fainboim L, Heathcote J, Homberg JC, Hoofnagle JH, Kakumu S, Krawitt EL, Mackay IR, MacSween RN, Maddrey WC, Manns MP, McFarlane IG, Meyer zum Buschenfelde KH, Zeniya M, et al. International Autoimmune Hepatitis Group Report: review of criteria for diagnosis of autoimmune hepatitis. J Hepatol. 1999 Nov;31(5):929-38. doi: 10.1016/s0168-8278(99)80297-9. No abstract available.
- Results publication: PubMed 29356770Mieli-Vergani G, Vergani D, Baumann U, Czubkowski P, Debray D, Dezsofi A, Fischler B, Gupte G, Hierro L, Indolfi G, Jahnel J, Smets F, Verkade HJ, Hadzic N. Diagnosis and Management of Pediatric Autoimmune Liver Disease: ESPGHAN Hepatology Committee Position Statement. J Pediatr Gastroenterol Nutr. 2018 Feb;66(2):345-360. doi: 10.1097/MPG.0000000000001801.
- 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 Autoimmune Hepatitis, run by University Hospital of Ferrara at 1 site in Italy.
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 2 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 30 Jan 2020. 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 Italy. 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?
Prevalence and incidence (time frame: from 01/01/1989 to 31/12/2019). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04247490, which also lists 6 linked publications.
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 30 Jan 2020.