Pediatric CholestasisPhase 2NCT02630875
A4250, an IBAT Inhibitor in Pediatric Cholestasis
A Phase 2 study testing A4250 in people with Pediatric Cholestasis, run by Albireo at 7 sites in 5 countries.
Trial summary
This study will evaluate A4250 (IBATinhibitor) as a treatment option in pediatric patients with chronic cholestasis with main emphasis on safety evaluation and on effects on pruritus Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- Pediatric Cholestasis
- What is being tested?
- A4250
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 7 sitesin 5 countries
- Study dates
- Aug 2015to 17 Mar 2017
- Phase
- Phase 2
- 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 1 year to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosis of pruritus due to chronic cholestasis based on history and investigator judgment. This will include but will not be restricted to patients with Progressive familial intrahepatic cholestasis (PFIC), Alagille syndrome (ALGS), Biliary Atresia and Sclerosing Cholangitis
Who cannot join
- Any condition that in the opinion of the investigator constitutes a risk for the patient or a contraindication for participation and completion of the study, or could interfere with study objectives, conduct, or evaluations
- Clinical or biochemical signs of decompensated liver disease
- Liver transplantation
Trial locations and contacts
7 sites in 5 countries.
7 sites in 5 countries
France2 sites
Pediatric Hepatology and Liver Transplantation, University Hospitals of Paris-Sud
Paris, Orsay
Department of Pediatric Gastroenterology Hepatology-Nutrition, Necker-Enfants maladies hospital
Paris
Germany2 sites
Pediatric Gastroenterology and Hepatology, Pediatric Surgery, Hannover
Hanover
Gastroenterology/Hepatology, University Hospital for Children and Adolescents
Tübingen
Denmark1 site
Department of Pediatric and Adolescent Medicine Rigshospitalet
Copenhagen
United Kingdom1 site
King's College Hospital NHS Foundation Trust
London
About this study
Official title: An Exploratory Phase II Study to Demonstrate the Safety and Efficacy of A4250 in Children With Cholestatic Pruritus
This study will evaluate A4250 (IBATinhibitor) as a treatment option in pediatric patients with chronic cholestasis with main emphasis on safety evaluation and on effects on pruritus
Read the detailed description
The primary aims of this Phase II exploratory study in patients treated with A4250 due to cholestasis induced pruritus are to:
- Assess the safety and tolerability of A4250, orally administered first as a single dose and then during a four week treatment period, as determined by the occurrence of treatment-emergent SAEs
- Explore changes in serum total bile acids after a four week treatment period
Secondary safety aims include assessment of the safety and tolerability of A4250, orally administered first as a single dose and then during a four week treatment period, as determined by the occurrence of treatment-emergent AEs and changes in safety parameters including laboratory tests and vital signs
Secondary efficacy aims are to:
- Demonstrate the efficacy of A4250, orally administered during a four week treatment period, on liver biochemistry variables and on pruritus parameters
- Evaluate the pharmacokinetic properties of A4250 orally administered first as a single dose and then after a four week treatment period
- Evaluate changes in VAS-itching score after a four week treatment period
- Conditions
- Pediatric Cholestasis
- Being tested
- A4250
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
A4250Drug
A4250
| Study group | Role | What they receive |
|---|---|---|
| A4250 1 | Active comparator | Dose I Receives: Drug: A4250 |
| A4250 2 | Active comparator | Dose 2 Receives: Drug: A4250 |
| A4250 3 | Active comparator | Dose 3 Receives: Drug: A4250 |
| A4250 4 | Active comparator | Dose 4 Receives: Drug: A4250 |
| A4250 5 | Active comparator | Dose 5 Receives: Drug: A4250 |
| A4250 6 | Active comparator | Dose 6 Receives: Drug: A4250 |
What is being measured
The primary outcome is the main question the study is designed to answer.
AE evaluation
Treatment-emergent SAEs Adverse events
Measured: 4 wks
Secondary outcomes (1)
Bile acid changes
Evaluation of bile acids
Measured: 4 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Dec 2015 (estimated)
- Study startAug 2015
- Primary completion17 Mar 2017
- Study completion17 Mar 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pediatric CholestasisStudies 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Albireo
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ipsen Medical Director, Study director, Ipsen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02630875
- Sponsor's protocol ID
- A4250-003
- Status last verified
- Mar 2024
- First submitted
- 23 Nov 2015
- Last updated
- 7 Mar 2024
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, annotated case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of study participants. Any requests should be submitted to www.vivli.org for assessment by an independent scientific review board.
Study at a glance
From the registry record, last updated by the sponsor on 7 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Exploratory Phase II Study to Demonstrate the Safety and Efficacy of A4250 in Children With Cholestatic Pruritus
- ClinicalTrials.gov ID
- NCT02630875
- Acronym / sponsor ID
- A4250-003
- Conditions
- Pediatric Cholestasis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- AlbireoIndustry (pharma, biotech, devices)
- Study start
- Aug 2015
- Primary completion
- 17 Mar 2017 (actual)
- Study completion
- 17 Mar 2017 (actual)
- First posted
- 15 Dec 2015 (estimated)
- 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.
This study will evaluate A4250 (IBATinhibitor) as a treatment option in pediatric patients with chronic cholestasis with main emphasis on safety evaluation and on effects on pruritus
Detailed description
The primary aims of this Phase II exploratory study in patients treated with A4250 due to cholestasis induced pruritus are to:
- Assess the safety and tolerability of A4250, orally administered first as a single dose and then during a four week treatment period, as determined by the occurrence of treatment-emergent SAEs
- Explore changes in serum total bile acids after a four week treatment period
Secondary safety aims include assessment of the safety and tolerability of A4250, orally administered first as a single dose and then during a four week treatment period, as determined by the occurrence of treatment-emergent AEs and changes in safety parameters including laboratory tests and vital signs
Secondary efficacy aims are to:
- Demonstrate the efficacy of A4250, orally administered during a four week treatment period, on liver biochemistry variables and on pruritus parameters
- Evaluate the pharmacokinetic properties of A4250 orally administered first as a single dose and then after a four week treatment period
- Evaluate changes in VAS-itching score after a four week treatment period
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| A4250 1 | Active comparator | Drug: A4250Dose I |
| A4250 2 | Active comparator | Drug: A4250Dose 2 |
| A4250 3 | Active comparator | Drug: A4250Dose 3 |
| A4250 4 | Active comparator | Drug: A4250Dose 4 |
| A4250 5 | Active comparator | Drug: A4250Dose 5 |
| A4250 6 | Active comparator | Drug: A4250Dose 6 |
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (1 listed)
- Diagnosis of pruritus due to chronic cholestasis based on history and investigator judgment. This will include but will not be restricted to patients with Progressive familial intrahepatic cholestasis (PFIC), Alagille syndrome (ALGS), Biliary Atresia and Sclerosing Cholangitis
Key exclusion criteria (3 listed)
- Any condition that in the opinion of the investigator constitutes a risk for the patient or a contraindication for participation and completion of the study, or could interfere with study objectives, conduct, or evaluations
- Clinical or biochemical signs of decompensated liver disease
- Liver transplantation
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 |
|---|---|---|
| A4250A4250 | Drug | A4250 1, A4250 2, A4250 3, A4250 4, A4250 5, A4250 6 |
Primary outcome
AE evaluation
Time frame: 4 wks
Treatment-emergent SAEs Adverse events
1 secondary outcome
Bile acid changes
Time frame: 4 weeks
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 7 Mar 2024.
- Individual participant dataSharing planned: Yes. Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, annotated case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of study participants. Any requests should be submitted to www.vivli.org for assessment by an independent scientific review board.
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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.
Ipsen Medical Director study director
Ipsen
7 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing A4250 in people with Pediatric Cholestasis, run by Albireo at 7 sites in 5 countries.
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 1 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 7 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in France, Germany, Denmark, Sweden, United Kingdom. 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
AE evaluation (time frame: 4 wks). 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 7 Mar 2024.