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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03659916




Registration number
NCT03659916
Ethics application status
Date submitted
24/08/2018
Date registered
6/09/2018
Date last updated
2/08/2019

Titles & IDs
Public title
Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFIC Types 1 & 2
Scientific title
An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children With Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2)
Secondary ID [1] 0 0
A4250-008
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Progressive Familial Intrahepatic Cholestasis Type 1 (PFIC1) 0 0
Progressive Familial Intrahepatic Cholestasis Type 2 (PFIC2) 0 0
Condition category
Condition code
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - A4250

Experimental: A4250 - Capsules for oral administration (120 ug/kg) once daily for 72 weeks


Treatment: Drugs: A4250
A4250 is a small molecule and selective inhibitor of IBAT

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change in Pruritus - Change in pruritus as indexed by caregiver-reported (Albireo ObsRO instrument) observed scratching
Timepoint [1] 0 0
From baseline up to week 72
Primary outcome [2] 0 0
Change in serum bile acids
Timepoint [2] 0 0
From baseline up to week 72
Secondary outcome [1] 0 0
All-cause mortality
Timepoint [1] 0 0
From baseline to weeks 24, 48, and 72
Secondary outcome [2] 0 0
Number of patients undergoing biliary diversion surgery
Timepoint [2] 0 0
From baseline to weeks 24, 48, and 72
Secondary outcome [3] 0 0
Number of patients being listed for liver transplantation
Timepoint [3] 0 0
From baseline to weeks 24, 48, and 72
Secondary outcome [4] 0 0
Change in growth - The linear growth deficit compared to the standard growth curve
Timepoint [4] 0 0
From baseline to weeks 24, 48, and 72
Secondary outcome [5] 0 0
Change in AST to platelet ratio idex (APRI) score
Timepoint [5] 0 0
From baseline to week 72
Secondary outcome [6] 0 0
Change in Fib-4 score
Timepoint [6] 0 0
From baseline to week 72
Secondary outcome [7] 0 0
Change in pediatric end-stage liver disease (PELD)/model for end-stage liver disease (MELD) score
Timepoint [7] 0 0
From baseline to week 72
Secondary outcome [8] 0 0
Change in use of antipruritic medication
Timepoint [8] 0 0
From baseline to weeks 24, 48, and 72

Eligibility
Key inclusion criteria
1. Completion of the 24-week Treatment Period of Study A4250-005 or withdrawn from Study
A4250-005 due to patient/caregiver judgment of lack of improvement/intolerable
symptoms after completing at least 12 weeks of treatment

2. Signed informed consent and assent as appropriate

3. Patients expected to have a consistent caregiver for the duration of the study

4. Caregivers (and age appropriate patients) must be willing and able to use an eDiary
device as required by the study
Minimum age
9 Months
Maximum age
19 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Decompensated liver disease: coagulopathy, history, or presence of clinically
significant ascites, variceal hemorrhage, and/or encephalopathy

2. Sexually active males and females who are not using a reliable contraceptive method
with =1% failure rate (such as barrier protection, hormonal contraception,
intra-uterine device, or complete abstinence) throughout the duration of the study and
90 days thereafter

3. Patients not compliant with treatment in study A4250-005

4. Any other conditions or abnormalities which, in the opinion of the investigator or
Medical Monitor, may compromise the safety of the patient, or interfere with the
patient participating in or completing the study

Study design
Purpose of the study
Treatment
Allocation to intervention
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
The Royal Children's Hospital - Melbourne
Recruitment postcode(s) [1] 0 0
- Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Pennsylvania
Country [2] 0 0
France
State/province [2] 0 0
Bron
Country [3] 0 0
France
State/province [3] 0 0
Le Kremlin-Bicêtre
Country [4] 0 0
France
State/province [4] 0 0
Marseille
Country [5] 0 0
France
State/province [5] 0 0
Paris
Country [6] 0 0
Germany
State/province [6] 0 0
Hannover
Country [7] 0 0
Germany
State/province [7] 0 0
Tübingen
Country [8] 0 0
Italy
State/province [8] 0 0
Torino
Country [9] 0 0
Netherlands
State/province [9] 0 0
Groningen
Country [10] 0 0
Poland
State/province [10] 0 0
Warsaw
Country [11] 0 0
Turkey
State/province [11] 0 0
Ankara
Country [12] 0 0
United Kingdom
State/province [12] 0 0
Birmingham
Country [13] 0 0
United Kingdom
State/province [13] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Albireo
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Open Label Extension Study to evaluate long term safety and persistence of effect of A4250 in
children with PFIC types 1 & 2. Study population will comprise subjects who have participated
in the pivotal study A4250-005.
Trial website
https://clinicaltrials.gov/show/NCT03659916
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Albireo Pharma
Address 0 0
Country 0 0
Phone 0 0
(857) 254-5555
Fax 0 0
Email 0 0
medinfo@albireopharma.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT03659916