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Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFIC

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ClinicalTrials.gov Identifier: NCT03659916
Recruitment Status : Recruiting
First Posted : September 6, 2018
Last Update Posted : January 21, 2020
Sponsor:
Information provided by (Responsible Party):
Albireo

Tracking Information
First Submitted Date  ICMJE August 24, 2018
First Posted Date  ICMJE September 6, 2018
Last Update Posted Date January 21, 2020
Actual Study Start Date  ICMJE September 28, 2018
Estimated Primary Completion Date December 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 6, 2019)
  • Change in Pruritus [ Time Frame: From baseline over 72 weeks ]
    Change in pruritus as indexed by caregiver-reported (Albireo ObsRO instrument) observed scratching
  • Change in serum bile acids [ Time Frame: From baseline up to week 72 ]
Original Primary Outcome Measures  ICMJE
 (submitted: September 4, 2018)
  • Change in Pruritus [ Time Frame: From baseline up to week 72 ]
    Change in pruritus as indexed by caregiver-reported (Albireo ObsRO instrument) observed scratching
  • Change in serum bile acids [ Time Frame: From baseline up to week 72 ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: September 6, 2019)
  • All-cause mortality [ Time Frame: From baseline to weeks 24, 48, and 72 ]
  • Number of patients undergoing biliary diversion surgery [ Time Frame: From baseline to weeks 24, 48, and 72 ]
  • Number of patients undergoing liver transplantation [ Time Frame: From baseline to weeks 24, 48, and 72 ]
  • Change in growth [ Time Frame: From baseline to weeks 24, 48, and 72 ]
    The linear growth deficit compared to the standard growth curve
  • Change in AST to platelet ratio idex (APRI) score [ Time Frame: From baseline to week 72 ]
  • Change in Fib-4 score [ Time Frame: From baseline to week 72 ]
  • Change in pediatric end-stage liver disease (PELD)/model for end-stage liver disease (MELD) score [ Time Frame: From baseline to week 72 ]
  • Change in use of antipruritic medication [ Time Frame: From baseline to weeks 24, 48, and 72 ]
Original Secondary Outcome Measures  ICMJE
 (submitted: September 4, 2018)
  • All-cause mortality [ Time Frame: From baseline to weeks 24, 48, and 72 ]
  • Number of patients undergoing biliary diversion surgery [ Time Frame: From baseline to weeks 24, 48, and 72 ]
  • Number of patients being listed for liver transplantation [ Time Frame: From baseline to weeks 24, 48, and 72 ]
  • Change in growth [ Time Frame: From baseline to weeks 24, 48, and 72 ]
    The linear growth deficit compared to the standard growth curve
  • Change in AST to platelet ratio idex (APRI) score [ Time Frame: From baseline to week 72 ]
  • Change in Fib-4 score [ Time Frame: From baseline to week 72 ]
  • Change in pediatric end-stage liver disease (PELD)/model for end-stage liver disease (MELD) score [ Time Frame: From baseline to week 72 ]
  • Change in use of antipruritic medication [ Time Frame: From baseline to weeks 24, 48, and 72 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFIC
Official Title  ICMJE 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)
Brief Summary Open Label Extension Study to evaluate long term safety and persistence of effect of A4250 in children with PFIC.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Progressive Familial Intrahepatic Cholestasis
Intervention  ICMJE Drug: A4250 (odevixibat)
A4250 is a small molecule and selective inhibitor of IBAT
Study Arms  ICMJE Experimental: A4250
Capsules for oral administration (120 ug/kg) once daily for 72 weeks
Intervention: Drug: A4250 (odevixibat)
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: September 6, 2019)
120
Original Estimated Enrollment  ICMJE
 (submitted: September 4, 2018)
60
Estimated Study Completion Date  ICMJE December 2021
Estimated Primary Completion Date December 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria Cohort 1:

  1. Completion of the 24-week Treatment Period of Study A4250-005 or withdrawn from Study A4250-005 due to patient/caregiver judgment of 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

Inclusion Criteria Cohort 2:

  1. A male or female patient with a clinical diagnosis of PFIC and with a body weight ≥5 kg
  2. Patient must have clinical genetic confirmation of PFIC
  3. Patient must have elevated serum bile acid levels
  4. Patient must have history of significant pruritus
  5. Age appropriate patients are expected to have a consistent caregiver for the duration of the study
  6. Caregivers and age-appropriate patients (≥8 years of age) must be willing and able to use an eDiary device as required by the study

Exclusion Criteria Cohort 1:

  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 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

Exclusion Criteria Cohort 2:

  1. Known pathologic variations of the ABCB11 gene that have been demonstrated to result in complete absence of the BSEP protein
  2. Patient with past medical history or ongoing presence of other types of liver disease including, but not limited to, the following:

    1. Biliary atresia of any kind
    2. Benign recurrent intrahepatic cholestasis, indicated by any history of normal serum bile acids
    3. Suspected or proven liver cancer or metastasis to the liver on imaging studies
    4. Histopathology on liver biopsy is suggestive of alternate non-PFIC related etiology of cholestasis
  3. Patient with past medical history or ongoing chronic (i.e., >3 months) diarrhea
  4. Any patient with suspected or confirmed cancers except for basal cell carcinoma
  5. Patient has had a liver transplant, or a liver transplant is planned within 6 months of the Screening/Inclusion Visit
  6. Decompensated liver disease
  7. Patient suffers from uncontrolled, recalcitrant pruritic condition other than PFIC
  8. Patient previously treated with an IBAT inhibitor and whose pruritus did not respond to treatment
  9. Sexually active males and females who are not using a reliable contraceptive method with ≤1% failure rate (such as hormonal contraception, intra-uterine device, or complete abstinence) throughout the duration of the study and 90 days thereafter
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE up to 100 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Albireo Pharma (857) 378-2035 medinfo@albireopharma.com
Listed Location Countries  ICMJE Australia,   Belgium,   Canada,   France,   Germany,   Israel,   Italy,   Netherlands,   Poland,   Spain,   Sweden,   Turkey,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03659916
Other Study ID Numbers  ICMJE A4250-008
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Albireo
Study Sponsor  ICMJE Albireo
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Albireo
Verification Date January 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP