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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
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

Brief Summary:
Open Label Extension Study to evaluate long term safety and persistence of effect of A4250 in children with PFIC.

Condition or disease Intervention/treatment Phase
Progressive Familial Intrahepatic Cholestasis Drug: A4250 (odevixibat) Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 120 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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)
Actual Study Start Date : September 28, 2018
Estimated Primary Completion Date : December 2021
Estimated Study Completion Date : December 2021


Arm Intervention/treatment
Experimental: A4250
Capsules for oral administration (120 ug/kg) once daily for 72 weeks
Drug: A4250 (odevixibat)
A4250 is a small molecule and selective inhibitor of IBAT




Primary Outcome Measures :
  1. Change in Pruritus [ Time Frame: From baseline over 72 weeks ]
    Change in pruritus as indexed by caregiver-reported (Albireo ObsRO instrument) observed scratching

  2. Change in serum bile acids [ Time Frame: From baseline up to week 72 ]

Secondary Outcome Measures :
  1. All-cause mortality [ Time Frame: From baseline to weeks 24, 48, and 72 ]
  2. Number of patients undergoing biliary diversion surgery [ Time Frame: From baseline to weeks 24, 48, and 72 ]
  3. Number of patients undergoing liver transplantation [ Time Frame: From baseline to weeks 24, 48, and 72 ]
  4. Change in growth [ Time Frame: From baseline to weeks 24, 48, and 72 ]
    The linear growth deficit compared to the standard growth curve

  5. Change in AST to platelet ratio idex (APRI) score [ Time Frame: From baseline to week 72 ]
  6. Change in Fib-4 score [ Time Frame: From baseline to week 72 ]
  7. Change in pediatric end-stage liver disease (PELD)/model for end-stage liver disease (MELD) score [ Time Frame: From baseline to week 72 ]
  8. Change in use of antipruritic medication [ Time Frame: From baseline to weeks 24, 48, and 72 ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   up to 100 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03659916


Contacts
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Contact: Albireo Pharma (857) 378-2035 medinfo@albireopharma.com

Locations
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Sponsors and Collaborators
Albireo
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Responsible Party: Albireo
ClinicalTrials.gov Identifier: NCT03659916    
Other Study ID Numbers: A4250-008
First Posted: September 6, 2018    Key Record Dates
Last Update Posted: January 21, 2020
Last Verified: January 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Albireo:
Pediatric
Cholestasis
Additional relevant MeSH terms:
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Cholestasis
Cholestasis, Intrahepatic
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Diseases
Liver Diseases