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Odevixibat for the Treatment of Progressive Familial Intrahepatic Cholestasis

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. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT04483531
Expanded Access Status : Approved for marketing
First Posted : July 23, 2020
Last Update Posted : July 29, 2021
Information provided by (Responsible Party):

Brief Summary:
To provide treatment access to patients with PFIC in the US who have pruritus and elevated serum bile acids and who are not able to enroll in A4250-008 (PEDFIC2) for the following reasons: 1) Do not meet eligibility criteria for PEDFIC 2; 2) Are not able to get to a PEDFIC 2 site for geographical reasons, and 3) Do meet the eligibility criteria for PEDFIC 2 after recruitment has been completed

Condition or disease Intervention/treatment
Progressive Familial Intrahepatic Cholestasis Drug: Odevixibat

Detailed Description:
Eligible patients will be enrolled into this expanded-access program and treated with an oral dose of 120 μg/kg/day of odevixibat and evaluated on an ongoing basis.

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Study Type : Expanded Access
Expanded Access Type : Treatment IND/Protocol
Official Title: Odevixibat (A4250) for the Treatment of Progressive Familial Intrahepatic Cholestasis (Expanded Access Program)

Intervention Details:
  • Drug: Odevixibat
    A4250 is a small molecule and selective inhibitor of IBAT
    Other Name: A4250

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All

Inclusion Criteria:

  1. A male or female patient of any age, with a clinical diagnosis of PFIC, and with a body weight ≥5 kg at Screening visit
  2. Patient must have a clinical diagnosis of PFIC
  3. Patient must have clinically confirmed pruritus
  4. Patient must have elevated serum bile acid levels, specifically measured to be ≥2 × the upper limit of normal (ULN) prior to start of medication
  5. Patient and/or legal guardian must sign informed consent (and assent) as appropriate. Patients who turn 18 years of age (or legal age per country) during

Exclusion Criteria:

  1. Patient is expected to have a liver transplant within 6 months of Screening
  2. Decompensated liver disease, coagulopathy, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy
  3. International normalized ratio (INR) >1.4 (the patient may be treated with Vitamin K intravenously, and if INR is ≤1.4 at resampling the patient may be started on program medication)
  4. Serum ALT >10 × ULN at Screening
  5. Serum ALT >15 × ULN at any time point during the last 6 months unless an alternate etiology was confirmed for the elevation
  6. Total bilirubin >10 × ULN at Screening
  7. Any patient who is pregnant, lactating, or planning to get pregnant
  8. Patients who qualify for enrollment in other Phase 2 or Phase 3 trials intended to support marketing approval in PFIC

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): NCT04483531

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United States, Massachusetts
Albireo Pharma Inc.
Boston, Massachusetts, United States, 02109
Sponsors and Collaborators
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Responsible Party: Albireo
ClinicalTrials.gov Identifier: NCT04483531    
Other Study ID Numbers: A4250-014
First Posted: July 23, 2020    Key Record Dates
Last Update Posted: July 29, 2021
Last Verified: July 2021
Keywords provided by Albireo:
Additional relevant MeSH terms:
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Cholestasis, Intrahepatic
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Diseases
Liver Diseases