Back to resultsOdevixibat for the Treatment of Progressive Familial Intrahepatic Cholestasis
Primary Purpose
Progressive Familial Intrahepatic Cholestasis
Status
Approved for marketing
Phase
Locations
United States
Study Type
Expanded Access
Intervention
Odevixibat
Sponsored by
About this trial
This is an expanded access trial for Progressive Familial Intrahepatic Cholestasis focused on measuring PFIC, pediatric, Cholestasis
Eligibility Criteria
Inclusion Criteria:
- A male or female patient of any age, with a clinical diagnosis of PFIC, and with a body weight ≥5 kg at Screening visit
- Patient must have a clinical diagnosis of PFIC
- Patient must have clinically confirmed pruritus
- Patient must have elevated serum bile acid levels, specifically measured to be ≥2 × the upper limit of normal (ULN) prior to start of medication
- 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:
- Patient is expected to have a liver transplant within 6 months of Screening
- Decompensated liver disease, coagulopathy, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy
- 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)
- Serum ALT >10 × ULN at Screening
- Serum ALT >15 × ULN at any time point during the last 6 months unless an alternate etiology was confirmed for the elevation
- Total bilirubin >10 × ULN at Screening
- Any patient who is pregnant, lactating, or planning to get pregnant
- Patients who qualify for enrollment in other Phase 2 or Phase 3 trials intended to support marketing approval in PFIC
Sites / Locations
- Albireo Pharma Inc.
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04483531
Brief Title
Odevixibat for the Treatment of Progressive Familial Intrahepatic Cholestasis
Official Title
Odevixibat (A4250) for the Treatment of Progressive Familial Intrahepatic Cholestasis (Expanded Access Program)
Study Type
Expanded Access
2. Study Status
Record Verification Date
July 2021
Overall Recruitment Status
Approved for marketing
Study Start Date
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Primary Completion Date
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Study Completion Date
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3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Albireo
4. Oversight
5. Study Description
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
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Progressive Familial Intrahepatic Cholestasis
Keywords
PFIC, pediatric, Cholestasis
7. Study Design
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
Odevixibat
Other Intervention Name(s)
A4250
Intervention Description
A4250 is a small molecule and selective inhibitor of IBAT
10. Eligibility
Sex
All
Eligibility Criteria
Inclusion Criteria:
A male or female patient of any age, with a clinical diagnosis of PFIC, and with a body weight ≥5 kg at Screening visit
Patient must have a clinical diagnosis of PFIC
Patient must have clinically confirmed pruritus
Patient must have elevated serum bile acid levels, specifically measured to be ≥2 × the upper limit of normal (ULN) prior to start of medication
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:
Patient is expected to have a liver transplant within 6 months of Screening
Decompensated liver disease, coagulopathy, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy
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)
Serum ALT >10 × ULN at Screening
Serum ALT >15 × ULN at any time point during the last 6 months unless an alternate etiology was confirmed for the elevation
Total bilirubin >10 × ULN at Screening
Any patient who is pregnant, lactating, or planning to get pregnant
Patients who qualify for enrollment in other Phase 2 or Phase 3 trials intended to support marketing approval in PFIC
Facility Information:
Facility Name
Albireo Pharma Inc.
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02109
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Odevixibat for the Treatment of Progressive Familial Intrahepatic Cholestasis
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