Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu

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. 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: NCT04483531
Expanded Access Status : Available
First Posted : July 23, 2020
Last Update Posted : August 25, 2020
Sponsor:
Information provided by (Responsible Party):
Albireo

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.

Layout table for study information
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

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.


Layout table for eligibility information
Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Criteria

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


Contacts
Layout table for location contacts
Contact: Clinigen Group 1-877-768-4303 Odevixibat@clinigengroup.com

Locations
Layout table for location information
United States, Massachusetts
Albireo Pharma Inc. Available
Boston, Massachusetts, United States, 02109
Sponsors and Collaborators
Albireo
Layout table for additonal information
Responsible Party: Albireo
ClinicalTrials.gov Identifier: NCT04483531    
Other Study ID Numbers: A4250-014
First Posted: July 23, 2020    Key Record Dates
Last Update Posted: August 25, 2020
Last Verified: August 2020
Keywords provided by Albireo:
PFIC
pediatric
Cholestasis
Additional relevant MeSH terms:
Layout table for MeSH terms
Cholestasis
Cholestasis, Intrahepatic
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Diseases
Liver Diseases