RECRUITING

Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome

Conditions

Quick Navigation

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

The purpose of this study is to assess the long-term safety and effectiveness of odevixibat in participants with Alagille syndrome (ALGS). The participants of this study will have ALGS a rare genetic disorder that can affect multiple organ systems of the body including the liver, heart, skeleton, eyes and kidneys. Common symptoms, which often develop during the first three months of life, include blockage of the flow of bile from the liver (cholestasis), yellowing of the skin and mucous membranes (jaundice), poor weight gain and growth and severe itching (pruritis). The drug used for the study is odevixibat and was authorized for the treatment of cholestatic pruritus in infants with ALGS over 12 months of age by the United States Food and Drug Administration on 13 June 2023.

Official Title

An Open Label Study to Evaluate the Long-term Safety and Efficacy of Odevixibat (A4250) in Patients With Alagille Syndrome (ASSERT-EXT)

Quick Facts

Study Start:2021-09-03

Study Completion:2027-04-16

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT05035030

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:Not specified

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
1. Completion of the 24-week Treatment Period of Study A4250-012 2. Signed informed consent and assent as appropriate. Patients who turn 18 years of age (or legal age per country) during the study will be required to re-consent to remain on the study 3. Caregivers (and age-appropriate patients) must be willing and able to use an electronic diary (eDiary) device as required by the study 4. Sexually active males and females must agree to use a reliable contraceptive method with ≤1% failure rate (such as hormonal contraception, intra-uterine device, or complete abstinence) from signed informed consent through 90 days after last dose of study drug. 1. Infant with clinically confirmed ALGS , ≤11 months of age at Study Day 1 2. Body weight ≥2 kg at Study Day 1 3. Gestational age ≥36 weeks. For children born with gestational age between 32 and 36 weeks, a postmenstrual age of ≥36 weeks is required . 4. Signed parent/legal guardian informed consent.	1. Decompensated liver disease, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy 2. Patients who were not compliant with study drug treatment or procedures in Study A4250-012 3. Any other conditions or abnormalities which, in the opinion of the investigator, may compromise the safety of the patient, or interfere with the patient participating in or completing the study 4. Known hypersensitivity to any components of odevixibat 1. 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. Progressive familial intrahepatic cholestasis (PFIC) 3. Benign recurrent intrahepatic cholestasis 2. Patient with a past medical history or ongoing presence of any other disease or condition known to interfere with the absorption, distribution, metabolism (specifically bile acid metabolism), or excretion of drugs in the intestine, including but not limited to, inflammatory bowel disease 3. Patient with past medical history or ongoing chronic diarrhea requiring intravenous fluid or nutritional intervention for treatment of the diarrhea and/or its sequelae 4. Patient has a confirmed past diagnosis of infection with human immunodeficiency virus or other present and active, clinically significant chronic infection 5. Recent infection requiring hospitalization or treatment with parenteral anti-infective within 4 weeks of Study Day 1 or completion of oral anti-infective treatment within 2 weeks prior to the Screening Visit 6. Cancer diagnosis (except for basal cell carcinoma) 7. Chronic kidney disease with an impaired renal function and a glomerular filtration rate \<70 mL/min/1.73 m2 8. Patient with surgical history of disruption of the enterohepatic circulation (biliary diversion surgery) within 6 months prior to the Screening Visit 9. Patient has had a liver transplant, or a liver transplant is planned within 6 months of Study Day 1 10. Decompensated liver disease, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy 11. International normalized ratio (INR) \>1.4 (the patient may be treated with Vitamin K, and if INR is ≤1.4 at resampling the patient may be enrolled) 12. Serum alanine aminotransferase (ALT) \>10 × upper limit of normal (ULN) at Screening 13. Serum ALT \>15 × ULN at any time point during the last 6 months unless an alternate etiology was confirmed for the elevation 14. Total bilirubin \>15 × ULN at Screening 15. Patient suffers from uncontrolled, recalcitrant pruritic condition other than ALGS. Examples include, but not limited to, refractory atopic dermatitis or other primary pruritic skin diseases. 16. Patient exposed to alcohol or substance abuse in utero 17. Bile acid or lipid binding resins and medications that slow gastrointestinal motility 18. Patient has had investigational exposure to a drug, biologic agent, or medical device within 30 days prior to the Screening Visit, or 5 half-lives of the study agent, whichever is longer 19. Any other conditions or abnormalities which, in the opinion of the investigator may compromise the safety of the patient, or interfere with the patient participating in or completing the study

Inclusion Criteria

Exclusion Criteria

1. Completion of the 24-week Treatment Period of Study A4250-012
2. Signed informed consent and assent as appropriate. Patients who turn 18 years of age (or legal age per country) during the study will be required to re-consent to remain on the study
3. Caregivers (and age-appropriate patients) must be willing and able to use an electronic diary (eDiary) device as required by the study
4. Sexually active males and females must agree to use a reliable contraceptive method with ≤1% failure rate (such as hormonal contraception, intra-uterine device, or complete abstinence) from signed informed consent through 90 days after last dose of study drug.
1. Infant with clinically confirmed ALGS , ≤11 months of age at Study Day 1
2. Body weight ≥2 kg at Study Day 1
3. Gestational age ≥36 weeks. For children born with gestational age between 32 and 36 weeks, a postmenstrual age of ≥36 weeks is required .
4. Signed parent/legal guardian informed consent.

1. Decompensated liver disease, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy
2. Patients who were not compliant with study drug treatment or procedures in Study A4250-012
3. Any other conditions or abnormalities which, in the opinion of the investigator, may compromise the safety of the patient, or interfere with the patient participating in or completing the study
4. Known hypersensitivity to any components of odevixibat
1. 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. Progressive familial intrahepatic cholestasis (PFIC)
3. Benign recurrent intrahepatic cholestasis
2. Patient with a past medical history or ongoing presence of any other disease or condition known to interfere with the absorption, distribution, metabolism (specifically bile acid metabolism), or excretion of drugs in the intestine, including but not limited to, inflammatory bowel disease
3. Patient with past medical history or ongoing chronic diarrhea requiring intravenous fluid or nutritional intervention for treatment of the diarrhea and/or its sequelae
4. Patient has a confirmed past diagnosis of infection with human immunodeficiency virus or other present and active, clinically significant chronic infection
5. Recent infection requiring hospitalization or treatment with parenteral anti-infective within 4 weeks of Study Day 1 or completion of oral anti-infective treatment within 2 weeks prior to the Screening Visit
6. Cancer diagnosis (except for basal cell carcinoma)
7. Chronic kidney disease with an impaired renal function and a glomerular filtration rate \<70 mL/min/1.73 m2
8. Patient with surgical history of disruption of the enterohepatic circulation (biliary diversion surgery) within 6 months prior to the Screening Visit
9. Patient has had a liver transplant, or a liver transplant is planned within 6 months of Study Day 1
10. Decompensated liver disease, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy
11. International normalized ratio (INR) \>1.4 (the patient may be treated with Vitamin K, and if INR is ≤1.4 at resampling the patient may be enrolled)
12. Serum alanine aminotransferase (ALT) \>10 × upper limit of normal (ULN) at Screening
13. Serum ALT \>15 × ULN at any time point during the last 6 months unless an alternate etiology was confirmed for the elevation
14. Total bilirubin \>15 × ULN at Screening
15. Patient suffers from uncontrolled, recalcitrant pruritic condition other than ALGS. Examples include, but not limited to, refractory atopic dermatitis or other primary pruritic skin diseases.
16. Patient exposed to alcohol or substance abuse in utero
17. Bile acid or lipid binding resins and medications that slow gastrointestinal motility
18. Patient has had investigational exposure to a drug, biologic agent, or medical device within 30 days prior to the Screening Visit, or 5 half-lives of the study agent, whichever is longer
19. Any other conditions or abnormalities which, in the opinion of the investigator may compromise the safety of the patient, or interfere with the patient participating in or completing the study

Contacts and Locations

Study Contact

Ipsen Recruitment enquiries

CONTACT

See e mail

clinical.trials@ipsen.com

Principal Investigator

Ipsen Medical Director

STUDY_DIRECTOR

Ipsen

Study Locations (Sites)

UCSF

San Francisco, California, 94158

United States

University of California San Francisco (UCSF)

San Francisco, California, 94158

United States

Johns Hopkins Hospital

Baltimore, Maryland, 21287

United States

Boston Children's Hospital

Boston, Massachusetts, 02115

United States

Children's Mercy Hospital and Clinics

Kansas City, Missouri, 64018

United States

The Childrens Hospital at Montefiore Albert Einstein School of Medicine

Bronx, New York, 10467

United States

Hassenfeld Children's Hospital at NYU Langone

New York, New York, 10016

United States

Texas Liver Institute

San Antonio, Texas, 78215

United States

Collaborators and Investigators

Sponsor: Albireo, an Ipsen Company

Ipsen Medical Director, STUDY_DIRECTOR, Ipsen

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2021-09-03

Study Completion Date2027-04-16

Study Record Updates

Study Start Date2021-09-03

Study Completion Date2027-04-16

Terms related to this study

Additional Relevant MeSH Terms

Alagille Syndrome