Alagille Syndrome MedDRA version: 20.0 Level: PT Classification code 10053870 Term: Alagille syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Sponsors
Albireo AB
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion 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 intra-uterine device or complete abstinence) from signed informed consent through 90 days after last dose of study drug; Are the trial subjects under 18? yes Number of subjects for this age range: 51 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 0 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 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;
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To demonstrate a sustained effect of odevixibat on pruritus in patients with ALGS who have completed study A4250-012 (ASSERT);Secondary Objective: To demonstrate a sustained effect of odevixibat on serum bile acids in patients with ALGS who have completed study A4250-012; To evaluate an effect of odevixibat on parameters related to quality of life; To evaluate the long-term safety and tolerability of repeated daily doses of odevixibat in patients with ALGS;;Primary end point(s): Change from baseline in scratching through Week 72 as measured by the Albireo ObsRO caregiver instrument.;Timepoint(s) of evaluation of this end point: Change from baseline in scratching through Week 72 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Change in serum bile acid levels from baseline to Week 72; • Change from baseline through Week 72 in patient reported and observer reported itching and scratching severity scores, respectively, for the morning and evening assessment; • Percentage of patients achieving a clinically meaningful decrease in pruritus (pruritus responders) at each visit as measured by the Albireo ObsRO/patient reported outcomes (PRO) instruments; • Change from baseline to Week 72 in sleep parameters as measured with the Albireo ObsRO/PRO instruments (e.g. tiredness and number of awakenings); • Change from baseline to Week 72 in Pediatric Quality of Life Inventory (PedsQL) scores • Assessment of Global Symptom Relief from baseline to Weeks 4, 12, 24, 48, and 72 as measured by patient, caregiver, and clinician Global impression of Symptoms (PGIS, CaGIS, CGIS) items • Assessment of Global Symptom Relief as measured by patient, caregiver, and clinician Global Impression of Change (PGIC, CaGIC, CGIC) items at Weeks 4, 12, 24, 48, and 72 • Change in serum bile acid levels from baseline through Week 72;Timepoint(s) of evaluation of this end point: • Change in serum bile acid levels from baseline through Week 72; • Change from baseline through Week 72; • Assessment of Global Symptom Relief from baseline to Weeks 4, 12, 24, 48, and 72; | — |
Countries
Belgium, France, Germany, Italy, Malaysia, Netherlands, Poland, Türkiye, United Kingdom, United States
Contacts
Public ContactClinical Operations Department
Albireo AB
Outcome results
None listed