A long term Investigation of the Safety and Efficacy of Odevixibat in Patients with Alagille syndrome

• Register: EUCTR
• Last refreshed on: 30 September 2024
• Main ID: EUCTR2021-000996-36-FR
• Date of registration: 25/06/2021
• Prospective Registration: Yes
• Primary sponsor: Albireo AB
• Public title: A long term Investigation of the Safety and Efficacy of Odevixibat in Patients with Alagille syndrome
• Scientific title: An Open Label Study to Evaluate the Long-term Safety and Efficacy of Odevixibat (A4250) in Patients with Alagille Syndrome (ASSERT-EXT)
• Date of first enrolment: 14/10/2021
• Target sample size: 60
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2021-000996-36
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Belgium, Canada, France, Germany, Israel, Italy, Malaysia, Netherlands, New Zealand, Poland, Turkey, United Kingdom, United States

Contacts

• Name: Clinical Operations Department
• Affiliation: Albireo AB

• Name: Clinical Operations Department
• Affiliation: Albireo AB

Key inclusion & exclusion 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 hormonal contraception, 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: 45 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 15 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
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;

Age minimum:
Age maximum:
Gender: Female: yes Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Alagille Syndrome
MedDRA version: 20.0 Level: PT Classification code 10053870 Term: Alagille syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders

Intervention(s)

Product Name: Odevixibat Product Code: A4250 Pharmaceutical Form: Capsule, hard INN or Proposed INN: Odevixibat CAS Number: 501692-44-0 Current Sponsor code: A4250 Other descriptive name: A4250 Concentration unit: µg microgram(s) Concentration type: equal Concentration number: 200- Product Name: Odevixibat Product Code: A4250 Pharmaceutical Form: Capsule, hard INN or Proposed INN: Odevixibat CAS Number: 501692-44-0 Current Sponsor code: A4250 Other descriptive name: A4250 Concentration unit: µg microgram(s) Concentration type: equal Concentration number: 400- Product Name: Odevixibat Product Code: A4250 Pharmaceutical Form: Capsule, hard INN or Proposed INN: Odevixibat CAS Number: 501692-44-0 Current Sponsor code: A4250 Other descriptive name: A4250 Concentration unit: µg microgram(s) Concentration type: equal Concentration number: 600- Product Name: Odevixibat Product Code: A4250 Pharmaceutical Form: Capsule, hard INN or Proposed INN: Odevixibat CAS Number: 501692-44-0 Current Sponsor code: A4250 Other descriptive name: A4250 Concentration unit: µg microgram(s) Concentration type: equal Concentration number: 1200-

Primary Outcome(s)

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;

Timepoint(s) of evaluation of this end point: Change from baseline in scratching through Week 72

Main Objective: To demonstrate a sustained effect of odevixibat on pruritus in patients with ALGS who have completed study A4250-012 (ASSERT)

Primary end point(s): Change from baseline in scratching through Week 72 as measured by the Albireo ObsRO caregiver instrument.

Secondary Outcome(s)

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;

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) 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

Secondary ID(s)

145988

A4250-015

Source(s) of Monetary Support

Albireo AB

Ethics review

Status: Approved
Approval date: 09/09/2021
Contact:

Results

• Date Posted: 01/01/1900