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

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

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

NL-OMON

Registry ID

NL-OMON54015

Enrollment

Registered

2021-09-02

Start date

2021-09-01

Completion date

Unknown

Last updated

2024-09-16

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Alagille Syndrome

Interventions

Odevixibat (A4250), 120 µg/kg/day once daily, orally administered

Eligibility

Age

No minimum to 17 Years

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

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
Change from baseline in scratching through Week 72 as measured by the Albireo observer-reported outcomes (ObsRO) caregiver instrument	—

Secondary

Measure	Time frame
• 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 assessments • 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	—

Measure

Time frame

• 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 assessments • 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

—

Countries

Netherlands

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)