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

Conditions

Alagille Syndrome

Brief summary

Change from baseline in scratching through Week 72 as measured by the Albireo ObsRO caregiver instrument.

Detailed description

• 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

Related papers

No related papers found yet.

Interventions

DRUGKAYFANDA 400 microgram hard capsules

DRUGKAYFANDA 600 microgram hard capsules

DRUGKAYFANDA 200 microgram hard capsules

DRUGKAYFANDA 1 200 microgram hard capsules

Eligibility

Sex/Gender

All

Age

0 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
Change from baseline in scratching through Week 72 as measured by the Albireo 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 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) item	—

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 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) item

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Countries

Belgium, France, Germany, Italy, Netherlands, Poland

Outcome results

None listed