2023-509028-17-00招募中3 期
An Open Label Study to Evaluate the Long-term Safety and Efficacy of Odevixibat (A4250) in Patients with Alagille Syndrome (ASSERT-EXT)
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- Ipsen Pharma
- 入组人数
- 35
- 试验地点
- 12
- 主要终点
- Change from baseline in scratching through Week 72 as measured by the Albireo ObsRO caregiver instrument.
研究概览
简要总结
To demonstrate a sustained effect of odevixibat on pruritus in patients with ALGS who have completed study A4250-012 (ASSERT)
入排标准
- 年龄范围
- 0 years 至 64 years（18-64 Years, 0-17 Years）
- 接受健康志愿者
- 是
入选标准
- •Completion of the 24-week Treatment Period of Study A4250-012
- •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
- •Caregivers (and age-appropriate patients) must be willing and able to use an electronic diary (eDiary) device as required by the study
- •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.
排除标准
- •Decompensated liver disease, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy
- •Patients who were not compliant with study drug treatment or procedures in Study A4250-012
- •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
- •Known hypersensitivity to any components of odevixibat
结局指标
主要结局
Change from baseline in scratching through Week 72 as measured by the Albireo ObsRO caregiver instrument.
Change from baseline in scratching through Week 72 as measured by the Albireo ObsRO caregiver instrument.
次要结局
- • 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
研究者
Clinical Operations Department
Scientific
Ipsen Pharma
研究点 (12)
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