A Phase 3 Double-blind, Randomized, Placebo-controlled Study of the Safety and Efficacy of Odevixibat (A4250) in Patients with Alagille Syndrome (ASSERT) - NA

ALBIREO AB0 个研究点目标入组 52 人开始时间: 2021年5月20日最近更新: 2023年9月11日

适应症

试验速览

阶段: 1 期
状态: 进行中（未招募）
发起方: ALBIREO AB
入组人数: 52

研究概览入排标准研究者记录与标识符相似试验

研究概览

简要总结

暂无简介。

研究设计

研究类型: Interventional clinical trial of medicinal product

入排标准

性别: All

入选标准

•1. A male or female patient (of any age) with genetically confirmed diagnosis of ALGS. A patient may be randomized based on genetic testing results in the medical record. If genetic testing results are not available, testing will be performed at Screening Visit 1, and the patient may not be randomized until the genetic diagnosis is confirmed.
•2. Patient must have a history of significant pruritus and a caregiver reported observed scratching or a patient-reported pruritus score at an average of =2 (on 0 to 4 scale), as measured by the Albireo ObsRO instrument (for patients <18 years of age) or the PRO instrument (for patients >18 years of age) in the 14 days prior to randomization. For each AM and PM weekly assessment a minimum of 4 out of 7 expected scores must be recorded. The mean of the weekly AM and the mean of the weekly PM scores will be averaged to determine the pruritus score as measured by ObsRO or PRO, if the patient is =18 years of age.
•3. Patient must have an elevated baseline serum bile acid level. Each of the serum bile acid levels obtained at Screening Visit 1 and Screening Visit 2 must be greater than the upper limit of normal (>ULN).
•4. Patient and/or legal guardian must sign 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 in order to remain in the study.
•5. Caregivers must be willing and able to use an eDiary device as required by the study and patients =8 years of age must be willing to use an eDiary if able to do so.
•6. Sexually active males and females must agree to use a reliable contraceptive method with =1% failure rate (such as hormonal contraception, intrauterine device, or complete abstinence) throughout the duration of the study and 90 days thereafter (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:
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 18
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

排除标准

•1. Patient with past medical history or ongoing presence of other types of liver disease including, but not limited to, the following:
•a) Biliary atresia
•b) Progressive Familial Intrahepatic Cholestasis (PFIC)
•c) Benign recurrent intrahepatic cholestasis
•d) Suspected or proven liver cancer or metastasis to the liver on imaging
•2. Patient with a past medical history or ongoing presence of any other disease or condition known to interfere with the absorption, distribution, metabolism (specifically bile acid metabolism), or excretion of drugs in the intestine, including but not limited to, inflammatory bowel disease
•3. Patient with past medical history or ongoing chronic (i.e. >3 months) diarrhea requiring intravenous fluid or nutritional intervention for treatment of the diarrhea and/or its sequelae
•4. Patient has a confirmed past diagnosis of infection with human immunodeficiency virus or other present and active, clinically significant chronic infection
•5. Recent infection requiring hospitalization or treatment with parenteral anti-infective within 4 weeks of randomization (Study Day 1) or completion of oral anti-infective treatment within 2 weeks prior to start of Screening Period
•6. Cancer within the last 5 years except for basal cell carcinoma
•7. Cancer >5 years prior to screening except for non-liver cancers with no evidence of recurrence
•8. Chronic kidney disease with an impaired renal function and a glomerular filtration rate <70 mL/min/1.73 m2
•9. Patient with surgical history of disruption of the enterohepatic circulation (biliary diversion surgery) within 6 months prior to start of Screening Period
•10. Patient has had a liver transplant or a liver transplant is planned within 6 months of randomization
•11. Decompensated liver disease, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy
•12. International normalized ratio (INR) >1.4 (the patient may be treated with Vitamin K intravenously, and if INR is =1.4 at resampling the patient may be randomized)
•13. Serum alanine aminotransferase (ALT) >10 × ULN at Screening
•14. Serum ALT >15 × ULN at any time point during the last 6 months unless an alternate etiology was confirmed for the elevation
•15. Total bilirubin >15 × ULN at Screening
•16. Patient suffers from uncontrolled, recalcitrant pruritic condition other than ALGS. Examples include, but not limited to, refractory atopic dermatitis or other primary pruritic skin diseases

研究者

发起方

ALBIREO AB

相似试验

进行中（未招募）

1 期

Evaluation of Efficacy and Safety of ELX/TEZ/IVA in Subjects Without an F508del MutatioMedDRA version: 20.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem Organ Class: 10010331 - Congenital, familial and genetic disordersCystic Fibrosis

EUCTR2021-005320-38-PTVertex Pharmaceuticals Incorporated307

招募中

Unknown

A Phase 3 Randomized, Multi-center Trial of oral Alzheimer's Disease treatment drug, AR1001, in Participants with Early Alzheimer’s Disease

KCT0009137AriBio200

进行中（未招募）

1 期

EUCTR2021-005320-38-HUVertex Pharmaceuticals Incorporated270

已完成

3 期

A Phase 3 Double-blind, Randomized, Placebo-controlled Study Evaluating the Efficacy and Safety of ELX/TEZ/IVA in Cystic Fibrosis Subjects 6 Years of Age and Older With a Non-F508del ELX/TEZ/IVA-responsive CFTR MutatioCystic Fibrosis

NL-OMON51518Vertex Pharmaceuticals15

进行中（未招募）

1 期

EUCTR2021-005320-38-SEVertex Pharmaceuticals Incorporated270