An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2) - PEDFIC 2 (2191/0009)

Albireo AB0 个研究点目标入组 8 人开始时间: 待定最近更新: 2024年4月15日

试验速览

阶段: 3 期
状态: 招募中
发起方: Albireo AB
入组人数: 8

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

入排标准

年龄范围: 0 至 64（—）

入选标准

•1. Completion of the 24-week Treatment Period of Study A4250-005 or withdrawn
•from Study A4250-005 due to patient/caregiver judgment of intolerable symptoms
•after completing at least 12 weeks of treatment. Patients who withdraw from
•A4250-005 due to a study drug related AE will not be eligible.
•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. Patients expected to have a consistent caregiver for the duration of the
•4. Caregivers (and age appropriate patients) must be willing and able to use an
•eDiary device as required by the study
•1. A male or female patient of any age, with a clinical diagnosis of PFIC,
•including episodic forms (i.e. benign recurrent intrahepatic cholestatis
•[BRIC]) and with a body weight greater or equal to 5 Kg at Visit S-1
•2. Patient must have clinical genetic confirmation of PFIC.
•3. Patients with PFIC, excluding BRIC, must have elevated serum bile acid
•concentration.
•4, Patients with PFIC, excluding BRIC, must have history of significant pruritus
•5. Patients with episodic forms of PFIC (i.e., BRIC) must have an emerging
•flare characterized by clinically significant pruritus and elevated serum bile
•acid levels/cholestasis as judged by the investigator.
•6. Patient and/or legal guardian must sign informed consent (and assent) as
•appropriate.
•7. Age appropriate patients are expected to have a consistent caregiver for the
•duration of the study.
•8. Caregivers and age-appropriate patients (greater or equal to 8 years of age
•if able) must be willing and able to use an eDiary device as required by the

排除标准

•Patients meeting any of the following criteria at Visit 1 will not be eligible
•for study participation:
•1. Decompensated liver disease: coagulopathy, history, or presence of
•clinically significant ascites, variceal hemorrhage, and/or encephalopathy
•2. Sexually active males and females who are not using a reliable contraceptive
•method with <=1% failure rate (such as barrier protection, hormonal
•contraception, intra-uterine 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). See Appendix 6 for further
•3. Patients not compliant with treatment in study A4250-005
•4. Any other conditions or abnormalities which, in the opinion of the
•investigator or Medical Monitor, may compromise the safety of the patient, or
•interfere with the patient participating in or completing the study
•1. Known pathologic variations of the ABCB11 gene that have been demonstrated
•to result in complete absence of the BSEP protein
•2. Patient with past medical history or ongoing presence of other types of
•liver disease.
•Note: Patients with clinically significant portal hypertension are allowed.
•3. Patient has had a liver transplant, or a liver transplant is planned within
•6 months of the Screening/Inclusion Visit
•4. Decompensated liver disease, coagulopathy, history, or presence of
•clinically significant ascites, variceal hemorrhage, and/or encephalopathy.
•5. INR >1.4 (the patient may be treated with Vitamin K intravenously, and if
•INR is less than or equal to 1.4 at resampling the patient may be included)
•6. Serum ALT>10 x upper limit of normal (ULN) at Screening
•7. Serum ALT>15 x ULN at any time point during the last 6 months unless an
•alternate etiology was confirmed for the elevation
•8. Total bilirubin >5 x ULN at Screening
•9. Patient suffers from uncontrolled, recalcitrant pruritic condition other
•10. Administration of bile acid or lipid binding resins and medications that
•slow GI motility.
•11. Any other conditions or abnormalities which, in the opinion of the
•investigator or Medical Monitor, may compromise the safety of the patient, or
•interfere with the patient participating in or completing the study

研究者

发起方

Albireo AB

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