EUCTR2017-002325-38-PL进行中（未招募）1 期
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)
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
- 120
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. Completion of the 24-week Treatment Period of Study A4250-005 or withdrawn from Study A4250-005 due to patient/caregiver judgment of intolerable symptoms after completing at least 12 weeks of treatment.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 study
- •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 cholestasis [BRIC]), and with a body weight =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 (=8 years of age, if able) must be willing and able to use an eDiary device as required by the study.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 120
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 4
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
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).
- •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 =1.4 at resampling the patient may be randomized)
- •6. Serum ALT >10 × upper limit of normal (ULN) at Screening
- •7. Serum ALT >15 × ULN at any time point during the last 6 months unless an alternate etiology was confirmed for the elevation
- •8. Total bilirubin >5 × ULN at Screening
- •9. Patient suffers from uncontrolled, recalcitrant pruritic condition other than PFIC.
- •10. Administration of bile acid or lipid binding resins and medicationsthat 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
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