2024-512086-14-00已完成3 期
A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia Who Have Undergone a Kasai Hepatoportoenterostomy (BOLD)
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Ipsen Pharma
- 入组人数
- 80
- 试验地点
- 19
- 主要终点
- The primary efficacy endpoint is the time from randomization to first occurrence of liver transplant, or death, during the 104 week treatment period.
研究概览
简要总结
The primary objective is to evaluate the efficacy of repeated once-daily doses of odevixibat versus placebo in children with biliary atresia (BA) post Kasai hepatoportoenterostomy(HPE) based native liver survival (NLS) of up to 104 weeks.
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 接受健康志愿者
- 否
入选标准
- •A male or female patient with a clinical diagnosis of BA
- •Age at Kasai HPE ≤90 days
- •Eligible to start treatment within 3 weeks post-Kasai HPE
排除标准
- •Patients with intractable ascites
- •Ileal resection surgery
- •ALT ≥10× upper limit of normal (ULN) at screening
- •Patient on total parenteral nutrition, or not able to take study drug orally, at randomization
- •Acute ascending cholangitis (patients may be randomized after resolution of acute ascending cholangitis)
- •Choledochal cystic disease
- •INR >1.6 (the patient may be treated with Vitamin K intravenously; sample may be redrawn and if INR is ≤1.6 at resampling the patient may be randomized)
- •Any other conditions or abnormalities, including congenital abnormalities, major cardiac surgery, hepatic, biliary, or GI disease which, in the opinion of the Investigator or Medical Monitor, may compromise the safety of the patient, the integrity of study results, or patient compliance with study requirements
- •Weight < 3.5kg at randomization
研究组 & 干预措施
Placebo, odevixibat matching hard white opaque capsules filled with pellets with no active treatment for oral administration
Placebo
干预措施: Placebo, odevixibat matching hard white opaque capsules filled with pellets with no active treatment for oral administration (Drug)
A4250, A4250
Test
干预措施: A4250 (Drug)
结局指标
主要结局
The primary efficacy endpoint is the time from randomization to first occurrence of liver transplant, or death, during the 104 week treatment period.
The primary efficacy endpoint is the time from randomization to first occurrence of liver transplant, or death, during the 104 week treatment period.
次要结局
- 1. Proportion of patients who are alive and have not undergone a liver transplant after 104 weeks.
- 2. Time to onset of first sentinel event during the 104-week treatment period. Sentinel events are defined in the protocol
- 3. Total bilirubin level after 13, 26, 52, and 104 weeks.
- 4. Serum bile acid level after 13, 26, 52, and 104 weeks.
- 5. Time to pediatric end-stage liver disease (PELD) score ≥15.
- 6. Safety parameters including AEs, SAEs, findings on physical examination, laboratory assessments (including fat-soluble vitamins and lipids) and abdominal ultrasound.
研究者
Clinical Operations Department
Scientific
Ipsen Pharma
研究点 (19)
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