A Phase 2, Open-label Study to Assess Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 9
- 试验地点
- 1
- 主要终点
- Mean Daily Copper Balance: Day 36 Through Day 39
研究概览
简要总结
This exploratory study will investigate the effects of ALXN1840 on copper balance in participants with Wilson disease (WD).
详细描述
Participants who are treatment experienced (which includes standard-of-care therapies or ALXN1840) and treatment naïve are eligible for this study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of WD by Leipzig Criteria ≥
- •Able to reside in the clinical research unit for intensive metabolic monitoring of copper and molybdenum.
- •Participants willing to adhere to copper/molybdenum-controlled diet during the study.
- •Willing and able to follow protocol-specified contraception requirements.
- •Capable of giving signed informed consent.
排除标准
- •Decompensated cirrhosis or model for end stage liver disease score >
- •Modified Nazer score >
- •Clinically significant gastrointestinal bleed within past 3 months.
- •Alanine aminotransferase > 2 × upper limit of normal.
- •Hemoglobin less than lower limit of the reference range for age and sex.
- •Significant medical history (current or past).
- •Previous treatment with zinc within 30 days prior to the Screening Visit.
- •Participants in renal failure, defined as in end-stage renal disease on dialysis (chronic kidney disease stage 5) or creatinine clearance < 30 milliliters/minute.
研究组 & 干预措施
ALXN1840
Participants will be administered ALXN1840 at a dose of 15 milligrams (mg)/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39
干预措施: ALXN1840 (Drug)
结局指标
主要结局
Mean Daily Copper Balance: Day 36 Through Day 39
时间窗: Accumulation: Day 36 through Day 39 (ALXN1840 30 mg)
Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period.
Mean Daily Copper Balance: Day 1 Through Day 8
时间窗: Accumulation: Day 1 through Day 8 (ALXN1840 15 mg)
Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period.
Mean Daily Copper Balance: Day 31 Through Day 35
时间窗: Accumulation: Day 31 through Day 35 (ALXN1840 30 mg)
Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period.
Mean Daily Copper Balance: Day 25 Through Day 28
时间窗: Accumulation: Day 25 through Day 28 (ALXN1840 15 mg)
Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and was calculated as the average daily copper balance over the collection period.
次要结局
- Change From Baseline In Mean Daily Copper Balance(Accumulation: Baseline, Day 1 through Day 8 (ALXN1840 15 mg) and Day 31 through Day 35 (ALXN1840 30 mg); Steady State: Baseline, Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg))
- Copper Quantified In Food, Drink, Feces, And Urine, Including Plasma Total And Labile Bound Copper (LBC)(Accumulation: Day 1 through Day 8 for 15 mg and Day 31 through Day 35 for 30 mg; Steady state: Day 25 through Day 28 for ALXN1840 15 mg and Day 36 through Day 39 for ALXN1840 30 mg)
- Change From Baseline In Total Molybdenum Excretion In Urine And Feces(Accumulation: Baseline, Day 1 through Day 8 (ALXN1840 15 mg) and Day 31 through Day 35 (ALXN1840 30 mg); Steady State: Baseline, Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg))
- Mean Daily Molybdenum Balance At ALXN1840 Steady State(Steady state: Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg))
- Molybdenum Specified In ALXN1840 Doses Given And Quantified In Food, Drink, Feces, And Urine, Including Plasma At Steady State(Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg))
- Accumulation Of Molybdenum As Determined By Molybdenum Balance(Accumulation: Day 1 through Day 8 (ALXN1840 15 mg) and Day 31 through Day 35 ((ALXN1840 30 mg))
- Maximum Observed Plasma Concentration (Cmax) of Total Molybdenum and Plasma Ultrafiltrate (PUF) Molybdenum(Day 1 up to Day 39)
- Area Under The Concentration Time Curve (AUC0-inf) of Total Molybdenum and Plasma Ultrafiltrate (PUF) Molybdenum(Day 39)