Pilot Study Of Long-Acting Octreotide (Octreotide LAR® Depot) In The Treatment Of Patients With Severe Polycystic Liver Disease
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- Mayo Clinic
- 入组人数
- 42
- 试验地点
- 1
- 主要终点
- Percent Change in Liver Volume
研究概览
简要总结
This study will evaluate the effect of Octreotide LAR® on the liver volumes of patients with severe polycystic liver disease who are not candidates or decline surgical treatments such as liver cyst fenestration, liver resection or liver transplantation. A total of 42 patients will be recruited -14 who will receive placebo and 28 the study drug. Preliminary evidence indicates that this drug is safe and non-toxic in other disease states. Treatment with this drug holds promise not only for individuals with liver involvement, but also for many more patients with polycystic kidney disease.
详细描述
The primary aim of this study is to compare the effect of Octreotide LAR® Depot on the liver volume of patients with severe polycystic liver disease who are not candidates or decline surgical treatments such as liver cyst fenestration, liver resection or liver transplantation compared with placebo. The secondary aims of the study are: (1)Assess the effect of Octreotide LAR® Depot on the total kidney volume and iothalamate clearance in patients with polycystic kidney disease associated with severe polycystic liver disease who are not candidates or decline surgical treatments such as liver cyst fenestration, liver resection or liver transplantation. (2)Evaluate quality of life changes associated with the administration of Octreotide LAR® Depot in these patients. (3)Assess toxicity of Octreotide LAR® Depot in patients with polycystic liver disease (PLD).
Note: Subjects who completed this 1 year randomized trial were offered enrollment into an open-label (all subjects received Octreotide) extension trial for an additional two years of treatment.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age - 18 years and older
- •Diagnosis of Polycystic Liver Disease (PLD) associated with ADPKD or isolated Autosomal Dominant Polycystic liver Disease (ADPLD)
- •Severe PLD defined as a liver volume greater than 4000 mL or symptomatic disease due to mass effects from hepatic cysts
- •Not a candidate for or declining surgical intervention
排除标准
- •Women of childbearing potential or their sexual partners who are unwilling to employ adequate contraception
- •Creatinine greater than 3mg/dL or hemodialysis dependent
- •Cancer or major systemic disease that could prevent completion of the planned follow-up or interfere with data collection or interpretation
- •Uncontrolled diabetes mellitus as defined by blood glucose levels of greater than or equal to 250 mg/dL on 2 or more consecutive daily readings despite antidiabetic therapy
- •Neurologic/psychologic conditions preventing appropriate informed consent
- •Symptomatic gallstones or biliary sludge
- •Variceal bleeding or hepatic encephalopathy within prior 30 days
- •Uncontrolled hypertension (Systolic blood pressure greater than 160 mmHg; Diastolic blood pressure greater than 100 mmHg)
- •Current, or prior use of somatostatin analogue (octreotide, lanreotide) in past 6 months
- •History of significant adverse reaction to a somatostatin analogue
研究组 & 干预措施
Octreotide
Participants received Octreotide LAR® Depot injections (up to 40 mg) intramuscularly every 28 days (+/- 5 days) for one year
干预措施: Octreotide (Drug)
Placebo
Participants received an injection of placebo (sham) medication intramuscularly every 28 days (+/- 5 day) for one year
干预措施: Placebo (Drug)
结局指标
主要结局
Percent Change in Liver Volume
时间窗: Baseline, 12 months
Percent change from baseline in liver volume, measured in milliliters by Magnetic Resonance Imaging (MRI)or Computed Tomography (CT) scans
次要结局
- Percent Change in Renal Volume(Baseline, 12 months)
- Percent Change in Glomerular Filtration Rate (GFR)(Baseline, 12 months)
- Change in Subject Reported Outcomes Using Mean Health Related Quality of Life (HRQoL) Scores(Baseline, 12 months)
研究者
Marie Hogan
MD, PhD, Assistant Professor of Medicine, College of Medicine
Mayo Clinic