A Randomized, Double-blind Study to Assess the Safety and Efficacy of Different Dose Levels of Pasireotide (SOM230) Subcutaneous (sc) Over a 6 Month Treatment Period in Patients With de Novo, Persistent or Recurrent Cushing's Disease
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 162
- 试验地点
- 11
- 主要终点
- Number of mUFC (Urinary Free Cortisol) Responders by Randomized Dose Group
研究概览
简要总结
This study will evaluate the safety and efficacy of two different doses of Pasireotide in patients with de novo or recurrent/persistent Cushing's Disease.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Pasireotide 600 ug
At randomization, participants received 600 ug subcutaneously (sc) twice daily (bid). Participants continued at this dose until month 6 if their month 3 mean urinary free cortisol (mUFC) was <= 2 x the upper limit of normal (ULN) and the mUFC was below or equal to their baseline mUFC. Participants not meeting the mUFC criteria at month 3 were unblinded and required to increase their dose to 900ug bid on an open label basis. Participants had the option to continue in the extension phase as long as they did not meet any discontinuation criteria or until pasireotide was available commercially in their country.
干预措施: Pasireotide (Drug)
Pasireotide 900 ug
At randomization, participants received 900 ug subcutaneously (sc) twice daily (bid). Participants continued at this dose until month 6 if their month 3 mean urinary free cortisol (mUFC) was <= 2 x the upper limit of normal (ULN) and the mUFC was below or equal to their baseline mUFC. Participants not meeting the mUFC criteria at month 3 were unblinded and required to increase their dose to 1200 ug bid on an open label basis. Participants had the option to continue in the extension phase as long as they did not meet any discontinuation criteria or until pasireotide was available commercially in their country.
干预措施: Pasireotide (Drug)
结局指标
主要结局
Number of mUFC (Urinary Free Cortisol) Responders by Randomized Dose Group
时间窗: 6 months
A responder in the primary efficacy analysis was a patient with a mUFC≤ULN at Month 6 and whose dose was not increased prior to Month 6.
次要结局
- Change From Baseline in mUFC(baseline, 3 months, 12 months)
- Time to First UFC Response(12 months)
- Percent Change From Baseline in Serum Cortisol(baseline, 0.5, 1, 1.5, 2, 2.5, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48, 51, 54, 57, 60, 63, 66, 69, 72, 75, 78 months)
- Percent Change From Baseline in Mean Adrenocorticotropic Hormone (ACTH)(baseline, 0.5, 1, 1.5, 2, 2.5, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48, 51, 54, 57, 60, 63, 66, 69, 72, 75, 78 months)
- Mean Change From Baseline in Clinical Signs and Symptoms of Cushing's Disease: Sitting Sytolic Blood Pressure (SBP) and Sitting Diastolic Blood Pressure (DBP)(baseline, month 3, month 6, month 12, month 24, month 36, month 48, month 60)
- Mean Change From Baseline in Clinical Signs and Symptoms of Cushing's Disease: Body Mass Index (BMI)(baseline, month 3, month 6, month 12, month 24, month 36, month 48 and month 60)
- Mean Change From Baseline in Clinical Signs and Symptoms of Cushing's Disease: Waist Circumference(baseline, month 3, month 6, month 12, month 24, month 36, month 48, month 60)
- Mean Change From Baseline in Clinical Signs and Symptoms of Cushing's Disease: Total Cholesterol and Triglycerides(baseline, month 3, month 6, month 12, month 24, month 36, month 48, month 60)
- Mean Change From Baseline in Clinical Signs and Symptoms of Cushing's Disease: Beck Depression Inventory (BDI-II) Score(baseline, month 3, month 6, month 12, month 18, month 24)
- Mean Change From Baseline in Clinical Signs and Symptoms of Cushing's Disease: Ferriman-Galway Hirsutism Score(baseline, month 3, month 6, month 12, month 24, month 36, month 48, month 60)
- Mean Change From Baseline in Clinical Signs and Symptoms of Cushing's Disease: Bone Mineral Density (BMD)(baseline, month 3, month 6, month 12, month 24, month 36, month 48, month 60)
- Mean Change From Baseline in Clinical Signs and Symptoms of Cushing's Disease: Body Composition(baseline, month 3, month 6, month 12, month 24, month 36, month 48, month 60)
- Change From Baseline in Tumor Volume(baseline, 6, 12, 18, 24, 30, 36, 42, 48, 54, 60, 66, 72, 78 months)
- Percentage Change From Baseline in Health Related Quality of Life (HRQL) Score(baseline, 3 months, 6 months, 12 months)