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临床试验/NCT02468193
NCT02468193已完成2 期

A Phase II, Open-label, Dose Titration, Multi-center Study to Assess the Safety/Tolerability and Efficacy of Osilodrostat in Patients With All Types of Endogenous Cushing's Syndrome Except Cushing's Disease

Novartis Pharmaceuticals1 个研究点 分布在 1 个国家目标入组 9 人开始时间: 2015年9月24日最近更新:
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适应症
干预措施
相关药物

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
Percent Change in the Mean Urine Free Cortisol (mUFC) at the Individual Level at Week 12
研究概览研究设计入排标准研究组 & 干预措施结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

The study aim was to investigate the efficacy and safety of Osilodrostat in patients with Cushing's syndrome due to causes other than Cushing's disease in Japan.

详细描述

This was a Phase II, single arm, open-label, dose titration, multi-center study which consisted of two distinct Study Periods plus an optional extension period in non-CD patients with CS. The 3 Study Periods (two distinct Study Periods plus an optional extension period) were as follows:

Study Period I [Week 0 (Day 1) to Week-12]: Study Period I was the dose titration period to achieve a stable therapeutic dose and to assess the efficacy and safety of osilodrostat.

The dosing regimen of osilodrostat in this study was titrated according to the following escalation sequence: osilodrostat 2 mg bid, 5 mg bid, 10 mg bid, 20 mg bid, and 30 mg bid. Dose adjustments were based on the serum cortisol values measured by the local lab at each site. Osilodrostat titration was done weekly for the initial 4-weeks, up to a maximum dose of 10 mg bid.

The mean of three 24-hour UFC (mUFC) values were measured to evaluate the efficacy in this period.

Study Period II (After Week-12 to Week-48): Study Period II was the period to assess the sustainability of efficacy and long term safety.

研究设计

研究类型
Interventional
分配方式
Na
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 85 Years（Adult, Older Adult）
性别
All
接受健康志愿者
否

入选标准

  • •Patients with confirmed Cushing's syndrome [i.e. ectopic corticotropin syndrome, adrenal adenoma, adrenal carcinoma, ACTH-Independent Macronodular Adrenal Hyperplasia (AIMAH), or Primary Pigmented Nodular Adrenal Dysplasia (PPNAD)]
  • •For patients on medical treatment for hypercortisolism due to Cushing's syndrome, the washout periods had to be completed prior to baseline efficacy assessments

排除标准

  • •Patients with Cushing's disease
  • •History of hypersensitivity to osilodrostat or to drugs of similar chemical classes
  • •History of malignancy of any organ system, treated or untreated, within the past 5 years
  • •Patients receiving treatment for within 4 weeks or ≤5 x half-life of the agent (whichever is longer) before first dose of osilodrostat
  • •Patients with risk factors for QTc prolongation or Torsade de Pointes

研究组 & 干预措施

Osilodrostat

Experimental

Patients in this arm took the study drug, osilodrostat.

干预措施: Osilodrostat (Drug)

结局指标

主要结局

Percent Change in the Mean Urine Free Cortisol (mUFC) at the Individual Level at Week 12

时间窗: Baseline, 12 weeks

Percent change from baseline in the mUFC at the individual patient level

次要结局

  • Percent Change From Baseline in the mUFC at Individual Patient Level at Week 24 (Day 169) and Week 48 (Day 337)(Baseline, Week 24 (day 169) and Week 48 (day 337))
  • Absolute Change From Baseline in the mUFC at Week 12 (Day 85), Week 24 (Day 169) and Week 48 (Day 337)(Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337))
  • Percentage Change From Baseline in the mUFC at Week 12 (Day 85), Week 24 (Day 169) and Week 48 (Day 337)(Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337))
  • Percentage of Participants With mUFC Response of Complete, Partial, and Overall Response(12, 24 and 48 weeks)
  • Absolute Change From Baseline in Morning Serum Cortisol at Individual Level(Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337))
  • Percentage Change From Baseline in Morning Serum Cortisol at Individual Level(Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337))
  • Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level(Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337))
  • Percentage Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level(Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337))
  • Absolute Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels(Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337))
  • Percentage Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels(Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337))
  • Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level(Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337))
  • Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level(Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337))
  • Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level(Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337))
  • Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level(Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337))
  • Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level(Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337))
  • Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level(Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337))
  • Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level(Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337))
  • Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level(Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337))
  • Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level(Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337))
  • Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level(Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337))
  • Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level(Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337))
  • Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level(Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337))
  • Total Scores in Patient-Reported Outcomes Health-related Quality of Life (QoL) as Assessed by Cushing QoL at Individual Level(Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337))
  • Total Scores in Patient-Reported Outcomes Health-related Quality of Life (QoL) as Assessed by Beck Depression Inventory II (BDI-ll) Depression Score at Individual Level(Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337))
  • Plasma Concentrations of Osilodrostat (LCI699) at Week 0(Week 0)
  • Plasma Concentrations of Osilodrostat (LCI699) at Week 1(Week 1, 2 hours post-dose)
  • Plasma Concentrations of Osilodrostat (LCI699) at Week 2(Week 2)
  • Plasma Concentrations of Osilodrostat (LCI699) at Week 3(Week 3, 2 hours post-dose)
  • Plasma Concentrations of Osilodrostat (LCI699) at Week 4(Week 4, 2 hours post-dose)
  • Plasma Concentrations of Osilodrostat (LCI699) at Week 6(Week 6, 2 hours post-dose)
  • Plasma Concentrations of Osilodrostat (LCI699) at Week 8(Week 8, 2 hours post-dose)
  • Plasma Concentrations of Osilodrostat (LCI699) at Week 10(Week 10, 2 hours post-dose)
  • Plasma Concentrations of Osilodrostat (LCI699) at Week 12(Week 12)
  • Plasma Concentrations of Osilodrostat (LCI699) at Week 16(Week 16, 2 hours post-dose)
  • Plasma Concentrations of Osilodrostat (LCI699) at Week 20(Week 20, 2 hours post-dose)
  • Plasma Concentrations of Osilodrostat (LCI699) at Week 24(Week 24, 2 hours post-dose)

研究者

申办方类型
Industry
责任方
Sponsor

研究点 (1)

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