Extension to a Multicenter, Open-label Study to Assess the Safety and Efficacy of 600 μg SOM230, Administered Subcutaneously, Bid in Patients With Cushing's Disease
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 19
- 试验地点
- 4
- 主要终点
- Percentage of Responders With Mean Urinary Free Cortisol (UFC) Within Normal Limits
研究概览
简要总结
Cushing's disease is a rare serious condition that is caused by an adrenocorticotropic hormone (ACTH) secreting pituitary adenoma. This study assessed the long-term safety and efficacy of pasireotide in participants with Cushing's disease.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants who have completed the 15 days of Pasireotide treatment in the CSOM230B2208 study and have achieved normalization of 24-hour urinary free cortisol. Participants who did not achieve normalization of 24 -hour urinary free cortisol may be enrolled if in the opinion of the investigator the participant is getting significant clinical benefits from treatment with Pasireotide .
- •The participant did not experience any unacceptable adverse events of tolerability issues during the original 15 day treatment.
- •Female participants of childbearing potential who have not undergone clinically documented total hysterectomy and/or ovariectomy or tubal ligation must agree to use barrier contraception throughout the course of the extension study, and for one month after the study has ended.
排除标准
- •Participant who have developed poorly controlled diabetes mellitus as indicated by ketoacidosis or hemoglobin (Hgb) A1C (HgbA1C) > 10 since starting [study CSOM230B2208].
- •Participant with persistent alanine aminotransferase (ALT)/ aspartate transaminase (AST) or alkaline phosphatase levels more than 2.5X upper limit of normal (ULN), serum creatinine > 2.0 X ULN, serum bilirubin > 2 X ULN.
- •Participant with abnormal coagulation (Prothrombin time (PT) and partial thromboplastin time (PTT) elevated by 30% above normal limits), white blood cells (WBC) <3.0x1'000'000'000/L; Hgb <12.0g/dL for females, Hgb <13.0g/dL for males; PLT <100x1'000'000'000/L.
- •Other protocol-defined inclusion / exclusion criteria may apply.
研究组 & 干预措施
Pasireotide 600 μg BID SC or Ramp up Dose 900 μg BID SC
Participants received pasireotide 600 micrograms (μg) twice daily (BID) subcutaneously (SC) to achieve or maintain urinary free cortisol (UFC) normalization. If UFC levels were increased at any time, participants received 900 μg BID SC, until no safety or tolerability concerns were observed as per investigators assessment. If the participant was unable to tolerate the 900 μg BID, dosing of 600 μg three times a day was given.
干预措施: Pasireotide (Drug)
结局指标
主要结局
Percentage of Responders With Mean Urinary Free Cortisol (UFC) Within Normal Limits
时间窗: Month 6
A participant was considered a responder if the mean UFC from the two 24-hour urine samples collected at Month 6 was within normal limits. The normal range for UFC is 55 to 276 nmol/day.
Change From Baseline in Mean Urinary Free Cortisol (UFC)
时间窗: Core Baseline, Days 14/15 (Core study), Months 6, 12, 24 and 102
24-hour urine samples were collected to obtain mean UFC measurements. A negative mean change from baseline indicates improvement.
次要结局
- Plasma Trough Concentrations (Ctrough) of Pasireotide in UFC Responders(Day 15 (Core study) and Month 6)
- Change From Baseline in Gene-expression and Protein in Blood and Urine for Biomarker Development(Baseline to end of the study)
- Change From Baseline in Serum Cortisol Levels(Core Baseline, Day 15 (Core study), Months 6, 12, 24, and Month 105 (end of the study))
- Number of Participants Who Had At Least One Adverse Event (AE)(Up to approximately 106 months)
- Change From Baseline in Plasma Adrenocorticotropic Hormone (ACTH) Levels(Core Baseline, Day 15 (Core study), Months 6, 12, 24 and Month 105 (end of the study))