2024-516825-30-00招募中2 期
A phase II, multicenter, open-label, non-comparative study to evaluate the pharmacokinetics, pharmacodynamics, and tolerability of osilodrostat in children and adolescent patients with Cushing's disease
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- Recordati AG
- 入组人数
- 12
- 试验地点
- 6
- 主要终点
- Pharmacokinetic parameters of osilodrostat
研究概览
简要总结
To evaluate the pharmacokinetics (PK) of osilodrostat in children and adolescents 6 to <18 years of age with Cushing's Disease
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 接受健康志愿者
- 是
入选标准
- •Male and female children and adolescents from 6 to < 18 years of age with Cushing's disease of endogenous origin who have failed surgery or are awaiting surgery or for whom surgery is not an immediate option
- •Body weight greater 30 kg
- •Confirmed diagnosis of Cushing's disease
- •Able to swallow study drug tablets (not crushed or split)
- •Parents or legal guardians able to provide consent/assent
排除标准
- •Macroadenoma complicated by compressive symptoms
- •Hypertensive patients with uncontrolled blood pressure
- •Patients who have undergone any major surgery within 1 month
- •Patients who have undergone trans-sphenoidal pituitary surgery within 6 weeks prior to screening, unless they have clear evidence of persistent hypercortisolemia or persistent biochemical changes consistent with Cushing's disease
- •Use of or anticipated use of systemic glucocorticoid medications 1 month prior to screening.
- •Uncontrolled hypothyroidism as evidenced by Free T4 < 0.8 ng/dl.
- •Uncontrolled hyperthyroidism
- •Diabetic patients with poorly controlled diabetes as evidenced by HbA1c > 8.5 % or not optimally treated for diabetes mellitus as judged by the investigator
- •Positive pregnancy test in females of childbearing potential
- •Female patients of childbearing potential who do not agree to use highly effective birth control methods
- •Pregnant or nursing (lactating) women.
- •Insufficient washout period from any other medication used to lower cortisol levels
- •Any medical condition that would, in the investigator's judgment, prevent the patient's participation in the clinical study due to safety concerns or compliance with clinical study procedures.
- •Use of concomitant prohibited medications (see section 6.2.2).
- •Use of other investigational drugs at the time of enrollment
- •History of hypersensitivity to drugs of the same or similar chemical classes as osilodrostat
- •History of malignancy of any organ system
- •Moderate to severe renal impairment
- •Serum ALT and/or AST > 3 x ULN, or total bilirubin > 1.5 x ULN
- •History of thrombosis
- •Risk factors for QTc prolongation or Torsade de Pointes, including: 9a. patients with a baseline QTcF > 450 ms 9b. personal or family history of long QT syndrome 9c. concomitant medications known to prolong the QT interval (see Section 6.2.2.1) 9d. patients with hypokalemia, hypocalcaemia, or hypomagnesaemia, if not corrected before pre-dose Day
- •In case of uncorrected hypokalemia (<3.5 mEq/L), the screening period may be used to correct hypokalemia prior to starting study drug. Use of potassium supplements and/or mineralocorticoid antagonists is permitted during the study. 9e. Patients with a history of significant cardiovascular disease (based on the opinion of the investigator) such as: structural cardiovascular abnormalities, arrhythmia, etc.
结局指标
主要结局
Pharmacokinetic parameters of osilodrostat
Pharmacokinetic parameters of osilodrostat
次要结局
- Proportion of patients with normal mUFC
- mUFC absolute values and change from baseline
- Safety and tolerability
研究者
Gaël Thomas
Scientific
Recordati AG
研究点 (6)
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