Multicenter, open-label, non-comparative study to evaluate the pharmacokinetics, pharmacodynamics, and tolerability of osilodrostat in children and adolescent patients with Cushing's syndrome.
Male and female children and adolescents from 2 to < 18 years of age with Cushing's syndrome of endogenous origin: Who have failed surgery (or) who are awaiting surgery (or) for whom surgery is not an immediate option. For patients who are awaiting surgery, the study treatment could be less than 12 weeks.
Patients must weigh > 10 kg.
Patients with macroadenoma complicated by compressive symptoms (requiring urgent surgical intervention) or at high risk for compressive symptoms due to mass effect of tumor (concern of corticotroph tumor progression).
Insufficient washout period from any other medication used to lower cortisol levels (5 half-lives of any drug).
Use of other investigational drugs at the time of enrollment, or within 30 days, or prior to completion of a wash-out duration that is at least 5 half- lives of the drug, at the time of enrollment, whichever is longer. Local regulations may require a longer wash-out period or specify other limitations for participation in an investigational trial, in which case they will be applicable as well.
History of hypersensitivity to drugs of the same or similar chemical classes as osilodrostat.
Single group
LCI699
Drugosilodrostat (LCI699) is in the form of tablets 1 milligram (mg), 5 mg, and 10mg or in form of capsules 0.1 mg, 0.2 mg, 0.5 mg, 1 mg or 5 mg, both the formulations for oral administration
Core Study: Evaluate the pharmacokinetics (PK) of osilodrostat using Pharmacokinetic parameters of osilodrostat up to Week 12 in children and adolescents 2 to less than 18 years of age with Cushing's Syndrome
evaluate the pharmacokinetics (PK) of osilodrostat in children and adolescents of 2 to less than 18 years of age with Cushing's Syndrome
Core Study: Percentage of patients with normal mean urinary free cortisol (mUFC) at week 3, 6, 9 and week 12 (or end of treatment)
The assessment in the core period will be done by taking the percentage of patients with normal mUFC at week 6 and week 12 (or end of treatment).
Core Study: Change from baseline in mean urinary free cortisol (mUFC) during the core study period
The assessment will be done by comparison of change from the baseline in mUFC during core study period on patients
Extension: Efficacy of osilodrostat as measured by mUFC levels up to Month 12
The assessment of efficacy of osilodrostat to be measured by change in baseline of mUFC levels up to 12 months on patients
Extension: Efficacy of osilodrostat as measured by mUFC levels up to Month 12
The assessment of efficacy of osilodrostat to be measured byproportion of patients with normal mUFC levels at each visit up to 12 months
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