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

CLCI699C2203

Multicenter, open-label, non-comparative study to evaluate the pharmacokinetics, pharmacodynamics, and tolerability of osilodrostat in children and adolescent patients with Cushing's disease.

2021-04-28

2025-11-21

2025-11-21

Recruiting

Early phase I

12

Inclusion Criteria: * Cushing's disease of endogenous origin: Who have failed surgery (or) who are awaiting surgery (or) for whom surgery is not an immediate option. * The diagnosis of Cushing's disease must be confirmed by each of the following: * The clinical criterion of decreasing growth percentiles with increasing weight (as evidenced by the presence of a contrast in height and BMI standard deviation (SD) scores, defined as height standard deviation score (SDS) \< 0 and BMI SDS \> 0, and a strong clinical suspicion of Cushing's disease, such as photographic evidence of a change in facial appearance); * Abnormal low-dose (0.5 mg Q6h x 48 hours) dexamethasone suppression test, defined as plasma cortisol levels \> 1.8 mcg/dl, at time point 48 hours after the first dose of dexamethasone; * Measurable morning ACTH levels, assessed before 10 am; * Two 24-hour urinary free cortisol values \> 1.3 x ULN * If the dexamethasone suppression test does not meet the above mentioned criteria, the diagnosis of Cushing's disease may be confirmed by the following: Midnight serum cortisol levels \> upper limit of normal (ULN), assessed while the patient is sleeping and after pre-cannulation (OR) two samples of late night salivary cortisol greater than ULN for the assay * Able to swallow study drug tablets (not crushed or split) * Parents or legal guardians able to provide consent/assent Exclusion Criteria: * 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) * Hypercortisolism not due to Cushing's disease * 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. * Body weight \<30kg Other protocol-defined inclusion/exclusion may apply.

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 12, 'type': 'ESTIMATED'}}

2024-05-22