Pharmacokinetic (PK), Pharmacodynamic (PD) and Tolerability of Osilodrostat in Pediatric Patients With Cushing's Disease

 Cushing's Disease / Posted 4 days ago

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

  • 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:
    • 1. 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);
    • 2. 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;
    • 3. Measurable morning ACTH levels, assessed before 10 am;
    • 4. 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
  • Study end date : 21/02/2023
  • Wales-Based Study Contact : Please speak to your clinician
Contact details
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Novartis Investigative Site Liverpool, United Kingdom, L12 2AP Novartis Investigative Site London, United Kingdom, E11BB
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 novartis.email@novartis.com

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