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

  • STATUS
    Recruiting
  • End date
    Nov 29, 2023
  • participants needed
    12
  • sponsor
    RECORDATI GROUP
Updated on 10 May 2022
dexamethasone
ACTH
cushing's syndrome
free cortisol
urinary free cortisol
dexamethasone suppression test
lci699
osilodrostat

Summary

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

Description

The period 1 study duration will be 12 weeks. The study will include a screening period of up to 4 weeks prior to Day 0 (baseline) (to allow for an adequate washout period from any medications that may modify cortisol levels). All subjects being treated with osilodrostat at 12 weeks and obtaining benefit from therapy, per investigator judgment, will be offered participation in an optional 9-month extension period, during which assessment of the PD activity and safety/tolerability of osilodrostat will be done. Patients who do not enter the optional extension period will have a safety follow up visit 4 weeks later.

Details
Condition Cushing's Disease
Treatment LCI699
Clinical Study IdentifierNCT03708900
SponsorRECORDATI GROUP
Last Modified on10 May 2022

Eligibility

Yes No Not Sure

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
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