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

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

ClinicalTrials.gov Identifier: NCT03708900

Novartis Reference Number: CLCI699C2203

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All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation. 

Study Description

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

Condition 
Cushing's Disease
Phase 
Phase 2
Overall status 
Recruiting
Enrollment count 
12 participants
Start date 
Oct 30, 2020
Completion date 
Nov 29, 2023
Gender 
All
Age(s)
6 Years - 18 Years (Child, Adult)

Interventions

Drug
LCI699
osilodrostat (LCI699) is in the form of tablets for oral administration and comes in the following tablet strengths: 1 milligram (mg), 5 mg, and 10mg.

Eligibility Criteria

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.

Study Locations

Belgium
Novartis Investigative Site
Recruiting
Jette, 1090
Brussel
Belgium
Bulgaria
Novartis Investigative Site
Recruiting
Varna, 9010
-
Bulgaria
Italy
Novartis Investigative Site
Recruiting
Pisa, 56124
PI
Italy
Slovenia
Novartis Investigative Site
Recruiting
Ljubljana, 1525
-
Slovenia
United Kingdom
Novartis Investigative Site
Recruiting
Liverpool, L12 2AP
-
United Kingdom
Novartis Investigative Site
Recruiting
London, E11BB
-
United Kingdom

Contacts

Name: 
Novartis Pharmaceuticals
Phone: 
1-888-669-6682
Name: 
Novartis Pharmaceuticals
Phone: 
+41613241111
Email: 

Have a question?

Call 1-999-669-6682 or email [email protected]