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 Syndrome

Status: Recruiting
Location: See all (12) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
SUMMARY

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

Eligibility
Participation Requirements
Sex: All
Minimum Age: 2
Maximum Age: 17
Healthy Volunteers: f
View:

• 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.

• The diagnosis of Cushing's syndrome must be confirmed by each of the following:

⁃ 3a) The clinical criterion of decreasing growth percentiles with increasing weight (as evidenced by the presence of a contrast in height and BMI SD scores, for example a SDS \< 0 and BMI SDS \> 0, or a strong clinical suspicion of Cushing's syndrome, such as photographic evidence of a change in facial appearance); 3b) Abnormal low-dose (0.5 mg Q6h x 48 hours, or overnight 15mcg/kg \[max 1 mg\]) dexamethasone suppression test, defined as plasma cortisol levels \> 1.8 mcg/dl, at time point 48 hours (0.5 mg Q6h x 48 hours) or 9 to 12 hours (overnight 15mcg/kg \[max 1 mg\]) after the first dose of dexamethasone; (OR) Midnight serum cortisol levels \> 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. 3c)Two 24-hour urinary free cortisol values \> 1.3 x ULN;

⁃ 4\. Able to swallow study drug tablets (not crushed or split) or the content of the capsules mixed with water.

⁃ 5\. Parents or legal guardians able to provide consent/assent.

Locations
United States
California
University of California San Francisco UCSF
RECRUITING
San Francisco
Maryland
National Institute of Child Health and Human Development
RECRUITING
Bethesda
Other Locations
Belgium
UZ Brussel
RECRUITING
Jette
Bulgaria
Multiprofile Hospital for Active Treatment Sveta Marina EAD
WITHDRAWN
Varna
France
CHU Bicetre APHP Paris Saclay
RECRUITING
Paris
Hospital Necker Enfants Malades
RECRUITING
Paris
Robert Debre Hospital
RECRUITING
Paris
Italy
Aziendal Ospedaliero Universitaria Pisana Presidio Ospedale di Cisanello
RECRUITING
Pisa
Ospedale Bambino Gesu
RECRUITING
Roma
Slovenia
University Clinical Center Ljubljana
WITHDRAWN
Ljubljana
United Kingdom
Alder Hey Childrens NHS Foundation Trust
WITHDRAWN
Liverpool
The Royal London Childrens Hospital
RECRUITING
London
Contact Information
Primary
Recordati
casi.m@recordati.it
+39 0248787456
Backup
Recordati
+4161 205 61 00
Time Frame
Start Date: 2021-04-28
Estimated Completion Date: 2025-11-21
Participants
Target number of participants: 12
Treatments
Experimental: LCI699 (osilodrostat)
Subjects with cushing's syndrome taking LCI699 (osilodrostat)
Related Therapeutic Areas
Sponsors
Leads: RECORDATI GROUP

This content was sourced from clinicaltrials.gov