NCT03708900

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

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
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: RECORDATI GROUP · Industry
• Sex: All
• Age: 2 Years – 17 Years
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

• Cushing Syndrome

Interventions

Type	Name	Description
DRUG	LCI699	osilodrostat (LCI699) is in the form of tablets 1 milligram (mg), 5 mg, and 10mg or in form of capsules 0.1 mg, 0.2 mg, 0.5 mg, 1 mg or 5 mg, both the formulations for oral administration

Timeline

• Start date: 2021-04-28
• Primary completion: 2027-07-21
• Completion: 2027-07-21
• First posted: 2018-10-17
• Last updated: 2026-03-27

Locations

15 sites across 7 countries: United States, Belgium, Bulgaria, France, Italy, Slovenia, United Kingdom

Regulatory

• FDA-regulated drug study