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
RECORDATI GROUP
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.
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…
Eligibility
- Age range
- 2–17 years
- Sex
- All
- Healthy volunteers
- No
Inclusion criteria 1. 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. 2. Patients must weigh \> 10 kg. 3. 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 scor…
Interventions
- DrugLCI699
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
Locations (15)
- University of California San Francisco UCSFSan Francisco, California
- ABMED Clinical Research CorpCape Coral, Florida
- Ann & Robert H. Lurie Children's HospitalChicago, Illinois
- National Institute of Child Health and Human DevelopmentBethesda, Maryland
- Texas Valley Clinical ResearchWeslaco, Texas
- UZ BrusselJette, Brussels Capital