Pharmacokinetic (PK), Pharmacodynamic (PD) and Tolerability of Osilodrostat in Pediatric Patients With Cushing's Syndrome
- Registration Number
- NCT03708900
- Lead Sponsor
- RECORDATI GROUP
- Brief 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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 12
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description LCI699 (osilodrostat) LCI699 Subjects with cushing's syndrome taking LCI699 (osilodrostat)
- Primary Outcome Measures
Name Time Method Core Study: Evaluate the pharmacokinetics (PK) of osilodrostat using Pharmacokinetic parameters of osilodrostat up to Week 12 in children and adolescents 2 to less than 18 years of age with Cushing's Syndrome up to Week 12 evaluate the pharmacokinetics (PK) of osilodrostat in children and adolescents of 2 to less than 18 years of age with Cushing's Syndrome
- Secondary Outcome Measures
Name Time Method Core Study: Percentage of patients with normal mean urinary free cortisol (mUFC) at week 3, 6, 9 and week 12 (or end of treatment) week 3, 6, 9 and week 12 (or end of treatment) The assessment in the core period will be done by taking the percentage of patients with normal mUFC at week 6 and week 12 (or end of treatment).
Core Study: Change from baseline in mean urinary free cortisol (mUFC) during the core study period week 3, 6, 9 and week 12 (or end of treatment) The assessment will be done by comparison of change from the baseline in mUFC during core study period on patients
Extension: Efficacy of osilodrostat as measured by mUFC levels up to Month 12 up to month 12 The assessment of efficacy of osilodrostat to be measured byproportion of patients with normal mUFC levels at each visit up to 12 months
assessment of the pharmacodynamics, safety and tolerability of osilodrostat. up to 12 months Change from baseline in height at each visit in extension period
assessment of the pharmacodynamics, safety and tolerability of osilodrostat up to 12 months up to 12 months Change from baseline in weight at each visit in extension period
assessment of the pharmacodynamics, safety and tolerability of osilodrostat.assessment of the pharmacodynamics, safety and tolerability of osilodrostat. up to 12 months Change from baseline in body mass index at each visit in extension period
Trial Locations
- Locations (12)
University of California San Francisco UCSF
🇺🇸San Francisco, California, United States
National Institute of Child Health and Human Development
🇺🇸Bethesda, Maryland, United States
UZ Brussel
🇧🇪Jette, Brussel, Belgium
Hospital Necker Enfants Malades
🇫🇷Paris, France
Multiprofile Hospital for Active Treatment Sveta Marina EAD
🇧🇬Varna, Bulgaria
Aziendal Ospedaliero Universitaria Pisana Presidio Ospedale di Cisanello
🇮🇹Pisa, PI, Italy
Robert Debre Hospital
🇫🇷Paris, France
CHU Bicetre APHP Paris Saclay
🇫🇷Paris, France
Ospedale Bambino Gesu
🇮🇹Roma, Italy
University Clinical Center Ljubljana
🇸🇮Ljubljana, Slovenia
Alder Hey Childrens NHS Foundation Trust
🇬🇧Liverpool, United Kingdom
The Royal London Childrens Hospital
🇬🇧London, United Kingdom