NCT03708900 · RECORDATI GROUP
Pharmacokinetic (PK), Pharmacodynamic (PD) and Tolerability of Osilodrostat in Pediatric Patients With Cushing's Syndrome
What this study is about
conducted at multiple hospitals, where both patients and doctors know the treatment given, non-comparative study to evaluate the how the drug moves through the body, how the drug affects the body, and tolerability of osilodrostat in children and adolescent patients with Cushing's syndrome.
View original scientific description
Multicenter, open-label, non-comparative study to evaluate the pharmacokinetics, pharmacodynamics, and tolerability of osilodrostat in children and adolescent patients with Cushing's syndrome.
Interventions
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
Primary outcome measures
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
Time frame: 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
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- 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 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 (overnigh
Where
- San Francisco, California
- Cape Coral, Florida
- Chicago, Illinois
- Bethesda, Maryland
- Weslaco, Texas
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Mar 27, 2026 · Source of record for eligibility and locations