Actively Recruiting
Pharmacokinetic (PK), Pharmacodynamic (PD) and Tolerability of Osilodrostat in Pediatric Patients With Cushing's Syndrome
Led by RECORDATI GROUP · Updated on 2026-03-27
12
Participants Needed
15
Research Sites
325 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Multicenter, open-label, non-comparative study to evaluate the pharmacokinetics, pharmacodynamics, and tolerability of osilodrostat in children and adolescent patients with Cushing's syndrome.
CONDITIONS
Official Title
Pharmacokinetic (PK), Pharmacodynamic (PD) and Tolerability of Osilodrostat in Pediatric Patients With Cushing's Syndrome
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male and female children and adolescents from 2 to less than 18 years of age with endogenous Cushing's syndrome who have failed surgery, are awaiting surgery, or for whom surgery is not an immediate option
- Patients must weigh more than 10 kg
- Confirmed diagnosis of Cushing's syndrome by clinical signs and specific cortisol tests including dexamethasone suppression test, midnight serum cortisol, or late-night salivary cortisol
- Able to swallow study drug tablets whole or capsules mixed with water
- Parents or legal guardians able to provide consent or assent
You will not qualify if you...
- Patients with macroadenoma causing or at risk for compressive symptoms needing urgent surgery
- Insufficient washout period from other cortisol-lowering medications
- Use of other investigational drugs within 30 days or insufficient washout
- History of hypersensitivity to osilodrostat or similar drugs
- History of malignancy within past 5 years, excluding localized basal cell carcinoma
- Moderate to severe kidney impairment
- Elevated liver enzymes or bilirubin above specified limits
- History of thrombosis
- Risk factors for QTc prolongation or Torsade de Pointes including certain heart conditions and electrolyte imbalances
- Uncontrolled high blood pressure or hypertension not optimally treated
- Major surgery within 1 month prior to screening
- Trans-sphenoidal pituitary surgery within 6 weeks prior to screening unless persistent hypercortisolism
- Use or anticipated use of systemic glucocorticoids within 1 month prior to screening
- Uncontrolled hypothyroidism or hyperthyroidism
- Poorly controlled diabetes or not optimally treated diabetes
- Positive pregnancy test or pregnancy or nursing in females of childbearing potential
- Female patients of childbearing potential not agreeing to use highly effective birth control
- Any medical condition or psychiatric disorder that prevents participation or risks safety
- Use of prohibited medications during the study
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 15 locations
1
University of California San Francisco UCSF
San Francisco, California, United States, 94143
Actively Recruiting
2
ABMED Clinical Research Corp
Cape Coral, Florida, United States, 33914
Actively Recruiting
3
Ann & Robert H. Lurie Children's Hospital
Chicago, Illinois, United States, 60611
Actively Recruiting
4
National Institute of Child Health and Human Development
Bethesda, Maryland, United States, 20892
Completed
5
Texas Valley Clinical Research
Weslaco, Texas, United States, 78596
Actively Recruiting
6
UZ Brussel
Jette, Brussels Capital, Belgium, 1090
Completed
7
Multiprofile Hospital for Active Treatment Sveta Marina EAD
Varna, Bulgaria, 9010
Withdrawn
8
Hospital Necker Enfants Malades
Paris, France, 75015
Completed
9
Robert Debre Hospital
Paris, France, 75019
Completed
10
CHU Bicetre APHP Paris Saclay
Paris, France, 94270
Completed
11
Aziendal Ospedaliero Universitaria Pisana Presidio Ospedale di Cisanello
Pisa, PI, Italy, 56124
Completed
12
Ospedale Bambino Gesu
Roma, Italy, 00165
Completed
13
University Clinical Center Ljubljana
Ljubljana, Slovenia, 1525
Withdrawn
14
Alder Hey Childrens NHS Foundation Trust
Liverpool, United Kingdom, L12 2AP
Withdrawn
15
The Royal London Childrens Hospital
London, United Kingdom, E11BB
Withdrawn
Research Team
R
Recordati
CONTACT
R
Recordati
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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