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A study of osilodrostat in children and adolescents with Cushing's disease
EU CTIS ID: 2024-516825-30-00
What this study is testing
To evaluate the pharmacokinetics (PK) of osilodrostat in children and adolescents 6 to <18 years of age with Cushing's Disease
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Male and female children and adolescents from 6 to < 18 years of age with Cushing's disease of endogenous origin who have failed surgery or are awaiting surgery or for whom surgery is not an immediate option
- Body weight greater 30 kg
- Confirmed diagnosis of Cushing's disease
- Able to swallow study drug tablets (not crushed or split)
- Parents or legal guardians able to provide consent/assent
You likely can't join if
- Macroadenoma complicated by compressive symptoms
- Hypertensive patients with uncontrolled blood pressure
- Patients who have undergone any major surgery within 1 month
- Patients who have undergone trans-sphenoidal pituitary surgery within 6 weeks prior to screening, unless they have clear evidence of persistent hypercortisolemia or persistent biochemical changes consistent with Cushing's disease
- Use of or anticipated use of systemic glucocorticoid medications 1 month prior to screening.
- Uncontrolled hypothyroidism as evidenced by Free T4 < 0.8 ng/dl.
See the full eligibility criteria
- Male and female children and adolescents from 6 to < 18 years of age with Cushing's disease of endogenous origin who have failed surgery or are awaiting surgery or for whom surgery is not an immediate option
- Body weight greater 30 kg
- Confirmed diagnosis of Cushing's disease
- Able to swallow study drug tablets (not crushed or split)
- Parents or legal guardians able to provide consent/assent
- Macroadenoma complicated by compressive symptoms
- Hypertensive patients with uncontrolled blood pressure
- Patients who have undergone any major surgery within 1 month
- Patients who have undergone trans-sphenoidal pituitary surgery within 6 weeks prior to screening, unless they have clear evidence of persistent hypercortisolemia or persistent biochemical changes consistent with Cushing's disease
- Use of or anticipated use of systemic glucocorticoid medications 1 month prior to screening.
- Uncontrolled hypothyroidism as evidenced by Free T4 < 0.8 ng/dl.
- Uncontrolled hyperthyroidism
- Diabetic patients with poorly controlled diabetes as evidenced by HbA1c > 8.5 % or not optimally treated for diabetes mellitus as judged by the investigator
- Positive pregnancy test in females of childbearing potential
- Female patients of childbearing potential who do not agree to use highly effective birth control methods
- Pregnant or nursing (lactating) women.
- Insufficient washout period from any other medication used to lower cortisol levels
- Any medical condition that would, in the investigator's judgment, prevent the patient's participation in the clinical study due to safety concerns or compliance with clinical study procedures.
- Use of concomitant prohibited medications (see section 6.2.2).
- Use of other investigational drugs at the time of enrollment
- History of hypersensitivity to drugs of the same or similar chemical classes as osilodrostat
- History of malignancy of any organ system
- Moderate to severe renal impairment
- Serum ALT and/or AST > 3 x ULN, or total bilirubin > 1.5 x ULN
- History of thrombosis
- Risk factors for QTc prolongation or Torsade de Pointes, including: 9a. patients with a baseline QTcF > 450 ms 9b. personal or family history of long QT syndrome 9c. concomitant medications known to prolong the QT interval (see Section 6.2.2.1) 9d. patients with hypokalemia, hypocalcaemia, or hypomagnesaemia, if not corrected before pre-dose Day 0. In case of uncorrected hypokalemia (<3.5 mEq/L), the screening period may be used to correct hypokalemia prior to starting study drug. Use of potassium supplements and/or mineralocorticoid antagonists is permitted during the study. 9e. Patients with a history of significant cardiovascular disease (based on the opinion of the investigator) such as: structural cardiovascular abnormalities, arrhythmia, etc.
The study team makes the final eligibility decision.
Where it's taking place
- United Kingdom
- United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United Kingdom; United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.