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New treatment option for Cushing Syndrome
Official title Pharmacokinetic (PK), Pharmacodynamic (PD) and Tolerability of Osilodrostat in Pediatric Patients With Cushing's Syndrome
ClinicalTrials.gov ID: NCT03708900
What this study is testing
What is LCI699?
LCI699 is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for cushing syndrome.
Also referred to as osilodrostat.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- Multicenter, open-label, non-comparative study to evaluate the pharmacokinetics, pharmacodynamics, and tolerability of osilodrostat in children and adolescent patients with Cushing's syndrome.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 17
You may be able to join if
- 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...
- Patients must weigh \> 10 kg.
- The diagnosis of Cushing's syndrome must be confirmed by each of the following: 3a) The clinical criterion of decreasing growth percentiles with...
You likely can't join if
- Patients with macroadenoma complicated by compressive symptoms (requiring urgent surgical intervention) or at high risk for compressive symptoms due...
- Insufficient washout period from any other medication used to lower cortisol levels (5 half-lives of any drug).
- Use of other investigational drugs at the time of enrollment, or within 30 days, or prior to completion of a wash-out duration that is at least 5...
- History of hypersensitivity to drugs of the same or similar chemical classes as osilodrostat.
- History of malignancy of any organ system (other than localized basal cell carcinoma of the skin), treated or untreated, within the past 5 years...
- Patients with moderate to severe renal impairment (estimated GFR \< 60 mL/min by the Creatinine-based "Bedside Schwartz" equation).
See the full eligibility 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...
- Patients must weigh \> 10 kg.
- 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...
- Patients with macroadenoma complicated by compressive symptoms (requiring urgent surgical intervention) or at high risk for compressive symptoms due to mass effect of tumor (concern of corticotroph tumor progression).
- Insufficient washout period from any other medication used to lower cortisol levels (5 half-lives of any drug).
- Use of other investigational drugs at the time of enrollment, or within 30 days, or prior to completion of a wash-out duration that is at least 5 half- lives of the drug, at the time of enrollment, whichever is longer...
- History of hypersensitivity to drugs of the same or similar chemical classes as osilodrostat.
- History of malignancy of any organ system (other than localized basal cell carcinoma of the skin), treated or untreated, within the past 5 years, regardless of whether there is evidence of local recurrence or metastases.
- Patients with moderate to severe renal impairment (estimated GFR \< 60 mL/min by the Creatinine-based "Bedside Schwartz" equation).
- Patients with serum ALT and/or AST \> 3 x ULN, or total bilirubin \> 1.5 x ULN.
- History of thrombosis.
- Patients with 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...
- Hypertensive patients with uncontrolled blood pressure defined as SBP \> 150 and/or DBP \> 100 or not optimally treated for hypertension as judged by the investigator.
- Patients who have undergone any major surgery within 1 month.
- Patients who have undergone trans-sphenoidal pituitary surgery within 6 weeks prior to screening are not eligible, unless they have clear evidence of persistent hypercortisolism or persistent biochemical changes...
- 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 hyper thyroidism.
- 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.
- 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. Any severe, acute, or...
- Use of concomitant prohibited medications
The study team makes the final eligibility decision.
Where it's taking place
- San Francisco, California, United States
- Cape Coral, Florida, United States
- Chicago, Illinois, United States
- Bethesda, Maryland, United States
- Weslaco, Texas, United States
- Jette, Brussels Capital, Belgium
- Varna, Bulgaria
- Paris, France
- Pisa, PI, Italy
- Roma, Italy
- Ljubljana, Slovenia
- Liverpool, United Kingdom
- London, United Kingdom
Compensation & support
Compensation mentioned.
ClinicalTrials.gov doesn't provide a reliable structured field for payment or travel support - confirm details with the study team.
Questions & answers
Do participants get paid in this trial?
This study's listing includes signals that participants may be compensated or receive a stipend. Amounts vary and are set by the study team - confirm the details with them.
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, 2 years to 17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include San Francisco, California, United States; Cape Coral, Florida, United States; Chicago, Illinois, United States; Bethesda, Maryland, United States; Weslaco, Texas, United States; Jette, Brussels Capital, Belgium and 7 more location(s). Enter your location above to see the nearest site and check your eligibility.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.