Osilodrostat for Cushing's Disease
Trial Summary
What is the purpose of this trial?
Multicenter, open-label, non-comparative study to evaluate the pharmacokinetics, pharmacodynamics, and tolerability of osilodrostat in children and adolescent patients with Cushing's syndrome.
Will I have to stop taking my current medications?
The trial requires a washout period (time without taking certain medications) for any drugs used to lower cortisol levels, which is 5 half-lives of the drug. If you're taking investigational drugs, you must stop them 30 days before joining the trial or after a washout period of at least 5 half-lives, whichever is longer.
What data supports the effectiveness of the drug osilodrostat for treating Cushing's disease?
What safety data exists for Osilodrostat in humans?
Osilodrostat, also known as Isturisa, has been studied for safety in treating Cushing's disease, a condition caused by high levels of cortisol (a stress hormone). Clinical studies have evaluated its long-term safety, and it is approved for use in both the United States and Europe, indicating it has been deemed generally safe for human use in this context.12346
How is the drug osilodrostat unique in treating Cushing's disease?
Osilodrostat is unique because it is an oral medication that works by inhibiting an enzyme called 11β-hydroxylase, which is involved in the production of cortisol, a hormone that is often overproduced in Cushing's disease. This makes it a novel option for patients who cannot undergo surgery or for whom surgery has not been successful.12345
Research Team
Recordati AG
Principal Investigator
Recordati AG
Eligibility Criteria
This trial is for children and adolescents with Cushing's disease who have either failed surgery, are waiting for it, or can't have it yet. They should show signs of the disease like growth issues despite weight gain and must be able to swallow tablets. Consent from parents or guardians is required.Inclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive osilodrostat for pharmacokinetics, pharmacodynamics, and tolerability evaluation
Follow-up
Participants are monitored for safety and effectiveness after treatment
Extension
Optional 9-month extension period for participants benefiting from therapy, assessing PD activity and safety/tolerability
Treatment Details
Interventions
- LCI699
LCI699 is already approved in European Union, United States for the following indications:
- Cushing's disease
- Cushing's disease
Find a Clinic Near You
Who Is Running the Clinical Trial?
Novartis Pharmaceuticals
Lead Sponsor
Dr. Vas Narasimhan
Novartis Pharmaceuticals
Chief Executive Officer since 2018
MD from Harvard Medical School
Dr. Shreeram Aradhye
Novartis Pharmaceuticals
Chief Medical Officer since 2021
MD
RECORDATI GROUP
Lead Sponsor