12 Participants Needed

Osilodrostat for Cushing's Disease

Recruiting at 21 trial locations
NP
R
Overseen ByRecordati
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial
Approved in 2 JurisdictionsThis treatment is already approved in other countries

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?

Osilodrostat has been shown to be effective in treating Cushing's disease, as it is an approved drug in the US and Europe for this condition. Clinical studies have demonstrated its ability to reduce excess cortisol levels, which is the main problem in Cushing's disease.12345

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

RA

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

I have Cushing's disease and surgery didn't work, is pending, or isn't an option for me.
My growth has slowed down while I've gained weight, and doctors suspect I might have Cushing's disease.
Parents or legal guardians able to provide consent/assent
See 5 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

up to 4 weeks

Treatment

Participants receive osilodrostat for pharmacokinetics, pharmacodynamics, and tolerability evaluation

12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Extension

Optional 9-month extension period for participants benefiting from therapy, assessing PD activity and safety/tolerability

9 months

Treatment Details

Interventions

  • LCI699
Trial Overview The study tests Osilodrostat (LCI699), focusing on how the drug moves in the body (PK), its effects on the disease (PD), and its safety in young patients with Cushing's disease. It's an open-label study, meaning everyone knows what treatment they're getting.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: LCI699 (osilodrostat)Experimental Treatment1 Intervention
Subjects with cushing's syndrome taking LCI699 (osilodrostat)

LCI699 is already approved in European Union, United States for the following indications:

🇪🇺
Approved in European Union as Isturisa for:
  • Cushing's disease
🇺🇸
Approved in United States as Isturisa for:
  • Cushing's disease

Find a Clinic Near You

Who Is Running the Clinical Trial?

Novartis Pharmaceuticals

Lead Sponsor

Trials
2,963
Recruited
4,275,000+
Founded
1996
Headquarters
Basel, Switzerland
Known For
Precision medicine
Top Products
Gleevec, Cosentyx, Entresto, Kisqali
Dr. Vas Narasimhan profile image

Dr. Vas Narasimhan

Novartis Pharmaceuticals

Chief Executive Officer since 2018

MD from Harvard Medical School

Dr. Shreeram Aradhye profile image

Dr. Shreeram Aradhye

Novartis Pharmaceuticals

Chief Medical Officer since 2021

MD

RECORDATI GROUP

Lead Sponsor

Trials
13
Recruited
4,500+

Findings from Research

Osilodrostat is an effective treatment for Cushing's disease, significantly reducing 24-hour mean urinary free cortisol levels in 86% of participants in a pivotal phase 3 study, compared to 29% in the placebo group.
While osilodrostat is generally safe, it requires careful monitoring for potential side effects like QT interval prolongation and increased cortisol precursors, which can lead to complications such as hypokalemia and hypertension.
Osilodrostat: A Novel Steroidogenesis Inhibitor to Treat Cushing's Disease.Dougherty, JA., Desai, DS., Herrera, JB.[2021]
Osilodrostat is an effective treatment for Cushing disease, rapidly reducing 24-hour urinary free cortisol levels and improving related health parameters such as glycemia, blood pressure, body weight, and quality of life.
While osilodrostat has tolerable adverse effects, including adrenal insufficiency and hypokalemia, careful monitoring and a slower dose increase can help manage these risks, especially due to potential drug interactions that may cause QT prolongation.
Osilodrostat: A Review of Recent Clinical Studies and Practical Recommendations for its Use in the Treatment of Cushing Disease.Yuen, KCJ.[2021]
Osilodrostat is an effective oral treatment for endogenous Cushing's syndrome, showing excellent efficacy in controlling hypercortisolism based on multiple studies, including LINC1, LINC2, and LINC3.
While osilodrostat is generally well-tolerated, it carries risks such as adrenal insufficiency, hypokalemia, and potential pituitary tumor enlargement, necessitating careful monitoring during treatment.
Osilodrostat oral tablets for adults with Cushing's disease.Martino, M., Aboud, N., Lucchetti, B., et al.[2022]

References

Osilodrostat: A Novel Steroidogenesis Inhibitor to Treat Cushing's Disease. [2021]
Osilodrostat: A Review of Recent Clinical Studies and Practical Recommendations for its Use in the Treatment of Cushing Disease. [2021]
Osilodrostat oral tablets for adults with Cushing's disease. [2022]
Osilodrostat: First Approval. [2020]
Drug Interaction Potential of Osilodrostat (LCI699) Based on Its Effect on the Pharmacokinetics of Probe Drugs of Cytochrome P450 Enzymes in Healthy Adults. [2020]
Long-term efficacy and safety of osilodrostat in patients with Cushing's disease: results from the LINC 4 study extension. [2023]
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