A phase II, multi-site, open-label, dose-titration trial to investigate the safety, tolerability, pharmacokinetics, and efficacy of Lu AG13909 in adults with Cushing’s disease. (CUSHING)
Recipient organisationNIHR Wellcome Trust Birmingham Clinical Research Facility
NIHR supportRecorded as supported by this research centre
PeriodFeb 2025 — Sept 2026
In plain English
AI plain-English summary
A new drug, Lu AG13909, is being tested in adults with Cushing’s disease to see if it can safely lower their dangerously high cortisol levels. Cushing’s disease is caused by a pituitary tumour that drives the body to produce too much of the stress hormone cortisol. Chronically elevated cortisol damages nearly every organ system—causing severe weight gain, muscle wasting, high blood pressure, diabetes, and increased risk of infection. While several drugs exist to treat the condition, many have significant safety concerns or limited effectiveness. This trial directly addresses that gap by testing a new compound with a different mechanism of action. If Lu AG13909 proves safe and effective, it could become a new medical therapy for patients who currently have few good options. The trial is structured in multiple phases, including a long-term extension period lasting 52 weeks, to assess both immediate and sustained effects. Success would not cure the underlying tumour, but it could offer a better-tolerated way to control cortisol levels, reducing the long-term damage that currently leaves many patients with permanent health complications.
View original technical description
This trial is an interventional, multi-site, open-label, multiple-ascending-dose trial with dose titration. It will evaluate the effects of Lu AG13909 in adult participants with Cushing’s disease. The main goals of this study are to learn about: a)The effect of Lu AG13909 on cortisol levels,b) The safety and tolerability of Lu AG13909, and c) The pharmacokinetic parameters of Lu AG13909 (how the trial study drug is absorbed, distributed, and processed by the body) The trial is divided into three parts: ? Part A, consisting of 3 periods: an IV Titration Period, a SC Period, and a Safety Follow-up Period ? Part B, consisting of 3 periods: a SC Titration Period, a Maintenance Period, and a Safety Follow-up Period ? Extension Period, consisting of a Long-Term Efficacy/Safety Period after Part B and a Safety Follow-up PeriodParticipants will attend only one Safety Follow-up Period before leaving the trial either after Part A, Part B, or the Extension Period. The total duration for Part B will be between 32 weeks and 52 weeks. Extension period will be 52 weeks. Part A is NA for UK. The trial will include adult men and women (18-70 years of age) with Cushing’s Disease who have a confirmed diagnosis of ACTH driven Cushing’s disease of pituitary source as per current guidelines. The trial drug has a therapeutic potential in the treatment of Cushing’s disease, which is characterized by chronically elevated ACTH and cortisol. In Cushing’s disease, there is a high unmet need for medical therapies. Several products are approved for pharmacological treatment of Cushing’s disease, but many have safety concerns and/or limited efficacy. It is possible that by participating in this trial, the participant’s condition may improve, but this cannot be guaranteed. Regardless of whether the individual participant benefits from participating in this trial, participation will contribute to the development of a potential future treatment option for Cushing’s disease.
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