Long-Term Use of Relacorilant for Cushing Syndrome

Not currently recruiting at 88 trial locations

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: Corcept Therapeutics

Must be taking: Relacorilant

Disqualifiers: Hypothyroidism, Hyperthyroidism, Hypertension, Renal failure

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial examines the long-term safety of relacorilant for individuals with Cushing syndrome, a condition where the body produces excess cortisol, the stress hormone. Participants must have completed a previous study on relacorilant and demonstrated benefit from the treatment. Those with uncontrolled thyroid issues or high blood pressure may not qualify. As a Phase 2 trial, the research focuses on assessing the treatment's effectiveness in an initial, smaller group.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It is best to discuss this with the trial team or your doctor.

Is there any evidence suggesting that relacorilant is likely to be safe for humans?

Research has shown that relacorilant is being tested for its safety in treating Cushing syndrome. In earlier studies, patients experienced improvements in symptoms like high blood pressure without serious heart problems, such as QT prolongation, which affects heart rhythm.

Another study found that relacorilant was generally well-tolerated by patients with Cushing syndrome, meaning most people did not have severe side effects.

While this trial examines long-term safety, earlier research offers encouraging signs that relacorilant is safe for people with this condition. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Cushing syndrome?

Most treatments for Cushing Syndrome, like ketoconazole or metyrapone, work by inhibiting cortisol production. However, relacorilant is unique because it targets the glucocorticoid receptor, blocking cortisol's effects without reducing its production. This approach can potentially minimize the side effects associated with lowering cortisol levels too much, offering a more balanced treatment. Researchers are excited about relacorilant because it might provide effective symptom control with fewer complications, improving overall quality of life for patients.

What evidence suggests that relacorilant might be an effective treatment for Cushing syndrome?

Research has shown that relacorilant, the treatment under study in this trial, holds promise for treating endogenous Cushing syndrome. Studies found that patients taking relacorilant experienced a noticeable drop in blood pressure, with an average decrease of 6.6 mmHg in the systolic reading. Patients also benefited from weight loss and a smaller waist size. These improvements suggest that relacorilant may help manage Cushing syndrome symptoms by adjusting the body's response to cortisol, a stress hormone often elevated in this condition. Overall, these findings support relacorilant's potential effectiveness for people with Cushing syndrome. ^{2 3 6 7 8}

Who Is on the Research Team?

Ying Ru, MD, PhD

Principal Investigator

Corcept Therapeutics

Are You a Good Fit for This Trial?

This trial is for patients with Cushing Syndrome who have finished a previous Corcept-sponsored relacorilant study and took their medication at least 80% of the time. They should not have dropped out early, nor should they have uncontrolled thyroid issues, poorly controlled high blood pressure, or severe kidney failure.

Inclusion Criteria

You have participated in a previous study on relacorilant for Cushing syndrome and followed the dosing schedule at least 80% of the time.

The doctor believes it is beneficial for you to keep taking relacorilant.

Exclusion Criteria

My thyroid condition is not under control.

My high blood pressure is not well-managed.

My kidney function is severely reduced.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Open-label extension

Participants continue therapy with relacorilant for long-term safety evaluation

36 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Relacorilant

Trial Overview The trial is testing the long-term safety of a drug called relacorilant in those with endogenous Cushing Syndrome. It's an open-label extension which means everyone knows they are getting relacorilant and there's no placebo group.

How Is the Trial Designed?

Group I: relacorilant (CORT125134) Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Corcept Therapeutics

Lead Sponsor

Trials

72

Recruited

7,700+

Dr. Joseph K. Belanoff

Corcept Therapeutics

Chief Executive Officer since 2014

MD from Stanford University

Dr. William Guyer

Corcept Therapeutics

Chief Medical Officer since 2021

PharmD

Published Research Related to This Trial

Endogenous Cushing's syndrome is a serious condition that can lead to increased health risks if not treated properly, and medical treatments are essential for managing cortisol levels, especially after surgery or while awaiting radiation therapy.

Recent advancements in medical treatments include new steroidogenesis inhibitors, pituitary-directed drugs, and glucocorticoid receptor antagonists, which may offer fewer side effects and target the underlying causes of Cushing's syndrome more effectively.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Medical Management of Cushing's Syndrome: Current and Emerging Treatments. Hinojosa-Amaya, JM., Cuevas-Ramos, D., Fleseriu, M.[2020]

Pasireotide, both in its twice-daily subcutaneous and once-monthly intramuscular formulations, has shown effective treatment outcomes for Cushing's disease in clinical trials, with a generally good safety profile, although it can cause hyperglycemia that requires monitoring.

Osilodrostat, a novel agent, has demonstrated rapid and sustained control of Cushing's disease in recent clinical trials, indicating its potential as an effective treatment option alongside other investigational drugs like levoketoconazole and mifepristone.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Medical Treatment of Cushing's Disease: An Overview of the Current and Recent Clinical Trials. Pivonello, R., Ferrigno, R., De Martino, MC., et al.[2021]

Recent advancements in medical therapies for Cushing's syndrome include drugs that target dopamine and somatostatin receptors, which can control cortisol production in up to 40% of patients, providing a viable option when surgery is not successful.

New drug candidates like osilodrostat, levoketoconazole, and CORT125134 are being evaluated in multicenter trials, offering hope for more effective and personalized treatments for Cushing's syndrome.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Advances in the medical treatment of Cushing's syndrome. Feelders, RA., Newell-Price, J., Pivonello, R., et al.[2022]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT02804750

NCT02804750 | Study to Evaluate CORT125134 in ... The purpose of this study was to evaluate the safety and efficacy of CORT125134 for treatment of endogenous Cushing's syndrome.

2. corcept.com corcept.com/publications/open-label-extension-study-evaluating-the-safety-and-efficacy-of-long-term-use-of-relacorilant-in-patients-with-endogenous-hypercortisolism-cushing-syndrome-interim-analysis-results-2025/

Open-label Extension Study Evaluating the Safety and ... Evaluating the safety and efficacy of long-term use of relacorilant in patients with endogenous hypercortisolism (cushing syndrome): interim analysis results.

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8317576/

Relacorilant, a Selective Glucocorticoid Receptor Modulator ... We evaluated the efficacy and safety of relacorilant in patients with endogenous Cushing syndrome (CS). Materials and Methods. A single-arm, open-label ...

4. academic.oup.com academic.oup.com/jes/article/8/Supplement_1/bvae163.1287/7814117

9323 Open-label Results From Grace, A Phase 3 Double ... Improvements in other signs and symptoms of CS were also observed, including weight loss (-3.3 ± 5.9 kg), reduction in waist circumference (-2.8 ...

5. empr.com empr.com/news/relacorilant-review-for-cushing-syndrome/

Relacorilant Under Review for Patients With Cushing ... Results showed patients treated with relacorilant saw a statistically significant improvement in mean systolic blood pressure (reduction of 6.6 ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT03604198

NCT03604198 | Extension Study to Evaluate the Safety of ... This is an open-label extension study to evaluate the long-term safety of relacorilant in patients with endogenous Cushing syndrome.

7. endocrinologyadvisor.com endocrinologyadvisor.com/news/relacorilant-review-for-cushing-syndrome/

Relacorilant Under Review for Patients With Cushing ... Results showed patients treated with relacorilant saw a statistically significant improvement in mean systolic blood pressure (reduction of 6.6 ...

8. sciencedirect.com sciencedirect.com/science/article/pii/S1530891X23005712

Relacorilant, a Selective Glucocorticoid Receptor ... This analysis shows that relacorilant was not associated with QT prolongation in healthy individuals or patients with Cushing syndrome.