Metopirone (metyrapone) / Perrigo Company - LARVOL DELTA

Administration of metyrapone via nasogastric tube in a critically ill patient: A case report. (PubMed, Ann Endocrinol (Paris)) - No abstract available

Journal • Critical care • Cushing’s Disease • Endocrine Disorders

Sleep Loss and Circadian Misalignment - Mechanisms of Insulin Resistance (clinicaltrials.gov) - P4 | N=48 | Recruiting | Sponsor: Washington State University | Not yet recruiting ➔ Recruiting

Enrollment open

Prenatal Δ9-tetrahydrocannabinol exposure rewires glucocorticoid-hippocampal crosstalk and impairs memory under stress in adolescent rat offspring. (PubMed, Eur J Pharmacol) - "These findings demonstrate that prenatal THC exposure programs a stress-related vulnerability of hippocampal memory processes, which can be mitigated by reducing excessive glucocorticoid availability."

Journal • Preclinical • Alzheimer's Disease • Cognitive Disorders

Multiple endocrine neoplasia type 1 with concurrent Cushing's disease, prolactinoma, and multifocal pancreatic neuroendocrine tumors including insulinoma: a case report. (PubMed, Ups J Med Sci) - "Transsphenoidal surgery failed to achieve remission, and metyrapone provided partial control...Lanreotide achieved complete resolution of hypoglycemia, while 68Ga-NOTA-exendin-4 positron emission tomography/computed tomography did not identify a dominant lesion, supporting conservative management...It underscores the importance of multidisciplinary, individualized management, particularly when tumor multifocality limits surgical options. It also emphasizes the need to recognize complex endocrine syndromes in general clinical practice."

Journal • Cushing’s Disease • Endocrine Disorders • Gastrointestinal Neuroendocrine Tumor • Hypoglycemia • Neuroendocrine Tumor • Oncology • Pancreatic Cancer • Solid Tumor

Metyrapone in the Management of Severe Hypercortisolism Secondary to Ectopic Cushing’s Syndrome. (PubMed, Case Rep Endocrinol) - "Given the rarity, heterogeneity, and aggressiveness of this condition, rapid diagnosis and standardized pharmacologic approach are difficult to achieve. More data are needed to guide optimal therapy selection, dosing, and titration in ECS."

Journal • Cushing’s Disease • Endocrine Disorders • Metabolic Disorders • Nephrology • Renal Disease

Metyrapone-Associated Improvements in Health-Related Quality of Life in Cushing's Syndrome. (PubMed, Eur J Endocrinol) - P3 | "Metyrapone treatment is associated with meaningful, progressive HRQoL improvements across multiple symptom domains over 36 weeks, with a pattern consistent with rapid cortisol-mediated relief (eating, mood), delayed recovery in social and sexual domains, and persistent residual burden in symptom clusters likely driven by incomplete phenotypic reversal within the study timeframe."

HEOR • Journal • CNS Disorders • Cushing’s Disease • Depression • Endocrine Disorders • Mood Disorders • Psychiatry • IL2RA • ISG20

Sustained shrinkage of bilateral adrenal adenomas during long-term osilodrostat therapy. (PubMed, Eur J Endocrinol) - "Osilodrostat showed marked clinical and biochemical efficacy. This case suggests that osilodrostat may contribute to adrenal mass reduction, warranting further investigation."

Journal • Cushing’s Disease • Endocrine Disorders • Hematological Disorders • Nephrology • Renal Disease

Adrenal insufficiency diagnosis: new approaches to indeterminate baseline cortisol. (PubMed, Curr Opin Endocrinol Diabetes Obes) - "Emerging evidence supports a multimodal diagnostic approach combining baseline cortisol with adjunctive markers such as DHEAS and salivary cortisone. These strategies may reduce reliance on dynamic testing while maintaining diagnostic accuracy, ultimately improving patient convenience and healthcare efficiency."

Journal • Endocrine Disorders • Nephrology • Renal Disease

Case Report: a family presenting with β-adrenergic/vasopressin-responsive bilateral macronodular adrenal disease with an ARMC5 mutation treated with metyrapone monotherapy for more than 5 years. (PubMed, Front Endocrinol (Lausanne)) - "One patient developed progressive hypokalemia, eventually leading to unilateral adrenalectomy. These findings suggest that metyrapone monotherapy serves as an effective noninvasive treatment strategy for select familial BMAD patients, although close monitoring is warranted for potential complications."

Journal • Monotherapy • Cushing’s Disease • Endocrine Disorders • Nephrology

Case Report: ACTH-independent Cushing's syndrome caused by isolated micronodular adrenal disease in a 23-month-old girl. (PubMed, Front Endocrinol (Lausanne)) - "Biochemical assessment confirmed adrenocorticotropic hormone (ACTH)-independent hypercortisolism, with suppressed ACTH, loss of circadian cortisol rhythm, lack of cortisol suppression following dexamethasone administration, and increased 24-hour urinary free cortisol excretion...Adrenal scintigraphy with ¹³¹I-NP-59 after metyrapone-induced suppression demonstrated bilateral adrenal uptake, slightly more prominent on the right...This case highlights that isolated micronodular adrenal disease should be considered in very young children with ACTH-independent Cushing's syndrome even when adrenal magnetic resonance imaging is normal. Functional adrenal imaging may be useful in selected cases, and unilateral adrenalectomy may achieve durable remission while delaying permanent adrenal insufficiency."

Journal • Cardiovascular • Cushing’s Disease • Dermatology • Endocrine Disorders • Hypertension • Nephrology • Pediatrics • Renal Disease

11-deoxycortisol, other hormones and pituitary function characteristics in the diagnosis of adrenal insufficiency in the overnight oral metyrapone test. (PubMed, Endocr Connect) - "The oral metyrapone test is very useful for the characterization of adrenal function. If available, analysis of 11-deoxycortisol is better than measurements of other adrenal steroid hormones or corticotropin. However, there may be a bias from previous studies on the metyrapone test. Therefore, analysis of corticotropin, androstenedione, and 17-hydroxyprogesterone concentrations at baseline and the day after metyrapone administration are also very helpul for evaluation of adrenal function when the results from other pituitary function tests are included in the diagnostic process."

Journal • Endocrine Disorders • Nephrology • Oncology • Pituitary Gland Carcinoma • Renal Disease

Innate anxiety in rats determines vulnerability to social fear conditioning and social fear memory. (PubMed, Psychoneuroendocrinology) - "Blocking glucocorticoid synthesis before social fear acquisition slightly reduced generalised social fear in HAB rats. These findings demonstrate that innate anxiety determines the long-term formation of individual social fear memory, as assessed after a 24-h retention interval, in adult male rats and support rSFC as a useful experimental model to investigate selected neurobiological mechanisms relevant to SAD."

Journal • Preclinical • CNS Disorders • Mood Disorders • Psychiatry • Social Anxiety Disorder

Persistent Adrenocortical Insufficiency After Long-Term Metyrapone Treatment for Cushing's Disease. (PubMed, AACE Endocrinol Diabetes) - "Initial high-dose dexamethasone suppression and corticotropin-releasing hormone (CRH) tests suggested ectopic ACTH syndrome; however, extensive imaging failed to identify a source. Metyrapone was initiated and escalated to 3000 mg/d, followed by block-and-replacement with hydrocortisone (20 mg/d)...This case suggests that prolonged global adrenocortical dysfunction persisting over 1 year after metyrapone discontinuation. Clinicians should be aware of this potential complication and ensure appropriate glucocorticoid replacement and careful long-term monitoring."

Journal • Cushing’s Disease • Endocrine Disorders • Nephrology • Neuroendocrine Tumor • Oncology • Pituitary Gland Carcinoma • Renal Disease • Solid Tumor

Managing mild autonomous cortisol secretion (MACS): evaluating the role of medical treatment. (PubMed, Front Endocrinol (Lausanne)) - No abstract available

Journal

Therapeutic mechanism of combination of Epimedii Folium and Ligustri Lucidi Fructus for glucocorticoid synthesis-restricted asthma in rats (PubMed, Zhongguo Zhong Yao Za Zhi) - "The classic asthma model induced by ovalbumin(OVA) sensitization and challenge, a complex asthma animal model with specific suppression of hypothalamic-pituitary-adrenal(HPA) axis at the synthesis level of endogenous GC by intraperitoneal administration of metyrapone(Met), was established...The combination of Epimedii Folium and Ligustri Lucidi Fructus exerts multi-faceted regulatory effects on both the HPA axis and GC synthesis/metabolism in the peripheral tissue, synergistically ameliorating endogenous GC dysregulation in synthesis-restricted asthma. These findings suggest the potential therapeutic value of this combination against GC resistance and related disorders."

Journal • Preclinical • Asthma • Immunology • Pulmonary Disease • Respiratory Diseases • CYP11B1

Asthma increased the risk of atrial fibrillation: insights from clinical association to genetic and molecular evidence. (PubMed, BMC Pulm Med) - "Our study provides evidence that genetic predisposition to asthma is significantly associated with an increased risk of AF in the European population. Transcriptomic data analyses suggest that CPA3 and its interactions with therapeutic drugs could serve as potential biomarkers, offering new opportunities to improve prognosis in patients with coexisting asthma and AF."

Journal • Asthma • Atrial Fibrillation • Cardiovascular • Immunology • Pulmonary Disease • Respiratory Diseases • CPA3

Metyrapone Study for Patients Diagnosed With Mild Autonomous Cortisol Secretion - MACS (clinicaltrials.gov) - P2 | N=90 | Not yet recruiting | Sponsor: Mayo Clinic | Initiation date: Apr 2026 ➔ Aug 2026

Trial initiation date

Baxdrostat versus Osilodrostat: Steroid Biosynthesis in Human Adrenocortical Cells. (PubMed, Endocr Connect) - "Cells were stimulated with adrenocorticotropic hormone (10 nM) and exposed for 72 h to baxdrostat, osilodrostat, or metyrapone (0-10 μM). Baxdrostat demonstrated greater functional selectivity for CYP11B2, with minimal cortisol-axis impact and smaller upstream precursor accumulation than osilodrostat. These human-tissue data inform dose selection and safety monitoring strategies for selective aldosterone synthase inhibition in aldosterone-mediated and treatment-resistant hypertension."

Journal • Cardiovascular • Hypertension • Oncology • CYP11B1

Pathologic Fractures in Pregnancy: A Rare Presentation of Cushing'S Syndrome (ENDO 2026) - "There are currently no medications approved to treat CS in pregnancy, and when medical management is being considered for those waiting for surgery or for those at imminent risk, the options are limited to metyrapone or ketoconazole (1). CS and pregnancy cause elevated cortisol levels and similar symptoms, which pose a diagnostic challenge. The preferred treatment is surgery (either pituitary or adrenal) in the second trimester, as many of the medications are lacking strong evidence of efficacy and safety and pose a significant risk for the mother and fetus. Coordination by a multidisciplinary care team with an individualized approach is recommended."

Cardiovascular • Congestive Heart Failure • Cushing’s Disease • Diabetes • Endocrine Disorders • Gynecology • Heart Failure • Hypertension • Infectious Disease • Metabolic Disorders • Musculoskeletal Diseases • Orthopedics • Osteoporosis

Safety and Clinical Impact of Overnight Metyrapone in Mild Autonomous Cortisol Secretion: An Open-Label Phase 2 Trial (ENDO 2026) - P2 | " Study design: Open-Label, Phase 2 Clinical Trial (NCT06106295) Participants: Adults with MACS (post-dexamethasone serum cortisol >1.8 μg/dL) and benign adrenal imaging, prospectively enrolled from December 2023 to January 2025...Diurnal salivary cortisol and cortisone amplitude (morning–bedtime difference) increased, but was not statistically significant... Overnight metyrapone in MACS demonstrated a favorable safety profile with no cases of AI. Treatment was associated with reduction in glucocorticoid/androgen ratio and improvement in patient-reported sleep quality and depressive symptoms. These findings support further investigation of metyrapone in MACS."

Clinical • P2 data • Cardiovascular • CNS Disorders • Depression • Dyslipidemia • Endocrine Disorders • Genetic Disorders • Hypertension • Metabolic Disorders • Musculoskeletal Pain • Nephrology • Obesity • Osteoporosis • Renal Disease

ACTH Independent Cushing Syndrome Complicated by Cardiogenic Shock and Heart Transplantation: Challenges in Management. (ENDO 2026) - "Biochemical testing showed elevated late-night salivary cortisol (448 and 671 ng/dL), 24-hour urinary free cortisol of 125.4 μg/24 h, and failure to suppress on dexamethasone testing (1-mg cortisol 18 μg/dL; 2-mg cortisol 16.9 μg/dL; 8-mg cortisol 21.5 μg/dL with dexamethasone level 4040 ng/dL). As steroids were gradually tapered, she was transitioned to metyrapone as a bridge to definitive adrenalectomy. Other medications were contraindicated due to concerns for hepatotoxicity, QTc prolongation, and interactions with post-transplant immunosuppressive therapy."

Alopecia • Cardiovascular • Cognitive Disorders • Congestive Heart Failure • Cushing’s Disease • Endocrine Disorders • Genetic Disorders • Heart Failure • Obesity • Solid Organ Transplantation • Transplantation • CTNNB1

Short-Term Steroidogenesis Inhibition with High-Dose Metyrapone Therapy to Improve Clinical Outcomes Prior to Surgical Resection of Cortisol-Producing Adrenocortical Carcinoma (ENDO 2026) - "Patient received dexamethasone 0.5 mg orally daily (block and replace therapy) when AM cortisol decreased to < 10 μg/dL. Due to prolonged QT interval of 582 ms (normal QTc < 460) and concurrent amiodarone therapy, osilodrostat was not indicated... This case highlights the critical role of short-term (less than two weeks) preoperative adrenal steroidogenesis inhibition in managing hypercortisolism associated with ACC. High-dose metyrapone rapidly controlled cortisol excess, leading to improvement in clinical and biochemical parameters and optimization prior to surgery in a medically complex patient."

Clinical • Clinical data • Adrenal Cortex Carcinoma • Back Pain • Cardiovascular • Congestive Heart Failure • Cushing’s Disease • Diabetes • Endocrine Disorders • Genetic Disorders • Genito-urinary Cancer • Heart Failure • Hypertension • Metabolic Disorders • Musculoskeletal Diseases • Musculoskeletal Pain • Obesity • Oncology • Solid Tumor • Type 2 Diabetes Mellitus • Women's Health

A Rare Etiology of Cushing Syndrome Highlighted by Mifepristone-Related Adverse Effects (ENDO 2026) - "Mifepristone was held, aggressive electrolyte repletion was initiated, and spironolactone was continued, resulting in clinical and biochemical improvement...Prior endocrine evaluation demonstrated elevated late-night salivary cortisol levels (0.218, 0.384, 0.227), normal 24-hour urinary free cortisol (16), incomplete suppression on dexamethasone testing (cortisol 6), and a fasting ACTH of 8 pg/mL... This case highlights bilateral adrenal adenomas as an uncommon cause of Cushing syndrome and illustrates a serious complication of mifepristone therapy. Bilateral adrenalectomy is the standard treatment of choice for overt Cushing's syndrome. Medical management options include osilodrostat, ketoconazole, metyrapone, and mifepristone."

Adverse events • Cardiovascular • Coronary Artery Disease • Cushing’s Disease • Diabetes • Endocrine Disorders • Hypertension • Metabolic Disorders • Type 2 Diabetes Mellitus

Recurrent Ectopic Cushing'S Syndrome Associated with Neuroendocrine Tumors (ENDO 2026) - "Ketoconazole and metyrapone were started for hypercortisolemia. Biochemical and clinical control of hypercortisolism in ECS associated with NETs remains difficult to achieve. Moreover, ECS can lead to complications including increased cardiovascular risk, musculoskeletal impairment, metabolic disorders, infections, thrombosis, decreased quality of life, and worse survival. In patients with recurrent ectopic Cushing's disease who do not respond to resection of the primary NET, bilateral adrenalectomy should be considered early to eliminate the source of cortisol, improve symptoms of ECS, and prevent complications of uncontrolled Cushing's syndrome."

Adrenal Cortex Carcinoma • Carcinoid Tumor • Cardiovascular • CNS Disorders • Cushing’s Disease • Endocrine Cancer • Endocrine Disorders • Hematological Disorders • Infectious Disease • Insomnia • Metabolic Disorders • Musculoskeletal Diseases • Nephrology • Neuroendocrine Carcinoma • Neuroendocrine Neoplasm • Neuroendocrine Tumor • Oncology • Renal Disease • Sleep Disorder • Solid Tumor • Thrombosis

Etomidate Infusion to Bridge to Endoscopic Ultrasound-Guided Radiofrequency Ablation of an ACTH-Secreting Pancreatic Neuroendocrine Tumor in A Patient with Severe Cushing Syndrome (ENDO 2026) - "She was initially started on oral ketoconazole and metyrapone. This case highlights two critical management strategies in severe hypercortisolism: 1) the use of intravenous etomidate to rapidly lower serum cortisol in a critically ill patient with ectopic Cushing syndrome as a bridge to definitive therapy, and 2) the application of EUS-guided RFA to treat functional pNETs in high-risk patients who are not surgical candidates."

Clinical • Cardiovascular • Cushing’s Disease • Diabetes • Endocrine Disorders • Gastrointestinal Neuroendocrine Tumor • Hypertension • Infectious Disease • Nephrology • Neuroendocrine Tumor • Oncology • Pancreatitis • Pneumonia • Pulmonary Embolism • Renal Disease • Respiratory Diseases • Solid Tumor