Trial reportPituitary2022
Long-term efficacy and safety of osilodrostat in Cushing's disease: final results from a Phase II study with an optional extension phase (LINC 2).
Trial report in Pituitary, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT01331239 (A Proof of Concept, Open-label, Forced Titration, Multi-center Study to Assess the Safety/Tolerability and Efficacy of 10-weeks Treatment of LCI699 in Patients With Cushing's Disease), which is not on this map. Cited by 28 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Proof of Concept, Open-label, Forced Titration, Multi-center Study to Assess the Safety/Tolerability and Efficacy of 10-weeks Treatment of LCI699 in Patients With Cushing's Disease
Who cites it
28 citing papers in PubMed, 36 citations in OpenAlex.
- Impact of Osilodrostat on Health-Related Quality of Life in Cushing's Disease: Analysis of Patient-Reported Outcomes From the Phase III LINC 3 and LINC 4 Clinical Trials.Clinical endocrinology · 2026Trial
- Prospective study of metyrapone in endogenous Cushing's syndrome (PROMPT).European journal of endocrinology · 2025Trial
- Osilodrostat treatment in patients with Cushing's disease of Asian or non-Asian origin: a pooled analysis of two Phase III randomized trials (LINC 3 and LINC 4).Endocrine journal · 2024Trial
- Improvement in clinical features of hypercortisolism during osilodrostat treatment: findings from the Phase III LINC 3 trial in Cushing's disease.Journal of endocrinological investigation · 2024Trial
- First-in-human study evaluating safety, pharmacokinetics, and pharmacodynamics of lorundrostat, a novel and highly selective aldosterone synthase inhibitor.Clinical and translational science · 2024Trial
- Real-World Osilodrostat Effectiveness and Safety in Nonpituitary Cushing Syndrome.The Journal of clinical endocrinology and metabolism · 2026Observational
- Symptom management with osilodrostat in multiple endocrine neoplasia type 1 with a Cushing syndrome presentation.JCEM case reports · 2026Article
- Individualized osilodrostat treatment for patients with ACTH-dependent Cushing's syndrome: real-world evidence.Frontiers in endocrinology · 2026Article
- Controlling cortisol excess and comorbidities in Cushing's syndrome with osilodrostat.Pituitary · 2025Article
- Phenotypic and functional characteristics of pituitary adenoma stem cells.World journal of clinical cases · 2025Article
- Safety management in Cushing syndrome during osilodrostat treatment based on morning blood cortisol level.Endocrine journal · 2025Article
- Osilodrostat Safety Profile: Findings from Real-World Data in the FAERS Database.Journal of clinical medicine · 2025Article
- Osilodrostat Treatment for Adrenal and Ectopic Cushing Syndrome: Integration of Clinical Studies With Case Presentations.Journal of the Endocrine Society · 2025Review
- Medical management pathways for Cushing's disease in pituitary tumors centers of excellence (PTCOEs).Pituitary · 2025Article
- Safety assessment of Osilodrostat: The adverse event analysis based on FAERS database by means of disproportionality analysis.PloS one · 2025Article
- Prolonged adrenal suppression after osilodrostat discontinuation in a patient with Cushing's disease with eventual hypercortisolism relapse: Case Report and literature review.Frontiers in medicine · 2025Article
- Case Report: Ectopic Cushing's syndrome caused by a pancreatic neuroendocrine tumor managed through dual steroidogenesis inhibition and endoscopic ultrasound-guided ethanol ablation.Frontiers in endocrinology · 2025Article
- Medical treatment of functional pituitary adenomas, trials and tribulations.Journal of neuro-oncology · 2024Review
- Prolonged Adrenal Insufficiency After Osilodrostat Exposure With Eventual Recovery of Adrenal Function.JCEM case reports · 2024Article
- Osilodrostat: A Novel Potent Inhibitor of 11-Beta-Hydroxylase for the Treatment of Cushing's Syndrome.TouchREVIEWS in endocrinology · 2024Review
Corrections and comments
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Authors and funding
10 authors at 9 institutions in 5 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundMany patients with Cushing's disease (CD) require long-term medical therapy to control their hypercortisolism. In the core phase of a Phase II study (LINC 2; NCT01331239), osilodrostat normalized mean urinary free cortisol (mUFC) in 78.9% of patients with CD. Here, we report long-term efficacy and safety data for osilodrostat following completion of an optional extension to LINC 2.
methodsAdult patients with CD were enrolled in a 22-week prospective Phase II study. Patients with mUFC ≤ upper limit of normal (ULN) or receiving clinical benefit at week 22 could enter the optional extension. The proportion of complete (mUFC ≤ ULN) or partial (mUFC > ULN but ≥ 50% decrease from baseline) mUFC responders was assessed over time.
resultsSixteen of 19 enrolled patients entered the extension. Median (range) osilodrostat exposure from baseline to study end was 5.4 years (0.04-6.7); median (range) average dose was 10.6 mg/day (1.1-47.9). Overall response rate (complete and partial mUFC responders) was consistently ≥ 50%. Sustained control of most cardiovascular-related parameters was observed during the extension. The long-term safety profile was consistent with that reported during the core phase. Testosterone levels (females) decreased towards baseline levels during long-term follow-up, with no new or worsening cases of hirsutism during the extension.
conclusionsIn the longest prospective study of a steroidogenesis inhibitor to date, osilodrostat provided sustained reductions in mUFC for up to 6.7 years of treatment, with no new safety signals emerging during the extension. These findings support osilodrostat as an effective long-term treatment for patients with CD.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.