{"doi":"10.1210/clinem/dgaa381","title":"A Phase 2, Multicenter Study of Nevanimibe for the Treatment of Congenital Adrenal Hyperplasia","abstract":"CONTEXT: Patients with classic congenital adrenal hyperplasia (CAH) often require supraphysiologic glucocorticoid doses to suppress adrenocorticotropic hormone (ACTH) and control androgen excess. Nevanimibe hydrochloride (ATR-101), which selectively inhibits adrenal cortex function, might reduce androgen excess independent of ACTH and thus allow for lower glucocorticoid dosing in CAH. 17-hydroxyprogesterone (17-OHP) and androstenedione are CAH biomarkers used to monitor androgen excess. OBJECTIVE: Evaluate the efficacy and safety of nevanimibe in subjects with uncontrolled classic CAH. DESIGN: This was a multicenter, single-blind, dose-titration study. CAH subjects with baseline 17-OHP ≥4× the upper limit of normal (ULN) received the lowest dose of nevanimibe for 2 weeks followed by a single-blind 2-week placebo washout. Nevanimibe was gradually titrated up if the primary outcome measure (17-OHP ≤2× ULN) was not met. A total of 5 nevanimibe dose levels were possible (125, 250, 500, 750, 1000 mg twice daily). RESULTS: The study enrolled 10 adults: 9 completed the study, and 1 discontinued early due to a related serious adverse event. At baseline, the mean age was 30.3 ± 13.8 years, and the maintenance glucocorticoid dose, expressed as hydrocortisone equivalents, was 24.7 ± 10.4 mg/day. Two subjects met the primary endpoint, and 5 others experienced 17-OHP decreases ranging from 27% to 72% during nevanimibe treatment. The most common side effects were gastrointestinal (30%). There were no dose-related trends in adverse events. CONCLUSIONS: Nevanimibe decreased 17-OHP levels within 2 weeks of treatment. Larger studies of longer duration are needed to further evaluate its efficacy as add-on therapy for CAH.","journal":"The Journal of Clinical Endocrinology & Metabolism","year":2020,"id":97860,"datarank":1.0424562779019064,"base_score":3.4965075614664802,"endowment":3.4965075614664802,"self_citation_contribution":0.5244761342199721,"citation_network_contribution":0.5179801436819342,"self_endowment_contribution":0.5244761342199721,"citer_contribution":0.5179801436819342,"corpus_percentile":null,"corpus_rank":null,"citation_count":32,"citer_count":26,"citers_with_citation_signal":18,"citers_with_endowment":18,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.9688,"is_data_producer":true,"deposit_databanks":{"ClinicalTrials.gov":["NCT02804178"]},"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2020-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":404052,"name":"Deborah P. Merke","orcid":"0000-0002-3746-0460","position":1,"is_corresponding":false},{"id":483426,"name":"Maria G. Vogiatzi","orcid":"0000-0002-1941-8277","position":2,"is_corresponding":false},{"id":479193,"name":"Alice Y. Chang","orcid":"0000-0001-7108-0499","position":3,"is_corresponding":false},{"id":364718,"name":"Adina F. Turcu","orcid":"0000-0001-9831-6190","position":4,"is_corresponding":false},{"id":415186,"name":"Elizabeth Joyal","orcid":null,"position":5,"is_corresponding":false},{"id":484079,"name":"Vivian Lin","orcid":null,"position":6,"is_corresponding":false},{"id":483427,"name":"Lauren Weintraub","orcid":"0009-0007-3802-711X","position":7,"is_corresponding":false},{"id":484080,"name":"Marianne R Plaunt","orcid":null,"position":8,"is_corresponding":false},{"id":484081,"name":"Pharis Mohideen","orcid":null,"position":9,"is_corresponding":false},{"id":369854,"name":"Richard J. Auchus","orcid":"0000-0001-6815-6181","position":10,"is_corresponding":false},{"id":483425,"name":"Diala El‐Maouche","orcid":"0000-0002-0936-2249","position":0,"is_corresponding":true}],"reference_count":22,"raw_metadata":null,"created_at":"2026-07-18T22:36:02.818637Z","pmid":"32589738","pmcid":null,"fwci":null,"citation_percentile":null,"influential_citations":0,"oa_status":null,"license":null,"views":0,"total_file_size_bytes":0,"version_count":0,"fair_f":null,"fair_a":null,"fair_i":null,"fair_r":null,"fair_zscore":null,"fair_rationale":null,"fair_model":null,"fair_agent_version":null,"fair_fulltext_source":null,"fair_has_llm":null,"fair_computed_at":null,"clinical_trials":[],"software_tools":[],"db_accessions":[],"linked_datasets":[],"topics":[]}