{"doi":"10.1093/brain/awad003","title":"The effect of GLP-1RA exenatide on idiopathic intracranial hypertension: a randomized clinical trial","abstract":"Therapeutics to reduce intracranial pressure are an unmet need. Preclinical data have demonstrated a novel strategy to lower intracranial pressure using glucagon-like peptide-1 (GLP-1) receptor signalling. Here, we translate these findings into patients by conducting a randomized, placebo-controlled, double-blind trial to assess the effect of exenatide, a GLP-1 receptor agonist, on intracranial pressure in idiopathic intracranial hypertension. Telemetric intracranial pressure catheters enabled long-term intracranial pressure monitoring. The trial enrolled adult women with active idiopathic intracranial hypertension (intracranial pressure >25 cmCSF and papilloedema) who receive subcutaneous exenatide or placebo. The three primary outcome measures were intracranial pressure at 2.5 h, 24 h and 12 weeks and alpha set a priori at less than 0.1. Among the 16 women recruited, 15 completed the study (mean age 28 ± 9, body mass index 38.1 ± 6.2 kg/m2, intracranial pressure 30.6 ± 5.1 cmCSF). Exenatide significantly and meaningfully lowered intracranial pressure at 2.5 h -5.7 ± 2.9 cmCSF (P = 0.048); 24 h -6.4 ± 2.9 cmCSF (P = 0.030); and 12 weeks -5.6 ± 3.0 cmCSF (P = 0.058). No serious safety signals were noted. These data provide confidence to proceed to a phase 3 trial in idiopathic intracranial hypertension and highlight the potential to utilize GLP-1 receptor agonist in other conditions characterized by raised intracranial pressure.","journal":"Brain","year":2023,"id":315606,"datarank":0.0,"base_score":0.0,"endowment":0.0,"self_citation_contribution":0.0,"citation_network_contribution":0.0,"self_endowment_contribution":0.0,"citer_contribution":0.0,"corpus_percentile":null,"corpus_rank":null,"citation_count":147,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.9591,"is_data_producer":false,"deposit_databanks":null,"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2023-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":1017014,"name":"Hannah Lyons","orcid":"0000-0003-0105-1064","position":1,"is_corresponding":false},{"id":994596,"name":"Jessica Walker","orcid":"0000-0003-2586-7172","position":2,"is_corresponding":false},{"id":1017015,"name":"Andreas Yiangou","orcid":"0000-0001-8905-5734","position":3,"is_corresponding":false},{"id":1017016,"name":"Olivia Grech","orcid":"0000-0001-5560-802X","position":4,"is_corresponding":false},{"id":1017017,"name":"Zerin Alimajstorovic","orcid":"0000-0001-5622-8301","position":5,"is_corresponding":false},{"id":297917,"name":"Nigel H. Greig","orcid":"0000-0002-3032-1468","position":6,"is_corresponding":false},{"id":319985,"name":"Yazhou Li","orcid":"0000-0002-6048-3660","position":7,"is_corresponding":false},{"id":1017018,"name":"Georgios Tsermoulas","orcid":"0000-0001-5658-2335","position":8,"is_corresponding":false},{"id":1017019,"name":"Kristian Brock","orcid":"0000-0002-3921-0166","position":9,"is_corresponding":false},{"id":799288,"name":"Susan P. Mollan","orcid":"0000-0002-6314-4437","position":10,"is_corresponding":false},{"id":1017020,"name":"Alexandra J. Sinclair","orcid":"0000-0003-2777-5132","position":11,"is_corresponding":false},{"id":1017013,"name":"James Mitchell","orcid":"0000-0001-6785-9352","position":0,"is_corresponding":true}],"reference_count":61,"raw_metadata":null,"created_at":"2026-07-19T01:06:25.560098Z","pmid":"36907221","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":[]}