biostudies-literatureKondo TKyoto University HospitalTime Therapeutics, Inc.e051343https://www.ebi.ac.uk/biostudies/studies/S-EPMC8246358biostudies-literatureUnknown11(6)<h4>Introduction</h4>Alzheimer's disease (AD) is one of the most common causes of dementia. Pathogenic variants in the presenilin 1 (PSEN1) gene are the most frequent cause of early-onset AD. Medications for patients with AD bearing PSEN1 mutation (PSEN1-AD) are limited to symptomatic therapies and no established radical treatments are available. Induced pluripotent stem cell (iPSC)-based drug repurposing identified bromocriptine as a therapeutic candidate for PSEN1-AD. In this study, we used an enrichment strategy with iPSCs to select the study population, and we will investigate the safety and efficacy of an orally administered dose of bromocriptine in patients with PSEN1-AD.<h4>Methods and analysis</h4>This is a multicentre, randomised, placebo-controlled trial. AD patients with PSEN1 mBMJ openRepurposing bromocriptine for Aβ metabolism in Alzheimer's disease (REBRAnD) study: randomised placebo-controlled double-blind comparative trial and open-label extension trial to investigate the safety and efficacy of bromocriptine in Alzheimer's disease with presenilin 1 (PSEN1) mutations.PMC8246358N/A0709992110Endo KNakakura ATaruno YShigenobu KMorita SMori KYasuda KTakahashi RUchikawa OKutoku YTomimoto HSunada YIzumi YWatanabe TIshii KAmino YMaki TUozumi RFujita KInoue HTada HKinoshita AIshikawa HKondo TOkunomiya TShiota SShindo ASuehiro TIkeda MKanemaru KBanno HKawakatsu SfalseRepurposing bromocriptine for Aβ metabolism in Alzheimer's disease (REBRAnD) study: randomised placebo-controlled double-blind comparative trial and open-label extension trial to investigate the safety and efficacy of bromocriptine in Alzheimer's disease with presenilin 1 (PSEN1) mutations.<h4>Introduction</h4>Alzheimer's disease (AD) is one of the most common causes of dementia. Pathogenic variants in the presenilin 1 (PSEN1) gene are the most frequent cause of early-onset AD. Medications for patients with AD bearing PSEN1 mutation (PSEN1-AD) are limited to symptomatic therapies and no established radical treatments are available. Induced pluripotent stem cell (iPSC)-based drug repurposing identified bromocriptine as a therapeutic candidate for PSEN1-AD. In this study, we used an enrichment strategy with iPSCs to select the study population, and we will investigate the safety and efficacy of an orally administered dose of bromocriptine in patients with PSEN1-AD.<h4>Methods and analysis</h4>This is a multicentre, randomised, placebo-controlled trial. AD patients with PSEN1 m2021-01-01T00:00:00Z2021 Jun2026-05-08T21:37:02.053Z2022-02-10T19:29:57.362ZS-EPMC82463583419350410.1136/bmjopen-2021-051343