A multicenter, single-arm, phase II clinical trial of adrenomedullin in patients with cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy

• DOI: 10.1016/j.cccb.2024.100211
• Publication Date: 2024-02-06T01:36:27Z
• Authors: Kazuo Washida; Satoshi Saito; Tomotaka Tanaka; Yuriko Nakaoku; Hiroyuki Ishiyama; Soichiro Abe; Takehito Kuroda; Shinsaku Nakazawa; Chikage Kakuta; Katsuhiro Omae; Kenta Tanaka; Manabu Minami; Yoshiaki Morita; Tetsuya Fukuda; Akihiro Shindo; Takakuni Maki; Kazuo Kitamura; Hidekazu Tomimoto; Toshihiko Aso; Masafumi Ihara
• Keywords: CADASIL; Adrenomedullin; Clinical endpoint

Cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy (CADASIL), the most common form of hereditary cerebral small vessel disease (SVD), currently lacks disease-modifying treatments. Adrenomedullin (AM), a vasoactive peptide angiogenic, vasodilatory, anti-inflammatory, anti-oxidative properties, shows potential effects on neuro-glial-vascular unit. The AdrenoMedullin for CADASIL (AMCAD) study aims to assess efficacy safety AM in patients CADASIL. Overall, 60 will be recruited. AMCAD is multicenter, investigator-initiated, single-arm phase II trial. Patients confirmed diagnosis, based NOTCH3 genetic testing, receive an 8-h treatment (15 ng/kg/min) 14 days following baseline assessment (from day 1 14). Follow-up evaluations performed 15, 28, 90, 180. primary endpoint blood flow change rate frontal cortex, evaluated using arterial spin labeling magnetic resonance imaging, from 28. Summary statistics, 95% confidence intervals, one-sample t-test used analysis. represent therapeutic CADASIL, addressing unmet medical need this challenging condition. jRCT 2,051,210,117 (https://jrct.niph.go.jp/en-latest-detail/jRCT2051210117).