A randomised, open-label, parallel group phase 2 study of antisense oligonucleotide therapy in acromegaly
European Journal of Endocrinology May 29, 2018
Trainer PJ, et al. - Researchers performed a phase 2 randomised, open-label, parallel-group study to evaluate the potential of ATL1103 (ATL1103 is a second-generation antisense oligomer targeting the human GH receptor) as a treatment for acromegaly. For the purpose of this study, twenty-six patients with active acromegaly (IGF-I >130% upper limit of normal) were randomised to subcutaneous ATL1103 200 mg either once- or twice-weekly for 13 weeks, and monitored for a further 8-week washout period. Results revealed that ATL1103 was well tolerated, although 84.6% of patients experienced mild to moderate injection-site reactions (ISR). In patients with acromegaly, this research provided proof-of-concept that ATL1103 was able to significantly lower IGF-I.
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