Study of mRNA Drug XH02 for Treating Adults with Hypoparathyroidism Evaluating Different Doses of Intravenous PTH1-84 mRNA Therapy

What this Trial Is About

This research aims to evaluate the safety and effectiveness of a new mRNA-based drug called XH02 for adults with hypoparathyroidism, a condition where the body does not produce enough parathyroid hormone PTH. The study focuses on patients with chronic hypoparathyroidism caused by surgery, autoimmune, genetic, or unknown reasons, who have difficulty managing their condition with standard treatments. This early phase 1 trial is sponsored by Peking Union Medical College Hospital to better understand this novel therapys potential. The investigational treatment involves intravenous administration of a drug called PTH1-84 mRNA at different doses ranging from 20 micrograms to 160 micrograms. Participants will receive one of several dosages of this mRNA drug, which is designed to produce PTH after being administered. The study is non-randomized and unblinded, meaning all participants know the treatment they receive. The treatment period includes administration and monitoring, followed by a 90-day follow-up to observe effects and safety. During the study, participants will undergo various assessments, including blood tests measuring PTH levels, calcium, magnesium, phosphorus, vitamin D, and bone turnover markers at multiple time points before and up to 72 hours after administration. Urine tests will also monitor calcium excretion. Safety is closely monitored throughout the entire study period, including the screening, treatment, and follow-up phases. Participants will be observed for adverse events and serious adverse events to evaluate the treatments safety profile.

Efficacy and Safety of XH02 for the Treatment of Hypoparathyroidism