Investigational Drug Restores Parathyroid Function In Rare Disease

18 Oct 2023 • An investigational drug, encaleret, restored calcium levels in people with autosomal dominant hypocalcemia type 1 (ADH1), - a rare genetic disorder marked by an imbalance of calcium in the blood and urine, as well as abnormally low levels of parathyroid hormone, which regulates blood calcium levels. Results from the clinical trial are published in the New England Journal of Medicine.

In the mid-phase clinical trial, 13 participants with ADH1 received oral doses of the investigational drug for about 24 weeks. By the end of the trial, the treatment restored every participant’s blood calcium level to normal, and urine calcium approached normal levels. Levels of parathyroid hormone also normalized.

Encaleret appeared to be safe and caused no serious side effects. However, because parathyroid hormone raises blood calcium levels in part by drawing calcium from the bones, the researchers noted a need to assess the treatment’s long-term effects on the skeleton. The researchers also speculate encaleret may have broader implications. An ongoing clinical trial s testing whether the treatment can help correct calcium levels in people whose parathyroid glands have been damaged by surgery.

Source: NIH | Read full story

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