Long-Acting Parathyroid Hormone Analog for Treatment of Hypoparathyroidism

National Center For Advancing Translational Sciences

Investigators

Linked publications & trials

Abstract

Hypoparathyroidism is a disorder in which the parathyroid glands in the neck do not make enough parathyroid hormone (PTH). PTH controls levels of calcium, phosphorus and vitamin D in the blood and in bones. Too little PTH leads to deficiencies in these vitamins and minerals, causing symptoms such as muscle twitching and seizures. Hypoparathyroidism can run in families, but a more common cause is injury to the parathyroid glands during surgery. No treatment currently exists to correct PTH deficiency; instead, patients are given large amounts of calcium and vitamin D, which can damage the kidneys. In previous clinical trials, patients were given PTH replacement therapy, but the version of PTH used was short-acting and had to be given by injection frequently. The investigators have developed a longer-lasting version of therapeutic PTH, the effects of which last 24 to 48 hours. They will continue to prepare the compound for testing in human clinical trials. The team is collaborating on the completion of the following studies -Synthesis of Good Manufacturing Practice (GMP) and non-GMP material -Formulation development -Pharmacokinetic/absorption, distribution, metabolism and excretion (PK/ADME) studies -Investigational New Drug (IND)-directed toxicology