Hypoparathyroidism
Hypoparathyroidism (HP) is caused by low levels of the parathyroid hormone (PTH). Most people develop this disease after thyroid surgery as a result of accidental damage/removal of the parathyroid glands. HP causes low levels of calcium and elevated levels of phosphate within the blood. There are approximately 80,000 people living with this condition in the United States alone.
Symptoms of HP include tetany, tingling, paresthesia, weakness, memory loss, headache, and impaired judgement. However, it can also cause impaired renal function when calcium depositions present themselves in the kidneys, eyes, and brain. The standard treatment for HP has long been calcium supplements and active vitamin D analogs. However, these treatments are not fully effective and may actually contribute to renal disease in some individuals. HP patients are four times as likely to develop this renal disease than the healthy population.
It is clear we need better options for this population of patients. Thankfully, it was pretty clear to researchers what type of investigation was needed. After all, HP is one of very few hormonal insufficiency conditions which is not currently treated by a therapy that replaces the missing hormone.
Ascendis Pharmaceuticals has just announced that their Investigational New Drug application for this condition has been accepted by the FDA, meaning the company can move into a clinical trial. The organization will be investigating TransCon PTH as a treatment for HP. TransCon PTH is a long-acting prodrug of parathyroid hormone. They believe that this therapy could completely replace standard supplementation of vitamin D and calcium for patients.
About the Trial
This new trial for PH is called PaTH Forward. It is a Phase 2, global investigation that will be randomized and placebo-controlled. This trial will include 40 adult patients diagnosed with HP who are currently being treated with standard of care therapies. These participants will come from up to 40 trial sites across the world. Specifically, it will evaluate the effect of TransCon PTH when administered in three doses. It will also investigate a titration regime for the removal of standard of care therapies.
TransCon PTH will be delivered to patients in a pre-filled and ready-to-use pen. Patient reported outcomes will be measured throughout the study. The therapy will be administered for four weeks, after which all participants may enroll in an open-label extension study. This extension study will help researchers evaluate the long-term efficacy and safety of this treatment.
Researchers at Ascendis believe that this therapy could restore PTH levels in PH patients for 24 hours each day. That means this treatment would eliminate typical symptoms of the condition, fully addressing all complications of PH.
The company expects results from this trial will be available by the last quarter of this year.
You can read more about this trial and how exactly TransCon technology works here.
