A new study by Clarke and colleagues published in the Journal of Clinical Endocrinology & Metabolism highlights the safety and efficacy of TransCOn PTH (palopegteriparatide) to treat adults with hypoparathyroidism.
Hypoparathyroidism
Hypoparathyroidism is a rare endocrine disorder in which the parathyroid glands in the neck do not produce enough parathyroid hormone (PTH). Common signs and symptoms include abdominal pain, brittle nails, cataracts, dry hair and skin, muscle cramps, tetany, pain in the face, legs, and feet, seizures, tingling sensation, and weakened tooth enamel. It may be caused by injury to the parathyroid glands such as during surgery.
The PaTHway Trial
This PaTHway trial was a phase 3, 26-week, double-blind, placebo-controlled clinical trial followed by an open-label extension. The objective of this study was to assess the long-term safety and efficacy of palopegteriparatide in patients with hypoparathyroidism. Palopegteriparatide is a parathyroid hormone analog. A total of 78 patients completed week 52 of this study (26 weeks in the double-blind, placebo controlled phase plus 26 weeks in the open-label phase).
Results
The main efficacy endpoints were proportion of participants who achieved normal serum calcium levels and independence from conventional therapy by week 52. Other endpoints included patient-reported outcomes and bone mineral density. Safety was assessed by 24-hour urine calcium and treatment-emergent adverse events.
At week 52, 81% of participants met the main efficacy endpoints. 95% achieved independence from conventional therapy, with non requiring vitamin D. Patient-reported outcomes illustrated sustained improvements in quality of life, physical functioning, and well-being. Mean bone mineral density Z-scores decreased toward age- and sex-matched norms from baseline. Additionally, mean 24-hour urine calcium excretion decreased from 376 mg/day at baseline to 195 mg/day at week 52.
In regards to safety, most treatment-emergent adverse events were mild to moderate. No adverse events led to discontinuation during the open-label expansion.
To learn more about hypoparathyroidism and other rare endocrine disorders, visit https://checkrare.com/diseases/endocrine-disorders/
References:
Clarke BL, et al. Efficacy and Safety of TransCon PTH in Adults With Hypoparathyroidism: 52-Week Results From the Phase 3 PaTHway Trial. J Clin Endocrinol Metab. 2025 [In press]. https://doi.org/10.1210/clinem/dgae693