Document Type
Article
Publication Date
1-5-2024
Abstract
Hypoparathyroidism (HypoPT) is a rare disease, often inadequately controlled by conventional treatment. PARALLAX was a mandatory post-marketing trial assessing pharmacokinetics and pharmacodynamics of different dosing regimens of recombinant human parathyroid hormone 1-84 (rhPTH[1-84]) for treating HypoPT. The present study (NCT03364738) was a phase 4, 1-yr open-label extension of PARALLAX. Patients received only 2 doses of rhPTH(1-84) in PARALLAX and were considered treatment-naive at the start of the current study. rhPTH(1-84) was initiated at 50 μg once daily, with doses adjusted based on albumin-corrected serum calcium levels. Albumin-corrected serum calcium (primary outcome measure), health-related quality of life (HRQoL), adverse events, and healthcare resource utilization (HCRU) were assessed. The mean age of the 22 patients included was 50.0 yr; 81.8% were women, and 90.9% were White. By the end of treatment (EOT), 95.5% of patients had albumin-corrected serum calcium values in the protocol-defined range of 1.88 mmol/L to the upper limit of normal. Serum phosphorus was within the healthy range, and albumin-corrected serum calcium-phosphorus product was below the upper healthy limit throughout, while mean 24-h urine calcium excretion decreased from baseline to EOT. Mean supplemental doses of calcium and active vitamin D were reduced from baseline to EOT (2402-855 mg/d and 0.8-0.2 μg/d, respectively). Mean serum bone turnover markers, bone-specific alkaline phosphatase, osteocalcin, procollagen type I N-terminal propeptide, and type I collagen C-telopeptide increased 2-5 fold from baseline to EOT. The HCRU, disease-related symptoms and impact on HRQoL improved numerically between baseline and EOT. Nine patients (40.9%) experienced treatment-related adverse events; no deaths were reported. Treatment with rhPTH(1-84) once daily for 1 yr improved HRQoL, maintained eucalcemia in 95% of patients, normalized serum phosphorus, and decreased urine calcium excretion. The effects observed on urine calcium and the safety profile are consistent with previous findings.
CLINICAL TRIAL IDENTIFIER: NCT03364738.
Recommended Citation
Khan, Aliya A.; Abbott, Lisa G.; Ahmed, Intekhab; Ayodele, Olulade; Gagnon, Claudia; Finkelman, Richard D.; Mezosi, Emese; Rejnmark, Lars; Takacs, Istvan; Yin, Shaoming; and Ing, Steven W., "Open-Label Extension of a Randomized Trial Investigating Safety and Efficacy of rhPTH(1-84) in Hypoparathyroidism" (2024). Division of Endocrinology, Diabetes and Metabolic Diseases Faculty Papers. Paper 10.
https://jdc.jefferson.edu/endocrinologyfp/10
Creative Commons License
This work is licensed under a Creative Commons Attribution 4.0 License.
PubMed ID
38741607
Language
English
Comments
This article is the author's final published version in JBMR Plus, Volume 8, Issue 3, March 2024, Article number 010.
The published version is available at https://doi.org/10.1093/jbmrpl/ziad010. Copyright © The Author(s) 2024. Published by Oxford University Press on behalf of the American Society for Bone and Mineral Research.