The study, at Columbia University Irving Medical Center, will provide new insights into the disease’s long-term complications, including associated comorbidities and the effects on quality of life.
NEW YORK, May 23, 2023 (GLOBE NEWSWIRE) -- The Hypoparathyroidism Association today announced patient enrollment in a first-of-its-kind natural history study of hypoparathyroidism, designed to gain insight into the long-term complications of a rare disorder that affects an estimated 77,000 – 115,000 patients in the U.S. The study will be led by Mishaela Rubin, M.D., Associate Professor of Medicine at Columbia University Vagelos College of Physician and Surgeons, and is sponsored by the U.S. Food & Drug Administration’s Office of Orphan Products Development (OOPD).
Hypoparathyroidism is a rare endocrine disease in which insufficient levels of parathyroid hormone (PTH) lead to lower-than-normal levels of calcium and elevated phosphate levels in the blood. It most commonly results from damage to or removal of the parathyroid glands during neck surgery but may also be inherited and/or associated with other disorders. Common short-term complications of hypoparathyroidism include muscle cramps and cognitive dysfunction, with less common complications including seizures, arrhythmias, and wheezing. As a result of these symptoms, patients living with hypoparathyroidism often have a decreased quality of life.
While near-term complications are well known, no prospective studies have been done to evaluate long-term complications of the disease, such as kidney stones, chronic kidney disease, brain calcification, vascular disease, and bone fragility, or to determine the effects of standard therapy. Retrospective studies have suggested that standard approaches to managing hypoparathyroidism with high-dose calcium and active vitamin D may contribute to some complications.
"We lack knowledge about the risk of long-term calcification in hypoparathyroidism," said Dr. Rubin. "Understanding how frequent it is, who is likely to develop it, and how likely it is to progress can help us determine the best treatment approach with the goal of preventing it from occurring."
The natural history study will inform understanding of the longer-term effects of hypoparathyroidism and provide insight into the impacts of current disease management practices. The hope is that the data from the study will over time help to optimize disease management, with the goal of minimizing the impact of the disease on patients’ quality of life and long-term health.
The study is expected to enroll 94 adult patients aged 18 years and older on all types of hypoparathyroidism treatment, including conventional management or PTH treatment. Patients will be evaluated at Columbia University once a year for three years. Annual visits will include a physical exam, blood tests, evaluation of novel markers that may predict calcification, urine tests, EKG, cognitive assessments, imaging studies, and motor function tests.
The study will:
- Monitor participants for the development of kidney, brain, vascular, and bone complications.
- Investigate new predictors in blood and urine, including genetic regulators, for the development of complications.
- Investigate if long-term complications are decreased in participants who are using PTH.
“This study is particularly important to the hypoparathyroidism community as it underscores the needed paradigm shift surrounding treatment and understanding of the condition,” said Bari Vapnek, Director of Marketing and Public Relations for the HypoPARAthyroidism Association, Inc. “With new treatment options on the horizon, understanding the long-term complications of hypoparathyroidism can help both physicians and patients make informed decisions about their care pathways, including whether conventional treatment of calcium and vitamin D is appropriate for them. There is no ‘one size fits all approach’ in my opinion, and we are grateful to Dr. Rubin for her dedication to the hypoparathyroidism community.”
To learn more about the study, visit ClinicalTrials.gov using Study Identifier: NCT05793853 or contact Joana Gjeci at jg4032@cumc.columbia.edu.
About The Hypoparathyroidism Association
HypoPARAthyroidism Association, Inc., is dedicated to improving the lives of people impacted by hypoparathyroidism (HypoPARA) through education, support, research, and advocacy. We strive to be a reliable, dedicated, accessible, inclusive, empowering resource while being compassionate and professional.
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