Advancing Product Development for Hypoparathyroidism: A Prospective Natural History Study of the Clinical Outcomes and Regulation of Disordered Mineral Metabolism
Columbia University
Summary
This is a prospective three-year natural history study of adults with hypoparathyroidism. The goal is to monitor patients with hypoparathyroidism to define end-organ damage in the context of the disease. The study objectives are to: 1. Build a prospective cohort of patients to study HPT-associated end-organ damage. 2. Determine end-organ physiologic consequences of HPT. 3. Elucidate determinants of HPT-associated end-organ damage. Funding Source - FDA OOPD
Description
The goal of this study is to prospectively collect data on the natural history of hypoparathyroidism (HPT). This will enable longitudinal data collection of complications in this disease, specifically defining the epidemiology of end-organ complications of HPT that are related to high calcification propensity. It will also determine relationships between calcification burden and end-organ disease severity and progression risk and assess the utility of traditional and novel biomarkers of mineral and bone metabolism on disease diagnosis and monitoring. These data will inform future investigation…
Eligibility
- Age range
- 18–100 years
- Sex
- All
- Healthy volunteers
- Yes
Inclusion Criteria: * An understanding, ability and willingness to fully comply with study procedures and restrictions. * Ability to voluntarily provide written, signed and dated informed consent as applicable to participate in the study. * Male or female ≥18 years of age with HPT. All HPT sub-types are eligible, including surgical (HPT-S) and nonsurgical (HPT-NS) HPT: autoimmune, genetic (including but not limited to: DiGeorge syndrome, autoimmune polyendocrine syndrome type 1, hypoparathyroidism sensorineural deafness and renal disease syndrome, Kearns-Sayre syndrome, mitochondrial encephal…
Location
- Columbia University Medical Center - Harkness PavillionNew York, New York