RecruitingNCT05793853

Hypoparathyroidism Natural History Study

Advancing Product Development for Hypoparathyroidism: A Prospective Natural History Study of the Clinical Outcomes and Regulation of Disordered Mineral Metabolism

Sponsor

Columbia University

Enrollment

106 participants

Start Date

Aug 25, 2022

Study Type

OBSERVATIONAL

Conditions

Hypoparathyroidism

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

Eligibility

Min Age: 18 YearsMax Age: 100 Years

Inclusion Criteria5

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 encephalomyopathy with lactic acidosis and stroke-like episodes \[MELAS\] syndrome, mitochondrial trifunctional protein \[MTP\] deficiency syndrome, Kenny-Caffey syndrome, Sanjad-Sakati syndrome, autosomal dominant hypocalcemia), infiltrative (granulomatous), mineral deposition (copper, iron), metastatic, radiation and idiopathic HPT.
Diagnosis of HPT established based on historic hypocalcemia in the setting of inappropriately low serum PTH levels on two occasions.
All treatment regimens are permitted, including but not limited to conventional management with calcium (e.g. calcium citrate, calcium carbonate, etc), active vitamin D (calcitriol, alfacalcidol), parent vitamin D, magnesium, phosphate binders and thiazides. Use of PTH-like drugs are permitted.