Monitoring the natural history of hypoparathyroidism

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

This study is tracking adults with hypoparathyroidism over three years to see how the condition affects their health and to find better ways to manage its complications.

Quick facts

• Study type: Observational
• Enrollment: 106 (estimated)
• Ages: 18 Years to 100 Years
• Sex: All
• Sponsor: Columbia University (Academic / other)
• Drugs / interventions: radiation
• Locations: 1 site (New York, New York)
• Trial ID: NCT05793853 on ClinicalTrials.gov

What this trial studies

This observational study aims to collect longitudinal data over three years on adults with hypoparathyroidism to understand the disease's natural history. It focuses on defining end-organ damage associated with hypoparathyroidism and the epidemiology of complications related to high calcification propensity. The study will assess the relationship between calcification burden and the severity of end-organ disease, as well as evaluate traditional and novel biomarkers for monitoring the disease. The findings will inform future strategies for managing end-organ complications in hypoparathyroidism.

Who should consider this trial

Good fit: Ideal candidates include adults aged 18 and older with any subtype of hypoparathyroidism.

Not a fit: Patients without a diagnosis of hypoparathyroidism or those under 18 years old may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to improved understanding and management of complications associated with hypoparathyroidism.

How similar studies have performed: While the approach of studying the natural history of hypoparathyroidism is less common, similar observational studies in other endocrine disorders have shown success in understanding disease progression.

Eligibility criteria

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 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.

Exclusion Criteria:

* Functional HPT
* Transient HPT
* Pseudohypoparathyroidism
* Pregnancy

Where this trial is running

New York, New York

• Columbia University Medical Center - Harkness Pavillion — New York, New York, United States (Recruiting)

Study contacts

• Principal investigator: Mishaela Rubin, MD — Columbia University
• Study coordinator: Noelle Texeira

Conditions Hypoparathyroidism