Menu
Recruiting NCT05793853

Hypoparathyroidism Natural History Study

— Observational Hypoparathyroidism

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
This is an observational study: the protocol does not assign a study treatment.
Who it may be relevant to
Registry conditions: Hypoparathyroidism. Basic parameters: 18 years — 100 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

Overview

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

Detailed 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 investigations on the development, study, and implementation of HPT end-organ disease modifying strategies and impact clinical practice in hypoparathyroidism.

Primary outcome measures

  • Kidney function [Time frame: baseline, 6, 12, 18, 24, 30, 36 Months]
Secondary outcome measures (12)
  • Kidney calcification [Time frame: Baseline and 36 Months]
  • Brain calcification [Time frame: Baseline and 36 Months]
  • Vascular calcification [Time frame: Baseline and 36 Months]
  • Bone mineral density [Time frame: Baseline and 36 Months]
  • Bone microarchitecture and bone strength [Time frame: Baseline and 36 Months]
  • Cardiac function [Time frame: Baseline and 36 Months]
  • Transcriptomic signaling for calcification [Time frame: Baseline and 36 Months]
  • Biomarkers blood [Time frame: baseline, 6, 12, 18, 24, 30, 36 Months]
  • Biomarkers urine [Time frame: baseline, 6, 12, 18, 24, 30, 36 Months]
  • Dietary Intake [Time frame: baseline, 12, 24 and 36 Months]
  • Cognitive Function [Time frame: baseline, 12, 24 and 36 Months]
  • Neurologic Tests of Motor Function [Time frame: baseline, 12, 24 and 36 Months]

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: Yes

Study design

Observational model
Cohort

Study locations

United States · 1 center
  • Columbia University Medical Center - Harkness Pavillion — New York

Identifiers

NCT: NCT05793853 · AAAU3302 · R01FD007629

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗