Advancing Product Development for Hypoparathyroidism: A Prospective Natural History Study of the Clinical Outcomes and Regulation of Disordered Mineral Metabolism
Trial Snapshot
- Phase
- Not Applicable
- Status
- Recruiting
- Sponsor
- Columbia University
- Enrollment
- 106
- Locations
- 1
- Primary Endpoint
- Kidney function
Study Overview
Brief 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:
- Build a prospective cohort of patients to study HPT-associated end-organ damage.
- Determine end-organ physiologic consequences of HPT.
- 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.
Study Design
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Eligibility Criteria
- Ages
- 18 Years to 100 Years (Adult, Older Adult)
- Sex
- All
- Accepts 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 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
Outcomes
Primary Outcomes
Kidney function
Time Frame: baseline, 6, 12, 18, 24, 30, 36 Months
blood test for changes in eGFR (in mL/min/1.73m\^2)
Secondary Outcomes
- Sclerostin(Baseline and 36 months)
- Kidney calcification(Baseline and 36 Months)
- Vascular calcification(Baseline and 36 Months)
- Bone microarchitecture and bone strength(Baseline and 36 Months)
- FGF23(Baseline and 36 months)
- Brain calcification(Baseline and 36 Months)
- Biomarkers urine(baseline, 6, 12, 18, 24, 30, 36 Months)
- Cognitive Function(baseline, 12, 24 and 36 Months)
- Bone mineral density(Baseline and 36 Months)
- Cardiac function(Baseline and 36 Months)
- Transcriptomic signaling for calcification(Baseline and 36 Months)
- Dietary Intake(baseline, 12, 24 and 36 Months)
- Quality of Life Through Self-Reported Questionnaires(baseline, 12, 24 and 36 Months)
- Calcioprotein Maturation Time(Baseline and 36 months)
- Biomarkers blood(baseline, 6, 12, 18, 24, 30, 36 Months)
- Neurologic Tests of Motor Function(baseline, 12, 24 and 36 Months)
Investigators
Mishaela Rubin
Professor of Medicine
Columbia University