Hypoparathyroidism Natural History Study (SHINE)

December 8, 2025 updated by: Mishaela Rubin, Columbia University

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

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

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

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 Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
106

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Contact Backup

Study Locations

  • United States
    • New York
      • New York, New York, United States, 10032
        • Recruiting
        • Columbia University Medical Center - Harkness Pavillion
        • Principal Investigator:
          • Mishaela Rubin, MD
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years to 100 years (Adult, Older Adult)

Accepts Healthy Volunteers

Yes

Sampling Method

Non-Probability Sample

Study Population

Male or female ≥18 years of age with all hypoparathyroidism sub-types. Healthy male and female controls.

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Hypoparathyroidism Subjects
Patients who have the disease hypoparathyroidism, who are being followed to monitor various aspects of the disease over time. No interventions.
Control Subjects
Healthy individuals to be followed to compare to hypoparathyroidism patients

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Kidney function
Time Frame: baseline, 6, 12, 18, 24, 30, 36 Months
blood test for changes in eGFR (in mL/min/1.73m^2)
baseline, 6, 12, 18, 24, 30, 36 Months

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Kidney calcification
Time Frame: Baseline and 36 Months
Changes in kidney calcification and stones will be assessed by abdominal CT in an optional imaging sub group. Results will be assessed and reported by a clinician.
Baseline and 36 Months
Brain calcification
Time Frame: Baseline and 36 Months
Changes in brain calcification will be assessed by head CT in an optional imaging sub-group. Results will be assessed and reported by a clinician.
Baseline and 36 Months
Vascular calcification
Time Frame: Baseline and 36 Months
Changes in vascular calcification will be assessed by leg arterial calcifications on high resolution quantitative computed tomography in all, and aortic calcifications on abdominal CT and vertebral fracture assessment by DXA in optional imaging sub-group
Baseline and 36 Months
Bone mineral density
Time Frame: Baseline and 36 Months
Changes in dual energy X-ray absorptiometry will be assessed in an optional imaging sub-group
Baseline and 36 Months
Bone microarchitecture and bone strength
Time Frame: Baseline and 36 Months
Changes in high resolution peripheral quantitative computed tomography will be assessed
Baseline and 36 Months
Cardiac function
Time Frame: Baseline and 36 Months
Changes in EKG will be assessed
Baseline and 36 Months
Transcriptomic signaling for calcification
Time Frame: Baseline and 36 Months
Changes in microRNA will be assessed
Baseline and 36 Months
Biomarkers blood
Time Frame: baseline, 6, 12, 18, 24, 30, 36 Months
Changes in complete metabolic panel to see changes with eGFR (including albumin-corrected serum calcium), PTH, phosphorus, magnesium, 25(OH)D, 25(OH)D2, TSH, FT4.
baseline, 6, 12, 18, 24, 30, 36 Months
Biomarkers urine
Time Frame: baseline, 6, 12, 18, 24, 30, 36 Months
24 hour urine will be collected for changes in calcium, creatinine, total volume and protein
baseline, 6, 12, 18, 24, 30, 36 Months
Dietary Intake
Time Frame: baseline, 12, 24 and 36 Months
Food frequency questionnaires will be administered to measure changes in calcium, phosphorus, vitamin D, and sodium intake
baseline, 12, 24 and 36 Months
Cognitive Function
Time Frame: baseline, 12, 24 and 36 Months
Changes in cognitive function will be assessed by NIH Toolbox®; Letter Fluency by the Controlled Oral Word Association Test with the letters FAS; Sematic Fluency by Animal Fluency; List Learning and Memory by the Hopkins Verbal Learning Test; subjective cognitive function by FACT-Cog
baseline, 12, 24 and 36 Months
Neurologic Tests of Motor Function
Time Frame: baseline, 12, 24 and 36 Months
Repeated Chair Stand (RCS) test will be administered to see how many times a patient can sit in and stand from a chair in 30 seconds and "Timed Up and Go" Test will measure how many seconds it takes for a patient to walk to assess changes in motor function
baseline, 12, 24 and 36 Months
Quality of Life Through Self-Reported Questionnaires
Time Frame: baseline, 12, 24 and 36 Months
Quality of life will be assessed by SF-36, FACIT-IF (self-reported fatigue), PGI-S and PGI-I (patient global impression of severity and impact), Hospital Anxiety and Depression Scale (HADS) and the HPT Symptom Diary and changes will be tracked through visits
baseline, 12, 24 and 36 Months
Calcioprotein Maturation Time
Time Frame: Baseline and 36 months
Will be obtained through blood collection and measured in minutes and changes will be tracked through visits
Baseline and 36 months
Sclerostin
Time Frame: Baseline and 36 months
Will be obtained through blood collection and measured in pmol/L and changes will be tracked through visits
Baseline and 36 months
FGF23
Time Frame: Baseline and 36 months
Will be obtained through blood collection and measured in pg/mL and changes will be tracked through visits
Baseline and 36 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Columbia University

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
FDA Office of Orphan Products Development

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Mishaela Rubin, MD, Columbia University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
August 25, 2022

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 1, 2026

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 1, 2026

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
December 23, 2022

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 20, 2023

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
March 31, 2023

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
December 16, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 8, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
December 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • AAAU3302
  • R01FD007629 (U.S. FDA Grant/Contract)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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