Идёт набор NCT05793853

Hypoparathyroidism Natural History Study

— Наблюдательное Hypoparathyroidism

Ориентир для пациента и семьи

Простыми словами

Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.

Что изучают: Это наблюдательное исследование: исследуемое лечение участникам по протоколу не назначают.
Кому может быть актуально: Состояния в реестре: Hypoparathyroidism. Базовые параметры: 18 лет — 100 лет · Все.
Что важно проверить: Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
Где проводится: США
Следующий шаг: Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →

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Официальное название

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

Обзор

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

Подробное описание

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.

Первичные конечные точки

Kidney function [Срок оценки: baseline, 6, 12, 18, 24, 30, 36 Months]

Вторичные конечные точки (12)

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

Критерии участия

Критерии включения

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.