Hypoparathyroidism

Researchers are evaluating the safety and effectiveness of XH-02, an mRNA nucleic acid drug that produces parathyroid hormone (PTH) in the body, to treat adults with hypoparathyroidism. This condition involves low PTH levels leading to low calcium in the blood. Previous studies have shown that subcutaneous injection of XH-02 is safe and effective, and this expanded phase 2 study aims to confirm these findings in a larger group of patients. Participants will receive different doses of XH-02 by subcutaneous injection. Some will get a single dose ranging from 40 to 160 micrograms, while others will receive multiple doses of 40 to 160 micrograms daily or every other day for a total of five doses. The study includes several groups receiving these different dosing regimens to assess safety and treatment response. Throughout the study, participants will undergo various blood and urine tests to measure PTH levels, calcium, phosphorus, magnesium, vitamin D, and other markers. Safety is monitored by recording adverse events from the first dose up to 30 days for non-serious events and 3 months for serious events. The study will follow participants for these assessments to evaluate both safety and how well the drug works over time.

Researchers are collecting data in women who have been exposed to palopegteriparatide during pregnancy to understand the risks to the mother, fetus, and infant. This observational registry study aims to assess pregnancy outcomes, maternal complications, and any adverse effects on the developing fetus, newborn, and infant up to at least one year of age. The study focuses on women with hypoparathyroidism who have been treated with this medication around conception or during pregnancy. Participants include pregnant women who have taken at least one dose of YORVIPATH (palopegteriparatide) within 15 days before conception or during pregnancy. The medication is prescribed according to normal clinical practice. The study gathers data from these women without altering their treatment and includes only those who provide informed consent or assent with parental consent as applicable. During the study, researchers will collect information on the number of fetuses, pregnancy outcomes, congenital malformations, adverse events, hospitalizations, growth and development milestones, signs of calcium imbalance, infant developmental issues, mortality, and maternal complications. Data will be tracked up to 21 months after exposure to understand both short- and longer-term effects. Participants will provide medical information through healthcare providers, and ongoing monitoring will support safety assessments throughout this period.

Researchers are evaluating the safety, tolerability, and effectiveness of EXT608 in adults with hypoparathyroidism, a condition characterized by low parathyroid hormone levels affecting calcium balance. This Phase 2, multicenter, randomized, double-blind, placebo-controlled trial aims to better understand how this modified parathyroid hormone attached to vitamin D may work in this population. The study is sponsored by Extend Biosciences Inc. and focuses on adults aged 18 to 65 years who have had hypoparathyroidism for at least 12 months. Participants will receive either EXT608 or a placebo via subcutaneous injection once a week. The trial includes a multiple ascending dose phase, starting with a 50 microgram dose for 4 weeks, followed by individualized dosing for another 8 weeks. The placebo group will receive fixed doses for 4 weeks, then variable doses for 8 weeks. This dosing schedule allows researchers to assess the medication's effects at different dose levels and monitor participants closely. During the 12-week treatment period, participants will be monitored for safety and tolerability through tracking of adverse events and laboratory tests. Researchers will also assess pharmacokinetics, serum calcium levels, and calcium supplementation needs. Participants will be asked to comply with study procedures, including using a smartphone diary to record medication doses and symptoms. The trial includes detailed evaluation of laboratory results and clinical outcomes to better understand the impact of EXT608 for hypoparathyroidism.

Researchers are evaluating the safety, tolerability, and effectiveness of palopegteriparatide at doses greater than 30 mcg per day in adults with hypoparathyroidism. This phase 3 trial focuses on adults who have been living with chronic hypoparathyroidism for at least 26 weeks and are already receiving treatment with palopegteriparatide at doses of 30 mcg/day or higher. The study aims to provide more evidence on the treatment effects and safety of higher doses of this medication. Participants will receive subcutaneous injections of palopegteriparatide once daily, with doses starting at or above 30 mcg. They will be randomly assigned to one of two groups using different dose titration algorithms to find an optimal dose. Both groups receive the same medication delivered by a prefilled pen designed for single-patient use. The treatment period lasts for 78 weeks, during which the dose may be progressively adjusted based on individual needs. Throughout the study, participants will have regular visits to monitor safety and treatment effects, including laboratory tests to check calcium levels and other relevant biomarkers. The primary outcome focuses on treatment efficacy measured after 26 weeks. The trial includes ongoing monitoring of laboratory parameters and safety assessments, with participation lasting up to 78 weeks in total. This helps researchers better understand how the medication works at higher doses in adults with hypoparathyroidism.

Researchers are conducting an observational study to understand how much palopegteriparatide passes into breast milk in lactating women who are being treated with YORVIPATH for hypoparathyroidism. The study focuses on women who have chosen to breastfeed while receiving this medication as part of their usual care. It aims to provide important information about the medication's transfer during breastfeeding. Participants in this study are lactating females currently taking palopegteriparatide under normal clinical care. The study observes these women without altering their treatment, tracking the medication levels in breast milk. The main study period lasts 6 days during which breast milk samples will be collected to evaluate the transfer of the drug. During participation, women will provide breast milk samples over the 6-day observation period. Researchers will assess the amount of palopegteriparatide in the milk to understand potential exposure to infants. Participants must have stable doses of the medication and primarily breastfeed their infants. The study includes informed consent and follows the participants' usual medical care throughout.

This research aims to validate the B-COMPASS, a computational model developed by the BEAMER project to improve patient adherence to treatment across various diseases. Adherence to treatment is a widespread issue leading to increased healthcare use and premature death, with about half of medications not taken as prescribed. The study evaluates the model's ability to predict adherence, identify patient support needs, and enhance healthcare engagement in six therapeutic areas, including cardiovascular, endocrinology, immunology, neurology, oncology, and rare diseases. Participants complete the BEAMER questionnaire, which the B-COMPASS uses to group patients based on their adherence needs and predict their adherence. Patients are randomized into a control group receiving standard care or an intervention group receiving enhanced engagement through educational materials for healthcare providers tailored to the patient's B-COMPASS profile. Engagement occurs in person or by phone, depending on patient schedules, and healthcare providers may also be randomized to limit knowledge of the B-COMPASS in control groups. Participants are involved in two main data collections spaced from 2 weeks to 6 months apart, during which adherence measures, patient support needs, and perceptions of engagement are assessed. The study measures the accuracy, validity, and reliability of B-COMPASS predictions, its impact on adherence, patient and provider perceptions, and cost-effectiveness. The total participation timeline varies by site and disease area, with continuous monitoring of healthcare utilization and patient experience.

Researchers are studying enamel defects that include hypomineralization and hypoplasia, focusing on three common dental conditions: Molar Incisor Hypomineralization (MIH), dental fluorosis, and amelogenesis imperfecta. The study aims to evaluate the use of a spectrophotometer, the Zfx SpectroShade®, to measure tooth color as a non-invasive tool for early diagnosis of these enamel abnormalities. Additionally, biological, structural, and physicochemical characteristics of these enamel pathologies will be analyzed from extracted teeth or enamel biopsies. The study involves two main visits. At the first visit, participants undergo a screening consultation where photographs and spectrophotometer images of at least two teeth are taken. At a follow-up visit within two months, patients requiring dental treatment will have selected teeth extracted or restored, allowing collection of samples for detailed laboratory analysis. These analyses include protein studies, electron microscopy, and various spectroscopic and elemental techniques performed at specialized laboratories. Participants will be monitored with careful documentation and follow-up, including consent and non-opposition forms, especially for minors. Researchers will analyze color parameters of affected teeth using the spectrophotometer and compare these across different enamel disorders. The study also collects extensive data on the biological and physical makeup of enamel abnormalities to support accurate diagnosis and characterization. Samples will be stored securely for up to ten years for possible future research. The total participation involves initial imaging and a follow-up dental procedure within two months.

Post-operative hypoparathyroidism (PH) is a challenging complication following total thyroidectomy, caused by accidental removal or damage to the parathyroid glands. Researchers are evaluating whether the PTeye™ device, which uses near-infrared autofluorescence (NIRAF) technology, can help reduce PH by better identifying these glands during surgery. This prospective, single-blinded, randomized controlled trial enrolls patients undergoing total thyroidectomy at a tertiary endocrine surgery unit in Hong Kong. Participants will be randomly assigned to one of two groups: one receiving conventional total thyroidectomy and the other undergoing surgery assisted by the PTeye™ parathyroid detection system. PTeye™ uses a probe that emits laser and detects near-infrared signals in real time to identify parathyroid glands early during the operation, aiming to avoid their inadvertent removal or devascularization. During the study, participants will receive medical care as usual. Researchers will measure the rate of significant post-operative hypoparathyroidism six months after surgery, along with secondary outcomes such as rates of unintended parathyroid removal, parathyroid auto-transplantation, and parathyroid hormone levels on the day of surgery, the following day, and at three and six months post-operation. The trial starts in September 2025 and continues until December 2028.

Researchers are collecting data to better understand thyroid, parathyroid, and adrenal diseases. This observational study involves patients with diagnosed or suspected endocrine diseases and aims to gather information from medical records and follow-ups. The data will help learn more about the participant's or their child's illness, treatment, recovery, and medical history over a long period. Participants' medical information will be recorded into a research database. Starting about 2 to 3 years after testing, diagnosis, or treatment, and continuing for up to 15 years after surgery, the research team will follow up by phone to ask about their current health and medical history. If participants have routine clinic visits near their follow-up time, the research team may meet them in person or send questionnaires by mail if needed. Participants or their children will be contacted every 2 to 3 years for phone interviews lasting about 20 to 30 minutes. The study will track their health status and medical history during these calls. The study aims to compile a comprehensive database over 15 years. Up to 15,000 patients will be enrolled, and participation involves data collection and periodic follow-up without altering usual care.

Researchers are studying pheochromocytomas and paragangliomas, rare tumors that often develop in the adrenal glands and release chemicals called catecholamines. These tumors can cause high blood pressure and serious health risks such as stroke, heart attack, and sudden death, especially during surgery or childbirth. The study aims to improve diagnosis, localization, and treatment of these tumors using advanced molecular, genetic, and imaging techniques. The study evaluates various diagnostic methods including blood and urine tests measuring catecholamines and their metabolites, standard imaging like CT, MRI, and specialized PET/CT scans with radioactive compounds such as 18F-DOPA and 18F-6F-DA. Participants with detected tumors may undergo surgery if feasible. If surgery is not an option, ongoing evaluations and medical treatments will be provided. Genetic testing is also performed to analyze DNA and explore tumor predispositions. Participants undergo comprehensive assessments including medical history, physical exams, electrocardiograms, and multiple imaging scans lasting up to two hours. Blood and urine samples are collected for biochemical and genetic analyses. Researchers monitor tumor characteristics, biochemical profiles, and treatment responses over time. The study also includes follow-up visits for ongoing evaluation, education of healthcare providers and patients, and may continue for many years.