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 medications 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.

This trial evaluates the safety and treatment effects of palopegteriparatide at doses above 30 mcgday in adults with hypoparathyroidism. It focuses on adults already receiving palopegteriparatide at doses of 30 mcgday or higher, aiming to provide more evidence about treatment impact and safety. The study is conducted in the United States and is a Phase 3 randomized clinical trial. Participants receive daily subcutaneous injections of palopegteriparatide, with dosing individually adjusted through either a novel or previously tested titration algorithm. All participants receive the active drug during the 78-week treatment period. The trial compares two dosing algorithms to find an optimal dose for each participant. Throughout the study, participants are monitored for efficacy primarily at 26 weeks and continue treatment for a total of 78 weeks. Assessments include laboratory tests to confirm vitamin D, magnesium, and calcium levels, as well as safety and tolerability evaluations. The trial aims to understand how well the drug works and its safety profile over this extended period.

This observational study focuses on lactating female participants who are receiving therapeutic doses of YORVIPATH palopegteriparatide as part of their usual medical care and who have chosen to breastfeed their infants. The purpose is to assess the potential transfer of palopegteriparatide into breast milk, providing important information about exposure during breastfeeding for females with hypoparathyroidism. Participants in this study are lactating females treated with YORVIPATH according to normal clinical practice. The study observes those who have been on a stable daily dose of YORVIPATH for at least 14 days prior to sample collection. This is an opportunistic study that does not involve changes to their treatment but monitors the presence of the drug in breast milk over a six-day period. During the study, participants provide breast milk samples to evaluate the transfer of palopegteriparatide. The main outcome measured is the amount of the drug present in breast milk over six days. Participants continue their usual care and dosing schedules, and researchers collect data without altering treatment. The total participation duration depends on dosing stability and sample collection timing, with no additional interventions beyond monitoring.

Researchers are studying the safety and effectiveness of canvuparatide as a once-weekly treatment for adults with hypoparathyroidism, a condition where the body produces insufficient parathyroid hormone HypoPT. This phase 3 trial compares canvuparatide to a placebo to better understand its effects in this patient population. Participants receive canvuparatide or placebo as a single-use drug-device combination product. Each injection delivers 1 mL of the drug or placebo via a manual subcutaneous injection once weekly. The study includes a randomized, parallel design where participants are assigned to either the canvuparatide or placebo group. Throughout the study, participants are monitored for 26 weeks to measure how many respond to treatment based on key criteria. Researchers will evaluate safety and efficacy during this period. The trial involves regular assessments including blood tests to monitor serum calcium and hormone levels. Participation lasts until study completion in August 2029, with primary outcomes assessed at 26 weeks.

Researchers are evaluating the effects and safety of once-daily palopegteriparatide injections in adolescents aged 12 to under 18 years who have long-term hypoparathyroidism. The study focuses on participants with clinically diagnosed hypoparathyroidism lasting at least 26 weeks and aims to understand how well this treatment works and its safety profile over a long period. Participants will receive palopegteriparatide by subcutaneous injection once daily for up to 234 weeks. During this time, researchers will monitor various health aspects including serum biochemistries, bone mineral density, renal calcifications, hospital visits, adverse events, and patient-reported symptom and impact scores. This is a single-group Phase 3 trial conducted in Europe. Participants will be involved in regular assessments throughout the study, including blood and urine tests, imaging for kidney calcifications, bone health evaluations, and symptom questionnaires. The main outcome is the percentage of participants achieving a specific efficacy endpoint at 26 weeks, with ongoing monitoring continuing through the full 234 weeks. Safety and treatment effects will be closely observed during the entire study period.

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 models 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 patients 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.