Acromegaly

Researchers are evaluating Debio 4126, a 12-week extended-release formulation of octreotide, in adults with acromegaly who have previously been treated with somatostatin analogs. The study aims to assess how well Debio 4126 maintains insulin-like growth factor 1 (IGF-1) levels at or below the upper limit of normal compared to a placebo during a double-blind period lasting 36 weeks. Participants will be randomly assigned to receive either Debio 4126 or a placebo by intramuscular injection once every 12 weeks for a total of three injections during the double-blind period. Those who meet specific IGF-1 criteria at week 34 may continue into an open-label period receiving Debio 4126 injections every 12 weeks for an additional 24 to 60 weeks. Rescue medication is available for participants whose acromegaly is not well controlled during the study. Throughout the study, participants will undergo regular assessments of IGF-1 and growth hormone levels, safety monitoring, and evaluations of local tolerability at the injection site. The main outcome is the percentage of participants maintaining IGF-1 levels at or below the upper limit of normal after 36 weeks. The study may last up to approximately 108 weeks for some participants, including follow-up and treatment periods.

Researchers are studying pituitary tumors, which are a varied group of tumors affecting the pituitary gland. This observational study focuses on collecting detailed clinical, biochemical, morphological, and pathological data from over 1600 patients who have undergone neurosurgical removal of pituitary tumors in the past 20 years. The goal is to develop a prognostic classification to better predict tumor behavior and outcomes, as current data on epidemiology and prognosis are inconclusive. The study involves patients who have had surgery for pituitary tumors, with data collected retrospectively and prospectively. The main focus is on monitoring the frequency of disease recurrence. Participants have been followed for at least two years after their tumor removal, allowing researchers to track tumor recurrence over time. Participants will be observed from enrollment through 24 months of follow-up to assess tumor recurrence. Researchers will gather information from medical records and follow-up visits to evaluate outcomes. This study does not involve any experimental treatment, but rather monitors surgical outcomes and tumor behavior to improve future understanding of pituitary tumors.

Researchers are evaluating the effects of a 15-week treatment with ALXN2420 compared to a placebo in adults with acromegaly, a condition caused by excess growth hormone. The study aims to see if ALXN2420, given alongside somatostatin analog therapy, can reduce levels of insulin-like growth factor 1 (IGF-1), which is linked to acromegaly symptoms. This is a Phase 2 randomized, double-blind trial sponsored by Alexion Pharmaceuticals, Inc. Participants will receive either ALXN2420 or a placebo through daily subcutaneous injections for 15 weeks during the Primary Evaluation Period. After this, an Open-label Extension period allows those initially on placebo to switch to ALXN2420, with dosing adjusted based on IGF-1 levels at Week 13. Follow-up assessments occur at Weeks 19, 26, 32, 39, 45, and 52 to monitor treatment effects. During the study, participants will undergo regular evaluations of their IGF-1 blood levels and complete symptom and quality of life questionnaires. Researchers will track changes from baseline in IGF-1, symptom severity, health surveys, and adverse events throughout the trial. Safety and the body’s response to ALXN2420 will also be monitored over the course of the study, which lasts up to 52 weeks including the extension.

Researchers are investigating tumors affecting the hypothalamic-pituitary unit in children and adults aged 2 to 70 years. The study aims to identify genetic factors involved in the development of these pituitary tumors and related disorders, as well as to develop new clinical studies for their recognition and treatment. It also explores the psychological effects of cortisol secretion in children with Cushing disease and seeks to understand inheritance patterns and associations with other family conditions. Participants undergo procedures including tissue specimen collection and MRI scans to study the molecular genetics of tumors and related developmental pathways. This observational protocol serves as a screening and training platform for medical fellows and students and supports the development of future clinical trials focused on diagnosis and therapy of pituitary tumors. During the study, participants provide peripheral blood and tumor tissue samples for molecular genetic testing. Researchers assess cognitive, psychological, and patient-reported health outcomes related to hypercortisolemia and adrenal insufficiency. The study also monitors clinical and genetic features of pituitary tumors and collects data on mental and social well-being. Participation involves ongoing assessments and follow-up to support future research and clinical care improvements.

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.

This research aims to identify the key factors that affect bone strength and predict the risk of fragility fractures in patients with acromegaly. It is a prospective observational clinical, molecular, and translational study designed to improve understanding of bone health in people with excess growth hormone. The main focus is on morphometric vertebral fractures in these patients over an 18-month period. Participants will undergo various assessments including blood sample collection for molecular and biochemical tests, several radiological tests such as DXA, vertebral fracture assessment, trabecular bone score, bone strength index, and high-resolution peripheral quantitative computed tomography, as well as DXA morphometry. They will also complete health-related quality of life questionnaires such as AcroQol and SF-36. These tests and questionnaires help evaluate bone strength and overall health status. During the study, participants will have outpatient visits for these procedures and assessments. The main outcome being measured is the rate of vertebral fractures over 18 months. Researchers will monitor bone health and fracture risk factors without administering any treatments. The study is sponsored by IRCCS San Raffaele and is expected to end in April 2026.

Researchers are studying patients with pituitary complex and rare diseases to better understand their clinical features and outcomes. This observational study at Peking Union Medical College Hospital aims to identify factors influencing remission rates, associated comorbidities, perioperative events, radiological findings, and pathological characteristics. By comparing different patient groups, the study seeks to improve knowledge about managing and predicting the course of these conditions. Participants include patients diagnosed with various pituitary disorders such as pituitary adenomas, craniopharyngiomas, Rathke's cleft cysts, sellar region germ cell tumors, and cavernous sinus syndrome. Those needing surgical removal or biopsy of pituitary pathologies will undergo transsphenoidal surgery or craniotomy. Treatments studied involve surgical removal and biopsy procedures to collect tissue for examination and diagnosis. During the study, participants will have detailed clinical evaluations, provide comprehensive medical histories including symptoms, treatments, and outcomes, and take part in follow-up assessments to monitor disease progression. Researchers will track remission starting one week after surgery and continuing up to three years, along with comorbidities and postoperative complications over the same period. The study collects imaging and pathological data to enhance understanding of these rare diseases.

Researchers are conducting an observational, longitudinal study focused on patients with pituitary disorders who are followed at the Unit of Endocrinology of Azienda Ospedaliero-Universitaria of Modena. The study is designed to collect detailed clinical and epidemiological data to better understand this patient population over time. It includes both a retrospective phase and a prospective phase to capture comprehensive patient information. The study has two phases: the first phase involves retrospectively registering all patients with pituitary disorders treated at the center. The second phase prospectively enrolls patients attending the Unit of Endocrinology to gather ongoing data. Patients' personal information, details about their pituitary disease, symptoms, physical exams, imaging results, visual field data, and treatments including surgeries, hormone tests, and therapies will be collected and stored in an anonymized database. Participants will be involved through data collection from their medical records and ongoing clinical visits. Researchers will assess various clinical measures such as radiological imaging, hormone levels, and therapy management. The main outcome is to characterize pituitary patients over a 10-year period, along with studying their management. The study is expected to continue monitoring patients throughout this time frame to gain insights into pituitary disease progression and treatment.

Researchers are studying acromegaly, a slow-progressing disease caused by excess growth hormone, which leads to characteristic changes in facial features, hands, and feet, along with various health complications. The study aims to develop a simple computerized screening system using 3D stereophotography to help detect acromegaly earlier, especially in Chinese patients, as current hormone tests are often expensive and unavailable in many hospitals. The study compares 3D stereophotography images and X-rays of the face, hands, and feet from acromegaly patients and healthy volunteers. By analyzing these differences, researchers hope to create a computer model that can accurately recognize acromegaly features for early screening. Participants include adults aged 20 to 80 years, both acromegaly patients and healthy individuals. Participants will have their body measurements and 3D stereophotography images taken and tracked over one year from the first imaging session. The main outcome measured is anthropometry within this one-year period. The study involves observational assessments without treatment interventions, focusing on non-invasive imaging and comparison to support the development of the recognition system. The research is sponsored by National Taiwan University Hospital and includes healthy volunteers.

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.