Acromegaly

Researchers are studying the effect of Debio 4126, a 12-week extended-release octreotide injection, in maintaining insulin-like growth factor 1 IGF-1 levels at or below the upper limit of normal in patients with acromegaly who have previously been treated with somatostatin analogs. This Phase 3 randomized trial compares Debio 4126 to a placebo during a double-blind period to evaluate its efficacy and safety. Participants in the double-blind period receive intramuscular injections of either Debio 4126 or placebo every 12 weeks for 36 weeks, totaling three injections. Those whose IGF-1 levels remain at or below the upper limit of normal at Week 34 may enter an open-label phase receiving Debio 4126 injections every 12 weeks for an additional 24 to 60 weeks. Rescue medication is allowed for participants whose acromegaly is not well controlled during specified times. Throughout the study, participants undergo evaluations of IGF-1 levels, growth hormone levels, and treatment tolerability, including assessments of injection site reactions and adverse events. The primary outcome is the percentage of participants with IGF-1 levels at or below the upper limit of normal at 36 weeks. Safety and efficacy measures continue for up to 108 weeks, with ongoing monitoring of medication levels and rescue treatment use.

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 studying tumors of the hypothalamic-pituitary unit in children and adults aged 2 to 70 years, aiming to understand the genetic causes and developmental pathways involved in pituitary gland tumors. This observational study also serves as a training protocol for fellows and students to learn about genetic defects, tumor recognition, management, and complications. The study includes research on new MRI tools and psychological effects of cortisol secretion in pediatric patients with Cushing disease. Participants with tumors or related hypothalamic-pituitary disorders undergo various procedures including MRI scans and tissue specimen collection. The study collects peripheral blood samples and tumor tissues for molecular genetic testing and whole exome sequencing. It also evaluates cognitive, psychological, and patient-reported outcomes related to hypercortisolemia and adrenal insufficiency in children with Cushing syndrome. Participants are assessed through imaging studies, biochemical tests, and genetic analysis. The study monitors psychological and cognitive health and collects patient-reported data on mental and social well-being. This protocol also functions as a screening platform for future clinical studies on diagnosis, treatment, and follow-up of pituitary tumors. Participation involves ongoing evaluation with no specified end date, supporting the development of new therapeutic approaches and research in pituitary tumor biology.

This research aims to collect long-term safety and effectiveness information on octreotide subcutaneous depot in patients with acromegaly in real-world settings. The study will observe patients who have started or will start this treatment, focusing on how the drug works and any side effects over up to two years. It is an observational study sponsored by Camurus AB. Participants will use commercially available octreotide subcutaneous depot treatment for up to two years while researchers monitor their health. The study does not assign treatment but observes patients under normal care conditions. The main focus will be on related adverse events, changes in hormone levels such as IGF-1 and growth hormone, and symptom changes measured by specific acromegaly questionnaires. During the study, participants will be regularly assessed for side effects and changes in their condition. Researchers will collect data on hormone levels, symptom severity using the Acromegaly Index of Severity scale, and quality of life through several questionnaires. Safety monitoring includes recording any adverse events and lab abnormalities. The total participation time may last up to two years, with ongoing observation from enrollment through the treatment 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.

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, Rathkes 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.

Pheochromocytomas and paragangliomas are rare tumors arising from adrenal glands or nearby tissues, causing high blood pressure due to excess catecholamine release. These tumors can be hard to detect and pose serious risks during surgery, anesthesia, or childbirth. This study aims to improve diagnosis, localization, and treatment methods by investigating genetic, molecular, imaging, and biochemical features of these tumors, including their behavior and potential therapies. Participants include adults and children aged 3 years and older with known or suspected pheochromocytoma or paraganglioma. The study involves blood and urine tests for catecholamines and related compounds, as well as various imaging scans such as CT, MRI, and specialized PETCT scans using radioactive tracers like 18F-DOPA and 18F-6F-DA. Genetic testing is also performed. If a tumor is found, surgery may be offered if surgery is not possible, ongoing evaluations and medical treatments continue. Participants undergo medical history reviews, physical exams, ECGs, and regular biochemical and imaging assessments. Follow-up visits monitor tumor presence and treatment effects. The study measures tumor genotypes, biochemical profiles, imaging characteristics, and explores potential treatments using cell studies. Education for healthcare providers and patients about these tumors is also a goal. Participation can last many years, with ongoing monitoring and data collection.