Hypopituitarism

Researchers are evaluating the safety and tolerability of GenSci134, a drug given by subcutaneous injection, in healthy adult volunteers and adults with Growth Hormone Deficiency (AGHD). This study has two parts: the first part involves healthy volunteers in two phases—single and multiple ascending doses—and the second part includes AGHD patients receiving a single ascending dose. The trial aims to assess safety, how the drug acts in the body (pharmacokinetics and pharmacodynamics), immune responses, and other exploratory effects. In the first part, Phase Ia tests single doses of GenSci134 in healthy adult males across seven dose groups with placebo and active controls. Phase Ib evaluates multiple doses in healthy adult males and females in three dose groups. The second part, Phase Ic, studies two dose groups of AGHD patients receiving single doses of GenSci134 compared to active controls. The active comparator is recombinant human growth hormone injection given daily for 28 days. Treatments are given subcutaneously, and the study is randomized and double-blind. Participants will undergo safety assessments including monitoring adverse events for up to three months after dosing. Researchers will collect data on drug absorption, effects on the body, immune response, and tolerability. Healthy volunteers and AGHD patients will be monitored through scheduled visits involving laboratory tests and evaluations to track any side effects and responses to the treatments. The total participation duration varies by study phase and dosing schedule.

Researchers are evaluating the long-term safety of lonapegsomatropin in children with growth hormone deficiency who are already being treated with this medication. This study is observational and aims to gather real-world safety data over time after the drug has been authorized for use. The focus is on monitoring potential risks such as the development of tumors and type 2 diabetes over a period of five years. Participants receive lonapegsomatropin, administered once weekly by subcutaneous injection as part of their usual care. The study does not involve additional interventions but observes patients who are already treated with this therapy. The observational period extends for at least five years to collect safety data and compare it with historical information from previous studies. During the study, researchers will monitor for the occurrence of benign and malignant tumors, type 2 diabetes, and other adverse events affecting the kidneys, liver, immune system, and nervous system. They will also track medication errors and measure the response of Insulin-like Growth Factor-1 (IGF-1) to treatment. Participants are followed up regularly to collect this information, with the study lasting until March 2033.

Researchers are studying how well setmelanotide works to reduce weight, hunger, and improve quality of life in patients aged 4 years and older with congenital Hypothalamic Obesity (cHO). This phase 3 trial evaluates the safety and effectiveness of setmelanotide compared to placebo in this population. Participants have specific diagnoses related to hypothalamic injury and hormone deficiencies, with elevated body mass index (BMI) or BMI percentiles as criteria. Participants will receive a daily subcutaneous injection of either setmelanotide or a placebo in a randomized 2:1 ratio. The treatment period lasts for 26 weeks on a therapeutic regimen. The study is double-blind and placebo-controlled to objectively assess the effects of setmelanotide on weight and hunger measures. During the trial, participants will complete assessments measuring changes in BMI, hunger scores, weight, and quality of life related to weight impact. Safety will be monitored by tracking adverse events throughout the 26-week period. Additional evaluations include changes in BMI z-scores for pediatric patients and physical functioning scores. The trial is sponsored by Rhythm Pharmaceuticals, Inc. and will follow participants from baseline through the 26-week treatment duration.

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.

This research aims to gather long-term evidence on the safety and effectiveness of SKYTROFA (lonapegsomatropin) in patients with growth hormone deficiency who are receiving routine clinical care. The study focuses on understanding how patients respond to this treatment over time without altering their usual medical management. It is a non-interventional observational study conducted across multiple centers in the USA. Patients will continue their regular treatment with SKYTROFA as managed by their healthcare providers, with no additional visits, examinations, or tests required beyond standard clinical care. The study will collect data from clinical outcome assessments (COAs) to evaluate treatment results and safety over a period of five years. No experimental interventions or placebos are involved. Participants will be monitored through their routine clinical visits, with researchers reviewing their health status and treatment effectiveness using standard assessments. The primary outcomes include measuring safety and treatment effectiveness over five years, along with evaluating clinical outcomes through COAs. This approach allows for natural observation of patient experiences without extra procedures or disruptions to their care.

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 evaluate the effects of additional human chorionic gonadotropin (hCG) injections for luteal phase support (LPS) on pregnancy outcomes in women with idiopathic hypogonadotropic hypogonadism (IHH). IHH is a rare condition caused by genetic variants leading to hormone secretion issues, resulting in delayed puberty, amenorrhea, infertility, and psychological challenges. Luteal phase deficiency (LPD) is a major cause of failure in assisted reproductive treatments and early pregnancy loss in these patients. This open-labeled, prospective, randomized clinical trial conducted in a single center investigates whether an extra hCG injection 48 hours after the routine trigger can improve pregnancy success rates and reduce complications like early pregnancy loss and ovarian hyperstimulation syndrome (OHSS). Participants are randomly assigned to one of two groups: one receives an additional hCG injection of 2000-5000 IU 48 hours after the routine hCG trigger and ovulation, alongside estrogen and dydrogesterone supplementation for luteal phase support. The other group receives only estrogen and dydrogesterone. The trial compares clinical pregnancy rate at 7 weeks, cumulative pregnancy rate at 12 weeks, and live birth rate at 42 weeks or beyond to assess the impact of the added hCG. During the study, women's reproductive and psychological health will be monitored, including pregnancy outcomes and mental health conditions such as depression and anxiety linked to reproductive needs. Researchers will track early pregnancy loss and OHSS occurrences. Participants will be followed through pregnancy milestones, with the total study duration covering up to the live birth period, allowing comprehensive evaluation of treatment effects and associated emotional outcomes.

This research aims to compare the thickness of the endometrial lining in adolescent and young adult females on estrogen replacement therapy using two types of progesterone treatments: daily oral micronized progesterone and the etonogestrel implant. The study focuses on patients aged 12 to 25 years with specific diagnoses requiring estrogen therapy, addressing gaps in knowledge about endometrial protection and bleeding patterns in this group. Participants will choose between two progesterone therapies: oral micronized progesterone taken daily at 100mg or the etonogestrel implant placed in a clinic. The study will last six months, during which participants will continue their estrogen therapy and start their selected progesterone treatment. Two pelvic ultrasounds will be performed, one at the start and one after six months of progesterone use, to measure changes in endometrial thickness. Throughout the study, participants will complete surveys about bleeding patterns, side effects, satisfaction, and adherence to their progesterone treatment. The study team will track medication use through pill counts or photos for those on oral progesterone. All ultrasound and survey data will be securely stored and analyzed to assess the mean change in endometrial thickness and other patient-reported outcomes over six months.

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 at the University of Texas Health Science Center at San Antonio are studying individuals with chromosome 18 abnormalities to better understand the genetic causes and effects of these conditions. The study aims to identify how growth hormone deficiency and other genetic factors impact brain structure and cognitive function, as well as physical and behavioral traits. The goal is to provide comprehensive medical and educational resources, perform clinical and basic research, and develop treatments to improve the lives of affected individuals. Participants undergo various evaluations including genetic testing of DNA from subjects and their parents to determine genotype. Clinical assessments include testing growth hormone and other hormone levels, psychiatric and neuropsychological evaluations, audiology and ENT exams, brain MRI scans, genetic dysmorphology, neurology, dental, speech pathology, gastrointestinal, orthopedic, and ophthalmologic examinations. These assessments are longitudinal, with participants of a wide age range, and not all tests apply to every participant at every visit. Participants will be involved in thorough clinical evaluations and multiple specialized exams over time to gather detailed health data. These include hormone tests, brain imaging, behavioral and cognitive assessments, and physical exams. Researchers will monitor growth hormone status and other health markers to understand the condition's impact. The study is ongoing and designed to provide long-term data to guide future treatments and support. Total participation time varies depending on individual assessments and follow-up needs.