Growth Hormone Deficiency

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 studying the development of neural flexibility, measured by MRI, as an indicator of cognitive flexibility in Chinese toddlers aged 32 to 42 months. The study also evaluates executive functions such as inhibition, shifting attention, emotional control, working memory, and planning using the Global Executive Composite score from a standardized rating scale. This research aims to better understand brain development and cognitive abilities during early childhood. Participants are divided into two groups based on their daily milk consumption: one group receives S-26 GOLD or ULTIMA GUM formula, while the other consumes cow's milk. Parents continue feeding their children these milk products throughout the study, and information about milk consumption is collected starting up to two months before and during the study period. The brain is examined using MRI, a safe and non-invasive imaging technology. During the study, toddlers undergo MRI scans and assessments of brain structure and function at the start and again at 42 months of age. Researchers also evaluate cognitive flexibility, behavior, curiosity, sleep quality, home environment, feeding practices, and fecal microbiota composition over time. Parents provide information through interviews, and the child's development and school readiness are monitored. The study runs from baseline through 42 months of age, with additional home environment data collected at 8 months after baseline.

This research aims to evaluate the effects of different doses of vosoritide and compare the therapeutic dose of vosoritide to human growth hormone (hGH) in children diagnosed with idiopathic short stature (ISS). The study is a Phase 2, randomized, controlled trial that seeks to understand how these treatments influence growth in affected children. Participants will first undergo a minimum 6-month observation period to assess their baseline growth. Then, those assigned to the vosoritide and placebo groups will receive randomized treatment for at least 6 months, with placebo limited to a maximum of 6 months. After this, open-label vosoritide treatment will continue until participants reach near-final adult height or at least 16 years for females and 18 years for males, whichever is later. Participants randomized to the hGH group will receive open-label hGH treatment for a minimum of 4 years. Study treatments involve daily injections. Throughout the study, participants will attend regular visits for clinical and imaging assessments, including evaluations of hips and lower extremities. Researchers will monitor safety concerns such as hypotension, fractures, and slipped capital femoral epiphysis, with oversight from an independent Data Monitoring Committee. The main outcomes measured include changes in annualized growth velocity at 6 months and height changes over 4 years. Follow-up assessments will continue as needed, including safety monitoring, until study completion, which could last up to 15 years.

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 evaluating the safety and effectiveness of a liposomal mitoxantrone combination regimen in treating relapsed and refractory solid tumors in children, adolescents, and young adults aged 6 to 24 years. This prospective, single-arm, multicenter clinical study aims to improve the objective response rate compared to historical data and to assess related side effects. The study focuses on specific tumor types including bone and soft tissue tumors and neuroblastoma among others. Participants will receive a treatment regimen combining mitoxantrone liposome, capecitabine, and anlotinib over 4 to 6 cycles, each lasting 21 days. Capecitabine is given twice daily on days 1 to 14, while anlotinib dosage is adjusted based on body weight and administered for 14 days per cycle. Those showing partial response or better may receive additional local treatments such as surgery or radiotherapy. During the study, participants will have regular checkups and tests to monitor tumor status, blood parameters, and other relevant health indicators. The main measurement is the objective response rate after up to 6 treatment cycles. Other outcomes include disease control rate, progression-free survival, overall survival, and safety monitoring for up to one year after treatment. The total observation period includes follow-up assessments to evaluate lasting effects and adverse events.

Researchers are studying the effects of growth hormone treatment on cognitive development and growth in infants born small for gestational age (SGA) who have not caught up in height or head circumference by the age of 6 months to 2 years. This study aims to evaluate whether early treatment with PEG-rhGH can improve IQ, behavior, and self-cognition in these children, as well as promote catch-up growth, especially during the critical developmental period before age two. Participants are assigned either to a treatment group receiving PEG-rhGH injections at an initial dose of 0.2 mg/kg once weekly by subcutaneous injection at bedtime for 104 weeks, with dosage adjustments based on IGF-1 levels, or to a control group that receives no treatment but undergoes the same follow-up assessments over the 104-week period. This non-randomized study compares growth and cognitive development outcomes between these two groups. Throughout the study, children will be monitored regularly for growth parameters including head circumference and height, as well as cognitive, motor, social, and behavioral development using standardized scales like the Griffiths mental development scale and the Achenbach children’s behavior scale. The study also evaluates brain structure and function through imaging and metabolic assessments, and safety is closely tracked over the two years of participation.

Researchers are observing children with Idiopathic Short Stature (ISS) to collect baseline growth data and other important health information. This observational study aims to better understand growth patterns in children diagnosed with ISS, focusing on how their height and body measurements change over time. The study is sponsored by BioMarin Pharmaceutical and involves children aged 2 to 16 years. The study does not involve any specific treatments but monitors participants regularly to track growth and related health variables. Data such as height, body mass index (BMI), and growth velocity will be collected every six months. The observation period can last up to 15 years, allowing researchers to gather long-term information about growth changes and possible medical events. Participants will have their height, BMI, and growth velocity measured every six months throughout the study. Researchers will also collect information on medical events and health outcomes related to short stature. The study includes regular evaluations to monitor safety and growth progression, with total participation lasting up to 15 years. This long follow-up helps to understand how children with ISS grow over time.

The purpose of this study is to learn about the safety of Somatrogon for the treatment of pediatric growth hormone deficiency (p GHD) in India. Pediatric GHD is a condition caused by too less amounts of growth hormone in the body. Children with GHD have a short height. GHD can be present at birth or develop later. The condition occurs if the pituitary gland makes too little growth hormone. This is a small gland at the bottom of the brain that releases hormones that affect growth and other body functions. This study is seeking for participants who are: * confirmed with p GHD. * given Somatrogon to be taken as an injection. The safety of Somatrogon injection will be checked based on side effects. These side effects can happen within 3 years after taking Somatrogon. A side effect is something (expected or unexpected) that you feel was caused by a medicine or treatment you take. The study doctor will collect side effect information and put the information on patient's case form. Follow-up of the patient's will be performed via clinic re-visit or over a call. It is not a rule for the participants to visit the clinic in this study. This study will help to see if Somatrogon is safe.

Researchers are investigating treatments for children aged 1 to 18 years with chronic kidney disease (CKD) and proteinuria, conditions where the kidneys do not function properly and leak protein into the urine. CKD can cause complications like high blood pressure, and treatments currently include ACE inhibitors (ACEI) or angiotensin receptor blockers (ARB) that help control blood pressure and protein levels. This study focuses on evaluating the safety and effects of adding finerenone to these standard treatments, aiming to better manage the overactive kidney system involved in these conditions. Participants will receive finerenone orally, adjusted by age and body weight, alongside their existing ACEI or ARB treatment. The study lasts about 19 months, with up to 18 months of finerenone treatment and a one-month follow-up. During this time, children who are new to finerenone will have at least 12 visits, while those already on finerenone will have at least 8 visits. At these visits, various health checks and sample collections will occur to monitor safety and kidney function. Throughout the study, researchers will regularly measure blood pressure, heart rate, temperature, height, and weight, and collect blood and urine samples to assess kidney function and protein levels. They will also perform heart tests using electrocardiograms and echocardiography. Participants and their guardians will answer questions about medication use and any medical problems experienced. Safety will be closely monitored by tracking any adverse events during the study and a health check will be done about 30 days after treatment ends.

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.