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 phasessingle and multiple ascending dosesand 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 cows 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 childs 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 conducting an observational study to collect baseline growth data in children diagnosed with Idiopathic Short Stature ISS. The study aims to better understand growth patterns by measuring various growth-related factors over time in this pediatric population aged between 2 and 16 years, with a focus on children experiencing significant short stature. This long-term study is sponsored by BioMarin Pharmaceutical to help characterize how children with ISS grow compared to typical growth standards. Participants will be observed without receiving any study treatments. Growth measurements such as annualized growth velocity, height Z-score, standing height, body mass index BMI, and BMI Z-score will be collected every six months. The study will also record medical events and explore relationships between genetic variants and growth velocity. This observational approach allows tracking changes over time without intervention. Families will be involved in regular assessments every six months for up to 15 years. These evaluations include physical growth measurements and monitoring of medical events related to short stature. Data from historic growth hormone hGH stimulation tests and treatment status will also be considered. The studys primary outcomes focus on changes in growth metrics, while secondary outcomes assess medical event rates and genetic associations. Participation requires ongoing cooperation for scheduled measurements and consent from parents or guardians.

This research aims to learn about the safety of Somatrogon, a long-acting growth hormone, for treating pediatric growth hormone deficiency p GHD in children aged 3 to 17 years in India. Pediatric GHD is a condition where the body produces too little growth hormone, leading to shorter height in affected children. The study focuses on participants confirmed with p GHD and prescribed Somatrogon injections. Participants will receive Somatrogon as part of their routine clinical care, and the study will observe their health over a period of up to three years. The safety will be monitored by tracking any side effects, expected or unexpected, that occur after taking Somatrogon. Follow-up will be conducted through clinic visits or phone calls, but regular clinic visits are not mandatory. During the study, researchers will collect information on adverse events and treatment adherence, including missed injections, over the three-year period. The goal is to understand the safety profile of Somatrogon in this patient group. Participants involvement includes providing informed consent, and assessments will be conducted as part of their usual care without additional interventions specifically for the study.

Researchers are studying children and young adults aged 1 to 18 years with chronic kidney disease CKD and proteinuria, a condition where the kidneys leak protein into the urine. This study aims to evaluate the long-term safety of finerenone when added to standard treatments called ACE inhibitors or angiotensin receptor blockers ARBs, which are commonly used to control blood pressure and protect kidney function. The research also seeks to understand how well finerenone can reduce protein levels in urine and support kidney health over time. Participants will receive finerenone in doses adjusted by age and body weight, taken orally for up to 18 months alongside their usual ACEI or ARB treatment. The study includes patients who previously took part in a related trial and will follow them for about 19 months, including a one-month follow-up after treatment ends. The research involves one group receiving finerenone openly without placebo or comparison groups. During the study, participants will attend at least 8 to 12 visits depending on their treatment start status. At these visits, doctors will measure vital signs like blood pressure, heart rate, weight, and height perform physical exams collect blood and urine samples to monitor kidney function and protein levels and conduct heart tests using electrocardiograms and echocardiography. Participants and their caregivers will also answer questions about medication use, side effects, and overall well-being. Safety will be closely monitored by tracking any medical problems that arise during the trial.

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.