California City

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 characteristics and outcomes of individuals with asthma across different levels of disease severity in routine clinical practice. The study aims to describe participants sociodemographic and clinical features, treatment patterns, disease burden, biomarkers, and both asthma-specific and general quality of life. This research includes both a cross-sectional analysis and a prospective follow-up to observe changes in disease progression over time. The study involves participants receiving standard asthma care, including treatment with varying doses of inhaled corticosteroids andor biologic therapies. Participants are grouped based on asthma control levels and biomarker status. The first part of the study collects baseline data cross-sectionally, while the second part follows participants prospectively to assess differences in asthma symptom control, treatment use, lung function, and comorbidities over a two-year period. Participants will be involved in scheduled data collection including patient and physician-reported outcomes, lung function tests, blood samples, and questionnaires assessing quality of life and symptom control. The study monitors treatment utilization and health resource use from the prior year and during follow-up visits at one and two years. The total participation period spans up to two years with ongoing assessment of disease characteristics and outcomes.