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Researchers are evaluating whether an investigational drug called OHB-607 can prevent Bronchopulmonary Dysplasia (BPD), a common chronic lung disease, in extremely premature infants. The study compares infants receiving OHB-607 alongside standard neonatal care to those receiving standard care alone to reduce the burden of this lung condition. This is a Phase 2b, multicenter, randomized, open-label study focused on safety and clinical efficacy. Participants will receive an intravenous infusion of OHB-607 from birth until reaching a postmenstrual age (PMA) of 29 weeks and 6 days. The study includes two arms: one group receives the investigational drug plus standard care, while the other group receives only standard neonatal care. The treatment period ends at 29 weeks plus 6 days PMA, after which infants are monitored. Throughout the study, researchers will track the incidence of severe BPD or death up to 36 weeks PMA, whichever occurs first. Assessments will include clinical evaluations and monitoring for safety and any side effects. The study also involves long-term follow-up to observe the infants' health outcomes beyond the treatment period. Participation involves consent from parents and collection of birth and medical history information.

Researchers are investigating the effects of multiple doses of vosoritide and comparing its therapeutic dose to human growth hormone (hGH) in children with idiopathic short stature (ISS). This Phase 2 study aims to understand how these treatments influence growth in this population. After an initial observation period of at least 6 months to measure baseline growth, participants are randomly assigned to receive either vosoritide, placebo, or hGH (the latter only in the United States). Those in the vosoritide and placebo groups undergo up to 6 months of randomized treatment, followed by open-label vosoritide until they reach near-final adult height or at least age 16 for females or 18 for males. Participants assigned to hGH receive open-label treatment for a minimum of 4 years. Throughout the study, safety is carefully monitored with clinical and imaging assessments focused on hips and lower extremities, as well as watching for hypotension, fractures, and slipped capital femoral epiphysis. An independent Data Monitoring Committee reviews safety data regularly. Study visits include a treatment completion visit about 4 weeks after the last dose, and follow-up assessments may continue annually through the end of the study. Key outcome measures include changes in annualized growth velocity at 6 months and changes in height and height Z-score after 4 years.

Researchers are evaluating the safety and appropriate dosing of an oral drug called CB-03-10 in people with advanced solid tumors that have not responded to standard treatments. This early phase 1 study aims to find the maximum tolerated dose and recommended dose for further studies while assessing the drug's effects in specific tumor types. Participants will undergo initial evaluations to determine the extent of their disease before starting treatment. The study has two parts: Part 1 uses a dose escalation method where participants receive increasing doses of CB-03-10 based on a standard oncology design to identify the safest maximum dose. Plasma samples will be collected at specific times to study the drug's behavior in the body. In Part 2, participants receive CB-03-10 at the dose identified in Part 1, focusing on particular tumor subtypes determined by a Safety Review Committee. Participants will be monitored weekly at first—two cycles in Part 1 and one cycle in Part 2—then every two weeks afterward. Disease reassessment happens at Week 8 and every 8 weeks after that. If participants show signs of improvement, they will be checked again after at least 4 weeks to confirm the response. The main outcomes measured are the highest dose that can be tolerated and any dose-limiting side effects within about one month of treatment.

Researchers are evaluating the effects of BMN 333 compared with vosoritide on growth in children with achondroplasia who have not previously received growth-promoting treatments. This study is a multicenter, multinational, randomized, active-controlled, seamless Phase 2/3 trial designed to select the best BMN 333 dose in Phase 2 and then compare it with vosoritide in Phase 3. The goal is to understand the safety and effectiveness of these treatments in improving growth in affected pediatric participants. The study includes two parts: Phase 2 will determine the optimal BMN 333 dose and whether to continue to Phase 3. Phase 3 will then compare the selected BMN 333 dose to vosoritide. Treatment lasts 52 weeks, with BMN 333 given once weekly by subcutaneous injection and vosoritide given once daily by subcutaneous injection. The total study duration, including screening and safety follow-up, can be up to 61 weeks. Participants will be monitored regularly throughout the study, with growth velocity measured at weeks 26, 39, and 52 to assess treatment effects. Safety follow-up visits are included after the treatment period. Researchers will collect data on growth rates and any side effects to evaluate how these treatments influence growth in children with achondroplasia over the course of the study.