Moriyama

Researchers are conducting a Phase 3 pediatric study to create a framework for evaluating the safety and effectiveness of drugs for managing obesity or overweight conditions in children and teens. The study focuses on participants who have struggled to lose weight despite structured diet and exercise programs. The goal is to assess treatments for long-term weight management in this young population. Participants will receive either the study drug Orforglipron or a placebo orally. The interventions will be detailed in specific substudies, called ISAs, which may start independently as new treatments become available. Results from all ISAs will be reported once all are completed, providing insights into the treatments effects on pediatric obesity or overweight. During the study, participants will be evaluated from baseline to week 72, with the primary outcome measuring the number of participants assigned to each ISA. Participants will undergo assessments including body mass index evaluations based on age- and gender-specific growth charts, and monitoring for weight-related health conditions. Safety and efficacy data will be collected throughout the study duration, which extends to March 2027.

Researchers are studying baricitinib to see if it can help preserve beta-cell function in children and adults aged 1 to 35 years who have been newly diagnosed with type 1 diabetes. This Phase 3 study aims to evaluate the treatments impact on preserving insulin production shortly after diagnosis. Participants will be followed for about 60 weeks to assess changes in key diabetes-related measures. Participants will be randomly assigned to receive either baricitinib or a placebo, both taken orally. The study compares these two groups to evaluate the effects of baricitinib on beta-cell function. The main measurement is the change in C-peptide area under the curve over 52 weeks, which indicates insulin production. Additional outcomes include changes in blood sugar control, insulin use, hypoglycemia events, and other health indicators. During the study, participants will attend visits for assessments and monitoring over approximately 60 weeks. Tests will include blood measurements like C-peptide and hemoglobin A1c, insulin dose tracking, and health surveys. Safety and drug levels will be monitored. Researchers will use these data to understand if baricitinib can help maintain beta-cell function in people newly diagnosed with type 1 diabetes.

Researchers are evaluating the effects of orforglipron, taken once daily by mouth, compared to placebo in adolescents aged 12 to 17 years who have obesity or are overweight with related health conditions. The study aims to assess the drugs efficacy, safety, and how it is processed by the body. This phase 3 trial is conducted under a master protocol and sponsored by Eli Lilly and Company. Participants will be randomly assigned to receive either orforglipron or a placebo orally once daily. Those who complete the initial PW01 study, including a safety follow-up period, may continue receiving orforglipron for an additional 156 weeks while maintaining lifestyle interventions such as diet and exercise counseling. The trial follows a double-blind design where neither participants nor researchers know the assigned treatment. During the roughly 18-month participation, adolescents will undergo regular assessments including body mass index BMI, body weight, waist circumference, blood pressure, cholesterol, glucose levels, insulin levels, body fat mass via DXA scans, and quality of life related to weight impact. Pharmacokinetic measurements will also be taken to study the drugs concentration over time. Safety and effectiveness will be monitored through these evaluations at baseline and at Week 72.

Researchers are evaluating NS-089NCNP-02 in a Phase 2, open-label study involving ambulant boys aged 4 to less than 15 years with Duchenne Muscular Dystrophy DMD caused by mutations suitable for exon 44 skipping. The study aims to assess the safety, tolerability, pharmacokinetics, and effects of the drug on muscle dystrophin levels and physical function. This research is sponsored by NS Pharma, Inc. and involves two parts with distinct participant cohorts. The study includes two parts Part 1 involves six participants Cohort 1 receiving escalating doses of NS-089NCNP-02 by weekly intravenous infusion over a 4-week treatment phase per dose level. Part 2 involves both Cohort 1 and 14 additional participants Cohort 2 receiving a selected dose of NS-089NCNP-02 once weekly for 24 weeks. The drug is administered as a solution for infusion, and dosing in Part 2 is based on the maximum tolerated dose determined in Part 1. Participants will undergo assessments at baseline, during treatment, and follow-up visits including pharmacokinetic blood and urine tests, muscle biopsies to measure dystrophin protein and mRNA, and physical function tests such as the North Star Ambulatory Assessment, timed walking and climbing tasks, muscle strength tests, and grip and pinch strength. Safety monitoring includes tracking adverse events throughout the study and follow-up. The overall participation lasts through the treatment phases and a follow-up phone call after Part 2 completion.

Spinal muscular atrophy SMA is a genetic disorder caused by changes in the SMN1 gene, leading to low SMN protein levels and problems with motor neurons. It is an inherited disease that mainly affects children and is a leading cause of infant death due to genetic conditions. This research aims to observe patients with SMA over time to understand long-term outcomes with new treatments and to evaluate the long-term safety and effects of the investigational therapy OAV-101. This is a prospective, multinational, non-interventional observational study where patients receive their usual clinical care without changes directed by the study. Treatments such as Zolgensma may be given according to normal clinical practice, but this is not controlled by the study protocol. Patients will be followed for up to 15 years or until death, with no extra visits or tests beyond what is normally done for SMA care. Participants will be evaluated through regular clinical assessments based on their normal care schedule, with data collected at baseline and at intervals of every 6 months for the first 2 years, then yearly for up to 15 years. Researchers will measure survival rates, motor function scores like CHOP-INTEND and HFMSE, hospitalizations, quality of life, use of ventilator, nutritional, and mobility supports, as well as any treatment-related side effects during this long-term follow-up.

Researchers are evaluating the effects of balcinrenone combined with dapagliflozin compared to dapagliflozin alone in patients who have chronic heart failure, impaired kidney function, and have recently experienced a heart failure event. This Phase III study is conducted internationally across about 700 sites and aims to assess how these treatments impact cardiovascular death and heart failure events. Participants will be randomly assigned to one of three groups balcinrenonedapagliflozin 15 mg10 mg plus placebo, balcinrenonedapagliflozin 40 mg10 mg plus placebo, or dapagliflozin 10 mg plus placebo. Each participant will take one capsule and one tablet daily. The study duration averages 22 months, including screening, about 20 months of blinded treatment, and a one-month follow-up with open-label dapagliflozin. During the study, participants will undergo assessments for heart failure events, hospitalizations, and cardiovascular death. Researchers will monitor these outcomes over about 38 months, including symptom scores and other health measures. Safety and treatment effects will be followed during the treatment and the one-month post-treatment period.