GOAL-Stop Trial Studying Stopping Versus Continuing Penicillin to Prevent Rheumatic Heart Disease Progression in Children and Teens

What this Trial Is About

Researchers are evaluating whether stopping secondary antibiotic prophylaxis SAP is not worse than continuing SAP in preventing worsening of rheumatic heart disease RHD in children and adolescents. This Phase 3 trial focuses on participants aged 5 to 20 years who have mild RHD and have already received at least two years of SAP. The goal is to see if stopping SAP affects heart disease progression compared to continuing treatment. The study is randomized and controlled with a single masking approach. Participants will be randomly assigned to either stop SAP or continue receiving monthly intramuscular benzathine benzylpenicillin G BPG injections for two years. Dosage is based on weight, with children under 30 kg receiving 600,000 IU and those 30 kg or above receiving 1.2 million IU every 28 days. The control group continues the standard care of monthly BPG injections, while the intervention group receives no prophylaxis for the same period. During the study, participants will be monitored for two years to assess the progression of RHD using echocardiographic evaluations based on the 2023 World Heart Federation criteria. Blinded adjudicators will review heart imaging results to determine disease changes. Researchers will also analyze outcomes for subgroups with either normalized or stable mild RHD and explore differences based on prior prophylaxis routes. The trial spans from enrollment through the end of the 2-year treatment period, with safety and disease progression closely followed.

A Non-Inferiority Trial of Stopping Penicillin in Early Rheumatic Heart Disease: GOAL-Stop