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

GOAL-Stop is a randomized, controlled, non-inferiority trial designed to evaluate whether discontinuing secondary antibiotic prophylaxis (SAP) is non-inferior to continuing SAP in preventing progression of rheumatic heart disease (RHD) among children and adolescents. The trial will enroll participants aged 5-20 years with previously diagnosed mild RHD who have received at least 2 years of SAP and who demonstrate either echocardiographic normalization or stability (persistent mild RHD). Participants will be randomized to either continue SAP or discontinue SAP for 2 years. The primary outcome is echocardiographic progression of RHD at 2 years, assessed by blinded adjudicators using the 2023 World Heart Federation criteria. Subgroup analyses will evaluate outcomes in participants with echocardiographic normalization versus stable mild RHD, and an exploratory analysis will assess whether outcomes differ by prior prophylaxis route (oral vs. intramuscular).

NCT Number	NCT07146048
Lead Sponsor	Children's Hospital Medical Center, Cincinnati Collaborators: Uganda Heart Institute, Open Philanthropy, Thrasher Research Fund
Conditions	Rheumatic Heart Disease
Enrollment	922 participants
Start Date	2026-03-23
Primary Completion	2029-02-20 (estimated)
Study Completion	2029-12-31 (estimated)
Updated on ClinicalTrials.gov	2026-06-22