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).
Participated in GOALIE
Concluded GOALIE with either echocardiographic normalization or stable mild RHD
Between ages 5-20 years at time of enrollment
RHD Stage C/D at GOALIE end of study echocardiogram
Relocation to extremely distant residence or school
Clinically documented penicillin allergy
Parallel
Secondary Prophylaxis
DrugContinue standard of care with continued secondary antibiotic prophylaxis
Presence of echocardiographic progression of RHD at two years
The Primary Outcome will be the presence of echocardiographic progression of RHD at two years, defined by the 2023 World Heart Federation Criteria (none, mild RHD (stage A/B), or moderate/severe RHD (stage C/D).
Non-inferiority among sub-groups
Number of participants who show echocardiographic progression (2023 World Heart Federation Criteria) between those with echocardiographic normalization and those with stabilized mild RHD
Exploratory sub-analysis
Number of echocardiographic progression outcomes based on the initial type of prophylaxis during the GOALIE Trial, oral prophylaxis vs. intramuscular prophylaxis.
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Children's Hospital Medical Center, Cincinnati
Lead sponsor
Uganda Heart Institute
Collaborator
Open Philanthropy
Collaborator
Thrasher Research Fund
Collaborator
