A Non-Inferiority Trial of Stopping Penicillin in Early Rheumatic Heart Disease: GOAL-Stop
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Secondary Prophylaxis.
- Who it may be relevant to
- Registry conditions: Rheumatic Heart Disease. Basic parameters: 5 years — 20 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Uganda
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Overview
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).
Interventions
- Drug Secondary Prophylaxis
Continue standard of care with continued secondary antibiotic prophylaxis
Primary outcome measures
- Presence of echocardiographic progression of RHD at two years [Time frame: Enrollment to the end of treatment at 2 years]
Secondary outcome measures (1)
- Non-inferiority among sub-groups [Time frame: Enrollment to the end of 2 years participation]
Eligibility criteria
Inclusion criteria
- Participated in GOALIE
- Concluded GOALIE with either echocardiographic normalization or stable mild RHD
- Between ages 5-20 years at time of enrollment
Exclusion criteria
- RHD Stage C/D at GOALIE end of study echocardiogram
- Relocation to extremely distant residence or school
- Clinically documented penicillin allergy
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Single blind
- Primary purpose
- Treatment
Study locations
Uganda · 1 center
- Uganda Heart Institute — Kampala
Identifiers
NCT: NCT07146048 · 2025-0604