Stop penicillin in children with mild rheumatic heart disease to see if it’s still safe
Official title: A Non-Inferiority Trial of Stopping Penicillin in Early Rheumatic Heart Disease: GOAL-Stop
Run by Children's Hospital Medical Center, Cincinnati · NCT07146048 · Data updated 2026-11-12
This study tests whether stopping secondary antibiotic prophylaxis is no worse than continuing it for 2 years in kids and teens with mild rheumatic heart disease who have already been treated.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Ages | 5 Years to 20 Years |
| Sex | All |
| Enrollment | 922 (estimated) |
| Sponsor | Children's Hospital Medical Center, Cincinnati Academic / other |
| Locations | 1 site (Kampala, UG) |
| Status | Recruiting |
| Started | March 23, 2026 |
| Main results expected | February 20, 2029 |
| Study ends | December 31, 2029 |
| Registry record updated | June 22, 2026 |
| Trial ID | NCT07146048 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Children and adolescents aged 5–20 years with prior GOALIE participation who show echocardiographic normalization or stable mild RHD after at least two years of secondary prophylaxis.
✕Probably not a fit
Participants with RHD progression or those who do not meet inclusion criteria (e.g., higher disease stage or incomplete GOALIE data) may not benefit and could be at risk if prophylaxis is stopped.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
GOAL-Stop is a randomized, non-inferiority trial enrolling children and adolescents aged 5 to 20 years who have had at least two years of secondary prophylaxis and show either normalized or stable mild RHD on echo. Participants are assigned to either continue or stop prophylaxis for two years, with the main outcome being changes seen on echocardiography using the 2023 World Heart Federation criteria, evaluated by blinded adjudicators. The trial will also explore differences between those with normalization versus stable mild disease, and whether prior prophylaxis route (oral vs. intramuscular) affects outcomes. Conducted with a lead sponsor in Cincinnati and collaborators in Uganda, the study targets patients who previously participated in GOALIE and meet age and disease criteria. Results could inform guidelines on the necessity of long-term antibiotic prophylaxis in this population.
Why it matters
Potential benefit: If stopping prophylaxis is non-inferior, some children could avoid unnecessary antibiotic exposure without increasing disease progression.
How similar studies have gone: Previous work in this area has used non-inferiority designs to question long-term prophylaxis, but results vary by population and disease stage; this study adds randomized, blinded adjudication in a pediatric, global-health context.
Who can join: the full rules
Show full inclusion / exclusion 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
Where it's running
-
Uganda Heart Institute
Kampala, Ug, Uganda Recruiting
Study contacts
- Principal investigator: Andrea Z Beaton, Dr — Children's Hospital Medical Center, Cincinnati
- Study coordinator: Ndate Fall, PhD
- Email: ndate.fall@cchmc.org
- Phone: 15135171327
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.