Rheumatic Heart DiseasePhase 3GOAL-StopNCT07146048
A Non-Inferiority Trial of Stopping Penicillin in Early Rheumatic Heart Disease: GOAL-Stop
A Phase 3 study testing Secondary Prophylaxis in people with Rheumatic Heart Disease, run by Children's Hospital Medical Center, Cincinnati at 1 site in Uganda.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 5 to 20 years
- Conditions the study is for
- Rheumatic Heart Disease
- What is being tested?
- Secondary Prophylaxis
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 922 participants
- Where
- 1 siteUganda
- Study dates
- 23 Mar 2026to 31 Dec 2029
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 20 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Participated in GOALIE
- Concluded GOALIE with either echocardiographic normalization or stable mild RHD
- Between ages 5-20 years at time of enrollment
Who cannot join
- RHD Stage C/D at GOALIE end of study echocardiogram
- Relocation to extremely distant residence or school
- Clinically documented penicillin allergy
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Uganda1 site, 1 recruiting
- Recruiting
Uganda Heart Institute
Kampala, UG
Emmy H Okello, Dr, +256775522284, emmyoks@gmail.com
Joselyn Rwebembera, Dr, +256779010527, jrwebembera@rrcuganda.org
About this study
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).
- Conditions
- Rheumatic Heart Disease
- Being tested
- Secondary Prophylaxis
- Keywords
- Rheumatic heart disease, global health, secondary prophylaxis, children
- Medical subject headings
- Rheumatic Heart Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Secondary ProphylaxisDrug
Continue standard of care with continued secondary antibiotic prophylaxis
| Study group | Role | What they receive |
|---|---|---|
| Stop prophylaxis | No intervention | The intervention arm will not receive prophylaxis. |
| Continue prophylaxis | Active comparator | The control arm will receive intramuscular benzathine benzylpenicillin G (BPG) prophylaxis (600,000 IU for children <30kg, 1.2 million IU for children ≥30kg), every 28 days for a 2-year period. The standard of care for RHD is BPG injections which is why the control arm is continuation of monthly injections. Receives: Drug: Secondary Prophylaxis |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: Enrollment to the end of treatment at 2 years
Secondary outcomes (1)
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
Measured: Enrollment to the end of 2 years participation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Aug 2025
- Study start23 Mar 2026
- Primary completion20 Feb 2029 (estimated)
- Study completion31 Dec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Rheumatic Heart DiseaseStudies of the same condition, open and under way.
- More trials run by Children's Hospital Medical Center, CincinnatiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 922 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Collaborators
- Uganda Heart Institute, Coefficient Giving, Thrasher Research Fund
- Responsible party
- Sponsor
- Investigators
- Andrea Z Beaton, Dr, Principal investigator, Children's Hospital Medical Center, Cincinnati
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07146048
- Sponsor's protocol ID
- 2025-0604
- Acronym
- GOAL-Stop
- Status last verified
- Jun 2026
- First submitted
- 20 Aug 2025
- Last updated
- 22 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided: Pending approval of consent form
Study at a glance
From the registry record, last updated by the sponsor on 22 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Non-Inferiority Trial of Stopping Penicillin in Early Rheumatic Heart Disease: GOAL-Stop
- ClinicalTrials.gov ID
- NCT07146048
- Acronym / sponsor ID
- GOAL-Stop / 2025-0604
- Conditions
- Rheumatic Heart Disease
- Keywords
- Rheumatic heart disease, global health, secondary prophylaxis, children
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 922 participants (estimated)
- Lead sponsor
- Children's Hospital Medical Center, CincinnatiUniversity, hospital or other
- Collaborators
- Uganda Heart Institute, Coefficient Giving, Thrasher Research Fund
- Study start
- 23 Mar 2026 (actual)
- Primary completion
- 20 Feb 2029 (estimated)
- Study completion
- 31 Dec 2029 (estimated)
- First posted
- 28 Aug 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Stop prophylaxis | No intervention | –The intervention arm will not receive prophylaxis. |
| Continue prophylaxis | Active comparator | Drug: Secondary ProphylaxisThe control arm will receive intramuscular benzathine benzylpenicillin G (BPG) prophylaxis (600,000 IU for children <30kg, 1.2 million IU for children ≥30kg), every 28 days for a 2-year period. The standard of care for RHD is BPG injections which is why the control arm is continuation of monthly injections. |
Who may qualify
- Ages
- 5 to 20 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- Participated in GOALIE
- Concluded GOALIE with either echocardiographic normalization or stable mild RHD
- Between ages 5-20 years at time of enrollment
Key exclusion criteria (3 listed)
- RHD Stage C/D at GOALIE end of study echocardiogram
- Relocation to extremely distant residence or school
- Clinically documented penicillin allergy
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Secondary ProphylaxisContinue standard of care with continued secondary antibiotic prophylaxis | Drug | Continue prophylaxis |
Primary outcome
Presence of echocardiographic progression of RHD at two years
Time frame: Enrollment to the end of treatment at 2 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).
1 secondary outcome
Non-inferiority among sub-groups
Time frame: Enrollment to the end of 2 years participation
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jun 2026.
- Related InfoLink provided by the sponsor.
- Individual participant dataSharing planned: Undecided. Pending approval of consent form
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Andrea Z Beaton, Dr principal investigator
Children's Hospital Medical Center, Cincinnati
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Secondary Prophylaxis in people with Rheumatic Heart Disease, run by Children's Hospital Medical Center, Cincinnati at 1 site in Uganda.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 22 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Uganda. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Ndate Fall, PhD (15135171327, ndate.fall@cchmc.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Presence of echocardiographic progression of RHD at two years (time frame: Enrollment to the end of treatment at 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jun 2026.