Rheumatic Heart Disease in ChildrenPhase 2GOALNCT03346525
Determining the Impact of Penicillin in Latent RHD: The GOAL Trial
A Phase 2 study testing intramuscular benzathine penicillin G (BPG) prophylaxis in people with Rheumatic Heart Disease in Children and Latent Rheumatic Heart Disease, run by Children's National Research Institute at 1 site in Uganda.
Trial summary
Rheumatic heart disease (RHD) affects at least 32.9 million people, mostly children living in low-resource settings. Long-term intramuscular benzathine penicillin G (BPG) prophylaxis is proven to prevent progression of chronic valve changes in patients with established rheumatic heart disease (RHD) and to allow… Read more about this study
- Who is it for?
- Ages 5 to 17 years
- Conditions the study is for
- Rheumatic Heart Disease in Children, Latent Rheumatic Heart Disease, Rheumatic Heart Disease
- What is being tested?
- intramuscular benzathine penicillin G (BPG) prophylaxis
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 807 participants
- Where
- 1 siteUganda
- Study dates
- 26 Jun 2018to 30 Nov 2020
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Children will be eligible for study participation if they are (1) between the ages of 5-17 years and (2) have a new diagnosis of latent RHD detected through primary school echo screening and confirmed by blinded consensus review. All children will be recruited from schools in Gulu District in Uganda.
Who cannot join
- Patients will be excluded from the study for the following reasons:
- Known history of ARF or RHD
- Newly diagnosed RHD by echo screening consider to be "missed clinical RHD" as compared to true latent RHD including: > mild pathological valvular regurgitation at the mitral valve or aortic valve, mitral stenosis (mean MV gradient ≥ 5mmHg) (WHF, definite B), aortic stenosis (mean AV gradient ≥ 20mmHg)
- Structural or functional cardiac defects, other than those consistent with RHD, that were known prior to or detected through echo screening (except patent foramen ovale, small atrial septal defect, small ventricular septal defect, small patent ductus arteriosus).
- Prior allergic reaction to penicillin
- Any known conditions predisposing to thrombocytopenia or hypercoagulability, or other contraindications to intramuscular injection
- Any known co-morbid conditions (HIV, renal deficiencies, severe malnutrition among others) that have resulted in prescription of regular antibiotic prophylaxis
Trial locations and contacts
1 site in 1 country.
About this study
Official title: The GOAL (GwokO Adunu pa Lutino) Trial: Determining the Impact of Penicillin on Latent Rheumatic Heart Disease: A Randomized Controlled Trial in Uganda
Rheumatic heart disease (RHD) affects at least 32.9 million people, mostly children living in low-resource settings. Long-term intramuscular benzathine penicillin G (BPG) prophylaxis is proven to prevent progression of chronic valve changes in patients with established rheumatic heart disease (RHD) and to allow regression of valve changes in patients with a history of acute rheumatic fever (ARF) with mild RHD. However, in low-resource settings ARF is an elusive diagnosis, and most patients (85%) are diagnosed only when RHD is severe and irreversible, medications ineffective, and surgical intervention is expensive and/or unavailable.
Identification of latent RHD might be an opportunity to substantially reduce RHD morbidity and mortality. However, detection of latent RHD is only important if outcomes are improved. The appropriate management of children with latent RHD is unknown and no formal recommendations exist. While some clinicians prescribe penicillin prophylaxis for children with latent RHD, clinical equipoise exists regarding the best practice.
Read more
To fill this gap, the investigators propose a randomized controlled trial in children with latent RHD to evaluate the efficacy of BPG prophylaxis compared to no prophylaxis. Our primary outcome measure is progression of valvular changes on echocardiogram at 2 years. A sample size of 916 children is needed to detect a 50% reduction of progression (expected range 7.5-12.5% progression in BPG-arm vs. 15%-25% progression in control-arm) with 90% power.
AIM 1: To compare the proportion of children (aged 5-17 years) with latent RHD receiving BPG prophylaxis who progress to worse valvular disease at 2-years compared to children not receiving BPG prophylaxis.
Hypothesis 1: Prophylaxis with BPG will result in fewer children with latent RHD showing progression of echocardiographic valve changes at 2 years compared to children with latent RHD not receiving BPG prophylaxis. (The investigators expect at least a 50% relative reduction in progression in the BPG arm: range 15%-25% control arm vs. 7.5-12.5% BPG-arm.)
AIM 2: To compare the proportion of children (aged 5-17 years) with latent RHD receiving BPG prophylaxis who regress to improved valvular disease at 2-years compared to children not receiving BPG prophylaxis.
Hypothesis 2: Prophylaxis with BPG will result in more children with latent RHD showing regression of echocardiographic valve changes by 2 years compared to children with latent RHD not receiving BPG prophylaxis. (The investigators expect at least a 50% relative increase in regression in the BPG arm: range 10-20% control arm vs. 20-40% BPG arm.)
This study is highly significant because it will establish if BPG prophylaxis improves outcomes for children with latent RHD. Feasibility will be ensured through the experience, resources, community support, and accessible patient population of our investigational team. The results of our study will have high impact, immediately informing international policy on the standard of care for children diagnosed with latent RHD and shaping, over 2-3 years, practical and scalable programs that could substantially decrease the global burden of RHD.
- Conditions
- Rheumatic Heart Disease in Children, Latent Rheumatic Heart Disease, Rheumatic Heart Disease, Heart Diseases
- Being tested
- intramuscular benzathine penicillin G (BPG) prophylaxis
- Keywords
- echocardiography, penicillin prophylaxis, intramuscular benzathine penicillin G, benzathine penicillin G, low-resource setting
- Medical subject headings
- Rheumatic Heart Disease, Heart Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
intramuscular benzathine penicillin G (BPG) prophylaxisDrug
Also called benzathine penicillin G, BPG, penicillin prophylaxis
Intramuscular BPG prophylaxis (600,000 IU for children <30kg, 1.2 million IU for children ≥30kg), every 28 days.
| Study group | Role | What they receive |
|---|---|---|
| BPG Arm | Experimental | Intramuscular BPG prophylaxis (600,000 IU for children <30kg, 1.2 million IU for children ≥30kg), every 28 days Receives: Drug: intramuscular benzathine penicillin G (BPG) prophylaxis |
| Control Arm | No intervention | No prophylaxis |
What is being measured
The primary outcome is the main question the study is designed to answer.
progression of valvular changes on echocardiogram at 2 years
To compare the proportion of children (aged 5-17 years) with latent RHD receiving BPG prophylaxis who progress to worse valvular disease at 2-years compared to children not receiving BPG prophylaxis
Measured: 2 years
Secondary outcomes (1)
regression of vavular changes on electrocardiogram at 2 years
To compare the proportion of children (aged 5-17 years) with latent RHD receiving BPG prophylaxis who regress to improved valvular disease at 2-years compared to children not receiving BPG prophylaxis
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Nov 2017
- Study start26 Jun 2018
- Primary completion30 Nov 2020 (estimated)
- Study completion30 Nov 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (27)Papers the sponsor has linked to this study.
- Other trials for Rheumatic Heart Disease in ChildrenStudies of the same condition, open and under way.
- More trials run by Children's National Research InstituteThe 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 807 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's National Research Institute
- Sponsor type
- University, hospital or other
- Collaborators
- Thrasher Research Fund, University of Cape Town, Uganda Heart Institute, Karp Family Foundation, Gift of Life International, Murdoch Childrens Research Institute
- Responsible party
- Principal investigator, Andrea Beaton, Children's Hospital Medical Center, Cincinnati
- Investigators
- Andrea Beaton, MD, Principal investigator, Cincinnati Children's
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03346525
- Sponsor's protocol ID
- GOAL: RHD RCT
- Other IDs
- E.W. "Al" Thrasher Award
- Acronym
- GOAL
- Status last verified
- Sep 2019
- First submitted
- 15 Nov 2017
- Last updated
- 1 Oct 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
WHO - Global Burden of Disease. 2015;2015.
Lue HC, Wu MH, Wang JK, Wu FF, Wu YN. Long-term outcome of patients with rheumatic fever receiving benzathine penicillin G prophylaxis every three weeks versus every four weeks. J Pediatr. 1994 Nov;125(5 Pt 1):812-6. doi: 10.1016/s0022-3476(94)70082-6.
PubMed 7965439Tompkins DG, Boxerbaum B, Liebman J. Long-term prognosis of rheumatic fever patients receiving regular intramuscular benzathine penicillin. Circulation. 1972 Mar;45(3):543-51. doi: 10.1161/01.cir.45.3.543. No abstract available.
PubMed 5012243Zhang W, Mondo C, Okello E, Musoke C, Kakande B, Nyakoojo W, Kayima J, Freers J. Presenting features of newly diagnosed rheumatic heart disease patients in Mulago Hospital: a pilot study. Cardiovasc J Afr. 2013 Mar;24(2):28-33. doi: 10.5830/CVJA-2012-076.
PubMed 23612950Okello E, Wanzhu Z, Musoke C, Twalib A, Kakande B, Lwabi P, Wilson NB, Mondo CK, Odoi-Adome R, Freers J. Cardiovascular complications in newly diagnosed rheumatic heart disease patients at Mulago Hospital, Uganda. Cardiovasc J Afr. 2013 Apr;24(3):80-5. doi: 10.5830/CVJA-2013-004.
PubMed 23736132Beaton A, Okello E, Lwabi P, Mondo C, McCarter R, Sable C. Echocardiography screening for rheumatic heart disease in Ugandan schoolchildren. Circulation. 2012 Jun 26;125(25):3127-32. doi: 10.1161/CIRCULATIONAHA.112.092312. Epub 2012 May 24.
PubMed 22626741Steer AC, Kado J, Wilson N, Tuiketei T, Batzloff M, Waqatakirewa L, Mulholland EK, Carapetis JR. High prevalence of rheumatic heart disease by clinical and echocardiographic screening among children in Fiji. J Heart Valve Dis. 2009 May;18(3):327-35; discussion 336.
PubMed 19557993Engel ME, Haileamlak A, Zuhlke L, Lemmer CE, Nkepu S, van de Wall M, Daniel W, Shung King M, Mayosi BM. Prevalence of rheumatic heart disease in 4720 asymptomatic scholars from South Africa and Ethiopia. Heart. 2015 Sep;101(17):1389-94. doi: 10.1136/heartjnl-2015-307444. Epub 2015 Jun 15.
PubMed 26076935Zuhlke LJ, Beaton A, Engel ME, Hugo-Hamman CT, Karthikeyan G, Katzenellenbogen JM, Ntusi N, Ralph AP, Saxena A, Smeesters PR, Watkins D, Zilla P, Carapetis J. Group A Streptococcus, Acute Rheumatic Fever and Rheumatic Heart Disease: Epidemiology and Clinical Considerations. Curr Treat Options Cardiovasc Med. 2017 Feb;19(2):15. doi: 10.1007/s11936-017-0513-y.
PubMed 28285457Remenyi B, Wilson N, Steer A, Ferreira B, Kado J, Kumar K, Lawrenson J, Maguire G, Marijon E, Mirabel M, Mocumbi AO, Mota C, Paar J, Saxena A, Scheel J, Stirling J, Viali S, Balekundri VI, Wheaton G, Zuhlke L, Carapetis J. World Heart Federation criteria for echocardiographic diagnosis of rheumatic heart disease--an evidence-based guideline. Nat Rev Cardiol. 2012 Feb 28;9(5):297-309. doi: 10.1038/nrcardio.2012.7.
PubMed 22371105Beaton A, Lu JC, Aliku T, Dean P, Gaur L, Weinberg J, Godown J, Lwabi P, Mirembe G, Okello E, Reese A, Shrestha-Astudillo A, Bradley-Hewitt T, Scheel J, Webb C, McCarter R, Ensing G, Sable C. The utility of handheld echocardiography for early rheumatic heart disease diagnosis: a field study. Eur Heart J Cardiovasc Imaging. 2015 May;16(5):475-82. doi: 10.1093/ehjci/jeu296. Epub 2015 Jan 5.
PubMed 25564396Weinberg J, Beaton A, Aliku T, Lwabi P, Sable C. Prevalence of rheumatic heart disease in African school-aged population: Extrapolation from echocardiography screening using the 2012 World Heart Federation Guidelines. Int J Cardiol. 2016 Jan 1;202:238-9. doi: 10.1016/j.ijcard.2015.08.128. Epub 2015 Sep 12. No abstract available.
PubMed 26402451Roberts K, Colquhoun S, Steer A, Remenyi B, Carapetis J. Screening for rheumatic heart disease: current approaches and controversies. Nat Rev Cardiol. 2013 Jan;10(1):49-58. doi: 10.1038/nrcardio.2012.157. Epub 2012 Nov 13.
PubMed 23149830Karthikeyan G. Measuring and reporting disease progression in subclinical rheumatic heart disease. Heart Asia. 2016 Nov 28;8(2):74-75. doi: 10.1136/heartasia-2016-010857. eCollection 2016. No abstract available.
PubMed 27942046Nascimento BR, Nunes MC, Lopes EL, Rezende VM, Landay T, Ribeiro AL, Sable C, Beaton AZ. Rheumatic heart disease echocardiographic screening: approaching practical and affordable solutions. Heart. 2016 May;102(9):658-64. doi: 10.1136/heartjnl-2015-308635. Epub 2016 Feb 18.
PubMed 26891757
Study at a glance
From the registry record, last updated by the sponsor on 1 Oct 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The GOAL (GwokO Adunu pa Lutino) Trial: Determining the Impact of Penicillin on Latent Rheumatic Heart Disease: A Randomized Controlled Trial in Uganda
- ClinicalTrials.gov ID
- NCT03346525
- Acronym / sponsor ID
- GOAL / GOAL: RHD RCT
- Conditions
- Rheumatic Heart Disease in Children, Latent Rheumatic Heart Disease, Rheumatic Heart Disease, Heart Diseases
- Keywords
- echocardiography, penicillin prophylaxis, intramuscular benzathine penicillin G, benzathine penicillin G, low-resource setting
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 807 participants (actual)
- Lead sponsor
- Children's National Research InstituteUniversity, hospital or other
- Collaborators
- Thrasher Research Fund, University of Cape Town, Uganda Heart Institute, Karp Family Foundation, Gift of Life International, Murdoch Childrens Research Institute
- Study start
- 26 Jun 2018 (actual)
- Primary completion
- 30 Nov 2020 (estimated)
- Study completion
- 30 Nov 2020 (estimated)
- First posted
- 17 Nov 2017 (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.
Rheumatic heart disease (RHD) affects at least 32.9 million people, mostly children living in low-resource settings. Long-term intramuscular benzathine penicillin G (BPG) prophylaxis is proven to prevent progression of chronic valve changes in patients with established rheumatic heart disease (RHD) and to allow regression of valve changes in patients with a history of acute rheumatic fever (ARF) with mild RHD. However, in low-resource settings ARF is an elusive diagnosis, and most patients (85%) are diagnosed only when RHD is severe and irreversible, medications ineffective, and surgical intervention is expensive and/or unavailable.
Identification of latent RHD might be an opportunity to substantially reduce RHD morbidity and mortality. However, detection of latent RHD is only important if outcomes are improved. The appropriate management of children with latent RHD is unknown and no formal recommendations exist. While some clinicians prescribe penicillin prophylaxis for children with latent RHD, clinical equipoise exists regarding the best practice.
To fill this gap, the investigators propose a randomized controlled trial in children with latent RHD to evaluate the efficacy of BPG prophylaxis compared to no prophylaxis. Our primary outcome measure is progression of valvular changes on echocardiogram at 2 years. A sample size of 916 children is needed to detect a 50% reduction of progression (expected range 7.5-12.5% progression in BPG-arm vs. 15%-25% progression in control-arm) with 90% power.
AIM 1: To compare the proportion of children (aged 5-17 years) with latent RHD receiving BPG prophylaxis who progress to worse valvular disease at 2-years compared to children not receiving BPG prophylaxis.
Hypothesis 1: Prophylaxis with BPG will result in fewer children with latent RHD showing progression of echocardiographic valve changes at 2 years compared to children with latent RHD not receiving BPG prophylaxis. (The investigators expect at least a 50% relative reduction in progression in the BPG arm: range 15%-25% control arm vs. 7.5-12.5% BPG-arm.)
AIM 2: To compare the proportion of children (aged 5-17 years) with latent RHD receiving BPG prophylaxis who regress to improved valvular disease at 2-years compared to children not receiving BPG prophylaxis.
Hypothesis 2: Prophylaxis with BPG will result in more children with latent RHD showing regression of echocardiographic valve changes by 2 years compared to children with latent RHD not receiving BPG prophylaxis. (The investigators expect at least a 50% relative increase in regression in the BPG arm: range 10-20% control arm vs. 20-40% BPG arm.)
This study is highly significant because it will establish if BPG prophylaxis improves outcomes for children with latent RHD. Feasibility will be ensured through the experience, resources, community support, and accessible patient population of our investigational team. The results of our study will have high impact, immediately informing international policy on the standard of care for children diagnosed with latent RHD and shaping, over 2-3 years, practical and scalable programs that could substantially decrease the global burden of RHD.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| BPG Arm | Experimental | Drug: intramuscular benzathine penicillin G (BPG) prophylaxisIntramuscular BPG prophylaxis (600,000 IU for children <30kg, 1.2 million IU for children ≥30kg), every 28 days |
| Control Arm | No intervention | –No prophylaxis |
Who may qualify
- Ages
- 5 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- Children will be eligible for study participation if they are (1) between the ages of 5-17 years and (2) have a new diagnosis of latent RHD detected through primary school echo screening and confirmed by blinded consensus review. All children will be recruited from schools in Gulu District in Uganda.
Key exclusion criteria (7 listed)
- Patients will be excluded from the study for the following reasons:
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 |
|---|---|---|
| intramuscular benzathine penicillin G (BPG) prophylaxisAlso: benzathine penicillin G, BPG, penicillin prophylaxisIntramuscular BPG prophylaxis (600,000 IU for children <30kg, 1.2 million IU for children ≥30kg), every 28 days. | Drug | BPG Arm |
Primary outcome
progression of valvular changes on echocardiogram at 2 years
Time frame: 2 years
To compare the proportion of children (aged 5-17 years) with latent RHD receiving BPG prophylaxis who progress to worse valvular disease at 2-years compared to children not receiving BPG prophylaxis
1 secondary outcome
regression of vavular changes on electrocardiogram at 2 years
Time frame: 2 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Oct 2019.
- BackgroundWHO - Global Burden of Disease. 2015;2015.
- Background: PubMed 7965439Lue HC, Wu MH, Wang JK, Wu FF, Wu YN. Long-term outcome of patients with rheumatic fever receiving benzathine penicillin G prophylaxis every three weeks versus every four weeks. J Pediatr. 1994 Nov;125(5 Pt 1):812-6. doi: 10.1016/s0022-3476(94)70082-6.
- Background: PubMed 5012243Tompkins DG, Boxerbaum B, Liebman J. Long-term prognosis of rheumatic fever patients receiving regular intramuscular benzathine penicillin. Circulation. 1972 Mar;45(3):543-51. doi: 10.1161/01.cir.45.3.543. No abstract available.
- Background: PubMed 23612950Zhang W, Mondo C, Okello E, Musoke C, Kakande B, Nyakoojo W, Kayima J, Freers J. Presenting features of newly diagnosed rheumatic heart disease patients in Mulago Hospital: a pilot study. Cardiovasc J Afr. 2013 Mar;24(2):28-33. doi: 10.5830/CVJA-2012-076.
- Background: PubMed 23736132Okello E, Wanzhu Z, Musoke C, Twalib A, Kakande B, Lwabi P, Wilson NB, Mondo CK, Odoi-Adome R, Freers J. Cardiovascular complications in newly diagnosed rheumatic heart disease patients at Mulago Hospital, Uganda. Cardiovasc J Afr. 2013 Apr;24(3):80-5. doi: 10.5830/CVJA-2013-004.
- Background: PubMed 22626741Beaton A, Okello E, Lwabi P, Mondo C, McCarter R, Sable C. Echocardiography screening for rheumatic heart disease in Ugandan schoolchildren. Circulation. 2012 Jun 26;125(25):3127-32. doi: 10.1161/CIRCULATIONAHA.112.092312. Epub 2012 May 24.
- Background: PubMed 19557993Steer AC, Kado J, Wilson N, Tuiketei T, Batzloff M, Waqatakirewa L, Mulholland EK, Carapetis JR. High prevalence of rheumatic heart disease by clinical and echocardiographic screening among children in Fiji. J Heart Valve Dis. 2009 May;18(3):327-35; discussion 336.
- Background: PubMed 26076935Engel ME, Haileamlak A, Zuhlke L, Lemmer CE, Nkepu S, van de Wall M, Daniel W, Shung King M, Mayosi BM. Prevalence of rheumatic heart disease in 4720 asymptomatic scholars from South Africa and Ethiopia. Heart. 2015 Sep;101(17):1389-94. doi: 10.1136/heartjnl-2015-307444. Epub 2015 Jun 15.
- Background: PubMed 28285457Zuhlke LJ, Beaton A, Engel ME, Hugo-Hamman CT, Karthikeyan G, Katzenellenbogen JM, Ntusi N, Ralph AP, Saxena A, Smeesters PR, Watkins D, Zilla P, Carapetis J. Group A Streptococcus, Acute Rheumatic Fever and Rheumatic Heart Disease: Epidemiology and Clinical Considerations. Curr Treat Options Cardiovasc Med. 2017 Feb;19(2):15. doi: 10.1007/s11936-017-0513-y.
- Background: PubMed 22371105Remenyi B, Wilson N, Steer A, Ferreira B, Kado J, Kumar K, Lawrenson J, Maguire G, Marijon E, Mirabel M, Mocumbi AO, Mota C, Paar J, Saxena A, Scheel J, Stirling J, Viali S, Balekundri VI, Wheaton G, Zuhlke L, Carapetis J. World Heart Federation criteria for echocardiographic diagnosis of rheumatic heart disease--an evidence-based guideline. Nat Rev Cardiol. 2012 Feb 28;9(5):297-309. doi: 10.1038/nrcardio.2012.7.
- Background: PubMed 25564396Beaton A, Lu JC, Aliku T, Dean P, Gaur L, Weinberg J, Godown J, Lwabi P, Mirembe G, Okello E, Reese A, Shrestha-Astudillo A, Bradley-Hewitt T, Scheel J, Webb C, McCarter R, Ensing G, Sable C. The utility of handheld echocardiography for early rheumatic heart disease diagnosis: a field study. Eur Heart J Cardiovasc Imaging. 2015 May;16(5):475-82. doi: 10.1093/ehjci/jeu296. Epub 2015 Jan 5.
- Background: PubMed 26402451Weinberg J, Beaton A, Aliku T, Lwabi P, Sable C. Prevalence of rheumatic heart disease in African school-aged population: Extrapolation from echocardiography screening using the 2012 World Heart Federation Guidelines. Int J Cardiol. 2016 Jan 1;202:238-9. doi: 10.1016/j.ijcard.2015.08.128. Epub 2015 Sep 12. No abstract available.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
Andrea Beaton, MD principal investigator
Cincinnati Children's
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing intramuscular benzathine penicillin G (BPG) prophylaxis in people with Rheumatic Heart Disease in Children and Latent Rheumatic Heart Disease, run by Children's National Research Institute 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 1 inclusion and 7 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: status not confirmed, as of the sponsor's last update on 1 Oct 2019. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
progression of valvular changes on echocardiogram at 2 years (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03346525, which also lists 27 linked publications.
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 1 Oct 2019.