A Phase 2 interventional study of phenoxymethyl penicillin and intramuscular benzathine penicillin G (BPG) prophylaxis in Rheumatic Heart Disease, sponsored by Children's Hospital Medical Center, Cincinnati. Active, not recruiting at 1 site in Uganda. Open to participants aged 5 Years to 17 Years. Per ClinicalTrials.gov, last updated 2026-06-22.
Sponsored by Children's Hospital Medical Center, Cincinnati · Phase 2, Interventional, and Treatment
The goal of this clinical trial is to determine if twice daily oral penicillin prophylaxis is non-inferior to monthly IM penicillin prophylaxis in preventing latent Rheumatic Heart Disease Progression in children between the ages of 5-17 years. The main objective is to compare the proportion of children aged 5-17 years with latent RHD receiving oral penicillin prophylaxis who progress to worse valvular disease at 2-years compared to children who receive IM penicillin prophylaxis.
GOALIS is a randomized controlled trial developed to provide high quality contemporary evidence on the efficacy of oral penicillin as RHD prophylaxis.
Aim 1: To compare the proportion of children aged 5-17 years with latent RHD receiving oral penicillin prophylaxis who progress to worse valvular disease at 2-years compared to children who receive IM penicillin prophylaxis.
Aim 2: To evaluate the economic equivalence and cost-effectiveness of oral penicillin compared to IM penicillin, after echocardiographic screening for latent RHD detection.
Aim 3: Compare patient-reported outcomes (treatment acceptance, treatment satisfaction, and health-related quality of life) between children receiving oral and IM penicillin prophylaxis.
59 studies on the registry are indexed under Rheumatic Heart Disease; 20 are open to participants now.
This study's planned enrollment of 1,004 is above the median of 221 across 35 interventional studies indexed under Rheumatic Heart Disease.
Browse Rheumatic Heart Disease studies →Children's Hospital Medical Center, Cincinnati is the lead sponsor of 661 studies on the registry; 134 are open to participants now.
Of its 54 completed or terminated interventional studies of FDA-regulated products, 30 (56%) have results posted.
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Operational Definition of Latent RHD Borderline RHD or Mild Definite RHD (to include no more than mild regurgitation at the mitral or aortic valve, normal mean mitral and aortic valve gradients, normal bi-ventricular function) according to the 2012 WHF consensus criteria.
Exclusion Criteria:
Oral phenoxymethyl penicillin (Pen V) prophylaxis 250mg twice daily.
Drug: phenoxymethyl penicillin
Intramuscular benzathine benzylpenicillin G (BPG) prophylaxis (600,000 IU for children \<30kg, 1.2 million IU for children ≥30kg), every 28 days
Drug: intramuscular benzathine penicillin G (BPG) prophylaxis
Prophylaxis
Also known as: Pen V
Prophylaxis
Also known as: benzathine penicillin G, BPG, penicillin prophylaxis
Progression
Progression of echocardiographic features of latent RHD to borderline to definite, or definite to mild or definite to moderate/severe
Time frame: 2 year endpoint
Demonstrate non-inferior costs and cost-effectiveness of oral penicillin compared to IM penicillin
The aim is to understand the costs and effectiveness of the intervention. Costs will include intervention and disease specific costs, capturing both health system and family out of pocket costs. Participants will complete a questionnaire asking about out-of-pocket cost (medications, supplies, consultation fees) and lost productivity (including missed school and workdays) due to the intervention. Costs will be measured in the local currency (Uganda Shillings) and presented in United States dollars (USD)
Time frame: baseline, 6, 12, 18, 24 months post randomization
Obtain cost data associated with the receipt of oral or IM penicillin in the community from a healthcare provider perspective.
In order to understand health center and community costs, 10 randomly selected health care facilities (including level II, III, and IV facilities) will be surveyed to obtain direct costs of medications, supplies as well as any administration and professional fees associated with outpatient and inpatient visits.
Time frame: 2 year endpoint
Treatment Acceptance
Treatment acceptance will be assessed with the widely-used Chronic Treatment Acceptance (ACCEPT) questionnaire, a generic medication acceptance instrument validated for chronic conditions. This 25-item survey consists of six areas of focus: medication convenience (3 items), length of treatment (2 items), medication constraints (9 items), medication side effects (5 items), medication effectiveness (3 items), medication advantages (3 items). Responses are rated on a 5-point Likert scale and scores of 1 through 4 indicating increasing levels of acceptance.
Time frame: baseline, 6, 12, 18, 24 months post randomization
Treatment Satisfaction
Treatment Satisfaction will be assessed with the 14-item Abbreviated Treatment Satisfaction Questionnaire (v 1.4). This widely used survey contains 4 areas of focus including satisfaction of treatment effectiveness (3 items), satisfaction with treatment side effects (5 items), satisfaction with treatment convenience (3 items), and a global treatment satisfaction (3 items). Scores are then transformed to a score of 0-100 to create the overall scale score and the subcategory scores, with higher scores representing higher treatment satisfaction on that domain.
Time frame: baseline, 6, 12, 18, 24 months post randomization
Health-Related Quality of Life
Health Related Quality of Life will be measured using the Pediatric Quality of Life Inventory Version 4.0 Generic Core (PedsQL 4.0) questionnaire. The PedsQLTM4.0 is a 23-item survey for children aged 5-18 which assesses HRQOL. It has four areas of focus: Physical functioning (8 items), emotional functioning (5 items), social functioning (5 items), school functioning (5 items). Each item has five possible responses measuring the extent to which the item was a problem during the past month. The possible responses are measured using a 5-point Likert scale from 0=never a problem to 4 = almost always a problem. These reverse scaled scores are then transformed to a score of 0-100 to create the overall scaled score and the subcategory scores, with higher scores representing better quality of life.
Time frame: baseline, 6, 12, 18, 24 months post randomization
Patient-reported outcomes
Interviews will be used to collect qualitative data around participants perceptions of the impact of RHD and its treatment on their activities of daily living and quality of life including experiences with IM or oral penicillin prophylaxis.
Time frame: baseline, 6, 12, 18, 24 months post randomization
This study is active, not recruiting, as verified in Jun 2026. You cannot join it, but the record below documents what was studied.
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Children's Hospital Medical Center, Cincinnati