A Long-term Follow up Study for Patients Who Participated in the GOAL Trial (GOAL-Post)

Completed

• Conditions: Rheumatic Heart Disease
• Interventions: Drug: Penicillin G Benzathine

• Registration Number: NCT05211024
• Lead Sponsor: Children's Hospital Medical Center, Cincinnati

Brief Summary

GOAL-Post is comprised of two studies that will help refine recommendations for the duration of secondary prophylaxis for children diagnosed with latent RHD, which is currently not known

Detailed Description

GOAL-Post is comprised of two studies that will help refine recommendations for the duration of secondary prophylaxis for children diagnosed with latent RHD, which is currently not known.

Aim 1: Compare the two-year risk of developing RHD (borderline or definite) between children and adolescents who completed the GOAL Trial with a normal echocardiogram (prior diagnosis of latent RHD) and age/sex/and geographically matched controls with repeated normal echocardiograms (normal in both the original GOAL screening in 2017/2018 and in the planned GOAL-Post screening in 2021).

Aim 2: Determine the five-year rate of RHD progression and regression among children with persistent latent RHD who receive secondary antibiotic prophylaxis (medium-term impact of prophylaxis). Five years includes time from initial GOAL enrollment to the end of GOAL-Post.

Aim 3: Create a RHD biobank that will support further research on RHD genetic susceptibility and pathophysiology

Study Timeline

• Study First Submitted: 2022-01-14
• Study First Submitted QC: 2022-01-14
• Study First Posted: 2022-01-27
• Study Start: 2022-01-28
• Primary Completion: 2024-03-30
• Study Completion: 2024-04-30
• Status Verified: 2024-08
• Last Update Submitted: 2024-08-23
• Last Update Posted: 2024-08-27

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 1423

Inclusion Criteria

Aim 1: Group A: Children and adolescents will be eligible for Aim 1 if they (1) are a prior GOAL participant deemed by the adjudication panel to have a normal echocardiogram at the 2-year endpoint, (2) are not receiving secondary antibiotic prophylaxis, and (3) agree to participate in the study via the study's informed consent/assent process.

Group B: Children and adolescents will be eligible for Aim 1 if they (1) have a normal echocardiogram at the start of the study, (2) Meet the age/sex/geographic match requirement (from former GOAL participants), and (3) have agreed to participate in the study via the study's informed consent/assent process.

Aim 2: Children and adolescents will be eligible for Aim 2 if they (1) are a prior GOAL participant deemed by the adjudication panel to have persistent latent RHD on echocardiogram at the 2-year endpoint, (2) have been prescribed secondary antibiotic prophylaxis, and (3) agree to participate in the study via the study's informed consent/assent process.

Aim 3: Children and adolescents will be eligible for Aim 3 if they are prior GOAL participants deemed by the adjudication panel to (1) have a normal echocardiogram at the 2-year endpoint (Aim 1 participants), (2) deemed by the adjudication panel to have persistent latent RHD at the 2-year endpoint (Aim 2 participants), or (3) deemed by the adjudication panel to have moderate/severe RHD at the 2-year endpoint. Aim 3 will include a separate consent/assent and participants will be able to participate in each aim independently.

Exclusion Criteria

Aim 1: Group A: Residence or school is no longer in Gulu District or one of the surrounding districts.

Group B: Known history of ARF or RHD, or evidence of RHD on baseline echocardiogram or 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).

Aim 2 + 3: Residence or school is no longer in Gulu District or one of the surrounding districts.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Aim 2	Penicillin G Benzathine	Children and adolescent who are prior GOAL participants who have persistent latent RHD on echocardiogram at the 2 year endpoint.

Primary Outcome Measures

Name	Time	Method
Regression	2 year endpoint	Regression of echocardiographic features of latent RHD
Progression	2 year endpoint	Progression of echocardiographic features of latent RHD to borderline to definite, or definite to mild or definitive to moderate/severe

Trial Locations

Locations (1)

Uganda Heart Institute; 🇺🇬 Kampala, Uganda