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

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

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

Study Overview

• Status: Active, not recruiting
• Conditions: Rheumatic Heart Disease
• Intervention / Treatment: Drug: Penicillin G Benzathine

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 Type: Observational
• Enrollment (Anticipated): 1102

Contacts and Locations

• Study Contact: Name: Mary Banks, MS; Email: mary.banks@cchmc.org

Study Locations

• Uganda
  • Kampala, Uganda
    • Uganda Heart Institute

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years to 17 years (Child)
• Accepts Healthy Volunteers: Yes

• Sampling Method: Probability Sample

Study Population

Aim 1 will enroll with normal echocardiograms after 2-years of participation in the GOAL Trial (Group A) and an appropriate number of age/sex matched controls (Group B) without echocardiographic evidence of RHD.

Aim 2 will enroll participants with persistent latent RHD after 2 years of participation in the GOAL Trial. (Fig. 2).

Description

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 Plan

How is the study designed?

Number of groups / cohorts

3

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Aim 1: Group A; Children and adolescent who are prior GOAL participants who have a normal echocardiogram at the 2 year endpoint and are no receiving secondary antibiotic prophylaxis
Aim 1 Group B; Children and adolescent who have a normal echocardiogram at the start of the study
Aim 2; Children and adolescent who are prior GOAL participants who have persistent latent RHD on echocardiogram at the 2 year endpoint.	Drug: Penicillin G Benzathine; Participants in Aim 2 are receiving 28 days interval BPG intramuscular injection as part of standard of care

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Progression	Progression of echocardiographic features of latent RHD to borderline to definite, or definite to mild or definitive to moderate/severe	2 year endpoint
Regression	Regression of echocardiographic features of latent RHD	2 year endpoint

Collaborators and Investigators

• Sponsor: Children's Hospital Medical Center, Cincinnati
• Collaborators: Uganda Heart Institute; Murdoch Children's Research Institute, Melbourne Australia

Study record dates

Study Major Dates

• Study Start (Actual): January 28, 2022
• Primary Completion (Anticipated): February 1, 2024
• Study Completion (Anticipated): June 1, 2024

Study Registration Dates

• First Submitted: January 14, 2022
• First Submitted That Met QC Criteria: January 14, 2022
• First Posted (Actual): January 27, 2022

Study Record Updates

• Last Update Posted (Actual): May 8, 2023
• Last Update Submitted That Met QC Criteria: May 5, 2023
• Last Verified: May 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cardiovascular Diseases; Infections; Bacterial Infections; Bacterial Infections and Mycoses; Streptococcal Infections; Gram-Positive Bacterial Infections; Rheumatic Fever; Heart Diseases; Rheumatic Heart Disease; Anti-Infective Agents; Anti-Bacterial Agents; Penicillins; Penicillin G Benzathine; Penicillin G; Penicillin G Procaine
• Other Study ID Numbers: 2021-0451

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No