NCT06113861

Brief Summary

Pediatric tuberculosis (TB) continues to pose diagnostic challenges in low- and middle-income countries with high rates of TB disease, due to the well-described impact of paucibacillary disease in children, and current TB culture and polymerase-chain reaction tests are of limited usefulness due to cost, restricted availability, and poor sensitivity in specimens available from younger children. Our team of experts from Tulane, Johns Hopkins University, Universidad Peruana Cayetano Heredia, and Asociación Benéfica Prisma have confronted all of these challenges through more than 25 years of collaboration in Peru and Bolivia. Our goal is to directly address the challenges of TB in children by evaluating a new diagnostic approach developed by MPI Tony Hu at Tulane University using a CRISPR-mediated TB assay (CRISPR-TB) optimized to detect circulating Mycobacterium tuberculosis cell-free DNA (Mtb-cfDNA), and used to analyze cryopreserved serum in pilot studies from adults and children with presumptive TB, their asymptomatic household contacts, and a cohort of symptomatic children living with HIV (CLHIV) at high risk for TB. Results from symptomatic adult cohorts yielded a pooled sensitivity of 93%; specificity of 93%; positive predictive value of 95%; and negative predictive value of 92%. In limited pilot studies in CLHIV CRISPR-TBD results accurately identified all confirmed TB (13/13) and most children with unconfirmed TB (80%; 52/65). We propose to enroll 200 presumptive TB cases and an equal number of well control subjects in each of 2 study populations (test population and validation population) identified through clinics associated with the "Dr. Mario Ortiz Suarez" Children's Hospital in Santa Cruz, Bolivia. We will determine the distribution of cfDNA concentrations in peripheral blood in a "test population" composed of two age-based groups of children (2 months-6 years, 7-14 years) with respiratory disease grouped by likelihood of TB based on the NIH consensus case definitions (confirmed TB, unconfirmed TB, and unlikely TB) and in age-matched controls grouped by presence of latent TB infection (LTBI), with cfDNA measured serially in time among TB cases receiving antibiotic therapy. We will also validate standard ranges of quantitative cfDNA established for clinical subgroups of children with TB disease or LTBI in an independent validation cohort. An additional aim will determine the correlation between quantitative cfDNA and quantitative imaging-based TB scores based on evidence of disease in the lung, the primary target organ in TB disease, by (1) chest radiograph, measured by computer-aided analysis using the CAD4TB v7 system, and by (2) lung ultrasound, performed with a portable/low-cost probe assisted by machine learning algorithms for automatic interpretation. These biomarkers will be tested as potential cofactors that may be combined with cfDNA levels in peripheral blood, to improve the detection of TB disease in children. The results of this study will be the first step in a process to find a path to allow detection of the many "unconfirmed" TB cases and ideally make the diagnosis of pediatric TB in reach for low resource settings where it is so critically needed.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
1,220

participants targeted

Target at P75+ for all trials

Timeline
29mo left

Started Apr 2024

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Progress46%
Apr 2024Oct 2028

First Submitted

Initial submission to the registry

October 27, 2023

Completed
6 days until next milestone

First Posted

Study publicly available on registry

November 2, 2023

Completed
6 months until next milestone

Study Start

First participant enrolled

April 15, 2024

Completed
3.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 1, 2027

Expected
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2028

Last Updated

September 5, 2025

Status Verified

September 1, 2025

Enrollment Period

3.5 years

First QC Date

October 27, 2023

Last Update Submit

September 3, 2025

Conditions

Keywords

Diagnostic testingChildrenImaging analysis

Outcome Measures

Primary Outcomes (1)

  • Cell free DNA level

    Cell free DNA level

    Baseline and 2 months post treatment for TB cases on therapy

Secondary Outcomes (1)

  • CAD4TB score

    At presentation for case grou

Study Arms (4)

Presumptive pediatric TB cases-- Test population

Inclusion criteria: Children ages 2 months to 14 years with symptomatic disease and suspicion of TB (based on Bolivian Ministry of Health guidelines). Exclusion criteria (prior treatment for TB in 12 mo, current treatment for TB, weight \< 2.5 kg., instability, positive COVID-19 test) Study subgroups are determined in part based on the results of diagnostic tests and on clinical response. A. Confirmed TB * Symptomatic by case definition from the 2015 NIH Expert Panel * Positive culture or positive Xpert MTB/RIF from at least one sample. B. Unconfirmed TB- * Symptomatic and clinical/radiographic evaluation consistent with TB, by case definition from the 2015 NIH Expert Panel * Negative M. tuberculosis culture and negative Xpert MTB/RIF from all samples. C. Unlikely TB--- * Symptomatic BUT clinical/radiographic eval does NOT meet criteria for Unconfirmed TB, per clinical definition-2015 Expert Panel * Negative culture and negative Xpert MTB/RIF from all specimens.

Diagnostic Test: Quantiferon GoldDiagnostic Test: HIV serologyDiagnostic Test: Respiratory secretion culture (sputum or gastric aspirate_Diagnostic Test: Chest radiographDiagnostic Test: Lung ultrasoundDiagnostic Test: cell free DNA test

Control group--Test population

Well Children Control Group-One for each child in groups A-C above, age matched for paired case. * Well children presenting for reasons other than respiratory diseases, and not symptomatic for TB. * Clinical and radiographic evaluation (if performed) NOT suggestive of TB, and NOT diagnosed or treated for TB during the follow-up period (minimum 8 weeks). * Divided into 2 subgroups based on presence or absence of LTBI (by Quantiferon Gold assay).

Diagnostic Test: Quantiferon GoldDiagnostic Test: HIV serologyDiagnostic Test: cell free DNA test

Presumptive pediatric TB cases-- Validation population

Inclusion criteria: Children ages 2 months to 14 years with symptomatic disease and suspicion of TB (based on Bolivian Ministry of Health guidelines). Exclusion criteria (prior treatment for TB in 12 mo, current treatment for TB, weight \< 2.5 kg., instability, positive COVID-19 test) Study subgroups are determined in part based on the results of diagnostic tests and on clinical response. A. Confirmed TB * Symptomatic by case definition from the 2015 NIH Expert Panel * Positive culture or positive Xpert MTB/RIF from at least one sample. B. Unconfirmed TB- * Symptomatic and clinical/radiographic evaluation consistent with TB, by case definition from the 2015 NIH Expert Panel * Negative M. tuberculosis culture and negative Xpert MTB/RIF from all samples. C. Unlikely TB--- * Symptomatic BUT clinical/radiographic eval does NOT meet criteria for Unconfirmed TB, per clinical definition-2015 Expert Panel * Negative culture and negative Xpert MTB/RIF from all specimens.

Diagnostic Test: Quantiferon GoldDiagnostic Test: HIV serologyDiagnostic Test: Respiratory secretion culture (sputum or gastric aspirate_Diagnostic Test: Chest radiographDiagnostic Test: Lung ultrasoundDiagnostic Test: cell free DNA test

Control group--Validation population

Well Children Control Group-One for each child in groups A-C above, age matched for paired case. * Well children presenting for reasons other than respiratory diseases, and not symptomatic for TB. * Clinical and radiographic evaluation (if performed) NOT suggestive of TB, and NOT diagnosed or treated for TB during the follow-up period (minimum 8 weeks). * Divided into 2 subgroups based on presence or absence of LTBI (by Quantiferon Gold assay).

Diagnostic Test: Quantiferon GoldDiagnostic Test: HIV serologyDiagnostic Test: cell free DNA test

Interventions

Quantiferon GoldDIAGNOSTIC_TEST

Test for TB infection

Control group--Test populationControl group--Validation populationPresumptive pediatric TB cases-- Test populationPresumptive pediatric TB cases-- Validation population
HIV serologyDIAGNOSTIC_TEST

Test for HIV infection

Control group--Test populationControl group--Validation populationPresumptive pediatric TB cases-- Test populationPresumptive pediatric TB cases-- Validation population

TB culture by MODS

Presumptive pediatric TB cases-- Test populationPresumptive pediatric TB cases-- Validation population
Chest radiographDIAGNOSTIC_TEST

Chest imaging test by traditional X ray

Also known as: Chest X ray
Presumptive pediatric TB cases-- Test populationPresumptive pediatric TB cases-- Validation population
Lung ultrasoundDIAGNOSTIC_TEST

Chest imaging test by ultrasound

Presumptive pediatric TB cases-- Test populationPresumptive pediatric TB cases-- Validation population
cell free DNA testDIAGNOSTIC_TEST

Blood test for TB infection

Control group--Test populationControl group--Validation populationPresumptive pediatric TB cases-- Test populationPresumptive pediatric TB cases-- Validation population

Eligibility Criteria

Age2 Months - 14 Years
Sexall
Age GroupsChild (0-17)
Sampling MethodNon-Probability Sample
Study Population

Children from clinic population in Santa Cruz Bolivia

You may not qualify if:

  • prior treatment for TB within the past year,
  • current treatment for prevention of TB,
  • weight \< 2.5 kg., or
  • clinical instability,
  • positive COVID-19 diagnostic test

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Hospital de Ninos "Mario Ortiz Suarez"

Santa Cruz, Bolivia

RECRUITING

Biospecimen

Retention: SAMPLES WITH DNA

Plasma samples

MeSH Terms

Conditions

Tuberculosis

Interventions

AIDS SerodiagnosisDiagnostic Imaging

Condition Hierarchy (Ancestors)

Mycobacterium InfectionsActinomycetales InfectionsGram-Positive Bacterial InfectionsBacterial InfectionsBacterial Infections and MycosesInfections

Intervention Hierarchy (Ancestors)

Serologic TestsImmunologic TestsClinical Laboratory TechniquesDiagnostic Techniques and ProceduresDiagnosisHIV TestingMicrobiological TechniquesInvestigative TechniquesImmunologic Techniques

Central Study Contacts

Richard Oberhelman, MD

CONTACT

Study Design

Study Type
observational
Observational Model
CASE CONTROL
Time Perspective
PROSPECTIVE
Target Duration
2 Months
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor

Study Record Dates

First Submitted

October 27, 2023

First Posted

November 2, 2023

Study Start

April 15, 2024

Primary Completion (Estimated)

October 1, 2027

Study Completion (Estimated)

October 1, 2028

Last Updated

September 5, 2025

Record last verified: 2025-09

Locations