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NexGen EBA Radiologic and Immunologic Biomarkers of Sterilizing Drug Activity in Tuberculosis

Phase 2
Completed
Conditions
Pulmonary Tuberculosis
Interventions
Drug: Treatment
Radiation: PET/CT Scan
Procedure: Sample Collection
Registration Number
NCT02371681
Lead Sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
Brief Summary

Background:

- Tuberculosis (TB) is a lung infection caused by bacteria. When people with TB cough, they may spread these bacteria. Researchers are looking for new TB medicines. They want to find a faster way to tell if a drug might combat TB.

Objective:

- To learn the effect of different anti-TB drugs on microbiological, radiographic and immunologic markers in people with TB.

Eligibility:

- Adults age 18-65 who weigh 30-90 kg and have common TB bacteria that can be treated with common TB medicines.

Design:

* Participants will be admitted to the hospital for screening. They will have medical history, physical exam, and chest radiograph. They will give blood, urine, and sputum samples.

* Participants will be put in 1 of 8 groups.

* Participants will get one or a combination of TB medicines daily for about 14 days.

* Each day, participants:

* Will discuss side effects.

* May have a physical exam.

* Will spit mucus into a cup. They may breathe in saline water through a nebulizer to make them cough.

* Participants will have blood taken 3-4 times during the study

* Participants will have 2-3 Fluorodeoxyglucose Positron Emission Tomography/Computed Tomography (FDG-PET/CT) scans. FDG is a radioactive sugar molecule which helps measure TB disease in the lungs. It will be injected into a vein. Participants will lie in a scanner that takes pictures.

* Around study day 14, participants will leave the hospital. They will be referred to a local TB clinic. There they will get the standard 4 TB medicines. Those in group 8 will already be on these medicines and will have another FDG-PET/CT on day 28.

* Participants will be in the study for up to 28 days.

Detailed Description

Early bactericidal activity (EBA), which measures decline in serial sputum colony forming unit (CFU) counts over the first 2-14 days of treatment, has been used extensively as a means of initially evaluating the potency of individual or combinations of antituberculous agents. This approach is endorsed by the Global Alliance for TB Drug Development and the US FDA. However, EBA seems to correlate poorly with the relative ability of an agent to prevent relapse and produce a durable cure (often referred to as sterilizing activity ). The reasons for this discrepancy may have to do with a limitation of sputum measurements to capture populations that persist beyond airway surfaces in discrete lesions such as granulomas, nodules, or cavities. The elimination of these persistent populations depend on the pharmacodynamic properties of a regimen and may be better captured by biologic and functional markers that can reflect dynamic treatment effects within these relevant host environments.

Recent studies of the response to TB chemotherapy have identified promising new biomarkers of sterilization in 2 areas. First, immunologic changes appear to have potential in small subject cohorts to predict sterilizing cure within 1 month after commencing treatment. Second, 18F-FDG PET/CT has been used in tuberculosis as a qualitative means of assessing drug response in small case series at multiple time points, starting as early as 1 month. PET activity reflects uptake and phosphorylation of FDG by neutrophils and macrophages, and CT provides structural information on disease pathology. Hence, PET/CT data may offer additional insights into lesion-specific sterilizing activity. This study will add 18F-FDG PET/CT scans and immunological assays at 0, 2, and (in the HRZE arm) 4 weeks to standard EBA methodology using regimens containing isoniazid (INH \[H\]), rifampin (RIF \[R\]), pyrazinamide (PZA \[Z\]), moxifloxacin (MXF \[M\]), and ethambutol (EMB \[E\]). We hypothesize that drug regimens associated with higher sterilizing activity (e.g., containing rifampin or pyrazinamide) will show distinctive early cytokine and chemokine patterns and discrete, quantifiable changes on PET/CT in certain lesion types during the 2-week period, compared to drug regimens with poor sterilizing activity (e.g., containing isoniazid or moxifloxacin). Demonstration of such an association would provide rationale for including radiologic and immunologic analysis, alongside conventional EBA, in early phase clinical studies of novel drugs, and would also provide important new insights into the biology of human and bacterial responses to TB drugs.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
262
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
1TreatmentTB drugs
1PET/CT ScanTB drugs
1Sample CollectionTB drugs
Primary Outcome Measures
NameTimeMethod
To characterize, in the context of a standard EBA study, the effect of various antituberculosis drugs on radiographic and immunologic markers as measured by PET/CT and immunologic assays, in subjects with drug sensitive pulmonary tuberculosis wh...14 days

A description of the individual markers that change over time is of interest to better understand both the markers and the effects of each treatment. A second analysis will focus on classification of whether a treatment arm includes: 1) only one agent ( singlet ), 2) only two agents (a doublet ), or 3) four agents (a quadruplet ).

Secondary Outcome Measures
NameTimeMethod
Rank order of drugs14 days

Comparison of the rank order of drugs based upon bacteriologic, radiologic and immunologic features.

PET/CT Changes14 days

Correlation of PET/CT changes with treatment response, microbiologic and immunologic outcomes

Trial Locations

Locations (2)

Stellenbosch University, Faculty of Medicine and Health Sciences

🇿🇦

Cape Town, South Africa

TASK Applied Sciences

🇿🇦

Cape Town, South Africa

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