MedPath

Novel Triple-dose Tuberculosis Retreatment Regimen

Phase 3
Recruiting
Conditions
Multidrug-resistant Tuberculosis
Resistance to Tuberculostatic Drugs
Pulmonary Tuberculosis
Tuberculosis
Interventions
Drug: 6EHRZ
Drug: 6EH³R³Z
Registration Number
NCT04260477
Lead Sponsor
Institute of Tropical Medicine, Belgium
Brief Summary

To determine if a high-dose first-line regimen is non-inferior (non-inferiority margin 10%) in terms of safety to the same regimen at regular dosing, in previously treated patients with rifampicin-susceptible recurrent Tuberculosis (TB).

Detailed Description

Stage 1: This is a pragmatic open-label multi-stage randomized clinical trial. Potential participants will be screened and enrolled in Damien Foundation (DF) clinics participating in the trial.

First we will perform a two-arm study with 6EHRZ as control arm and 6EH³R³Z as intervention arm. If at interim analysis the intervention arm is not considered to be non-inferior to the control arm, the intervention stops and enrolment will continue in a an adapted intervention arm and the control arm (6EHRZ). Otherwise, enrolment continues to 6EHRZ and 6EH³R³Z.

Observational study (stage 2): The DSMB members agreed due to safety concerns to continuing the study as a cohort with only the control arm. The control regimen will remain the same (6EHRZ).

As per routine practice, during treatment patients are in daily contact with the direct observed therapy (DOT) supervisor and minimally monthly clinic visits are scheduled for monitoring of safety and treatment response.

Additionally, liver function tests will be performed at fixed intervals during treatment. Six month and one year after treatment completion or cure the patient will be checked for relapse with systematic sputum acid-fast bacilli (AFB)-microscopy and TB culture.

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
370
Inclusion Criteria
  • All newly registered patients with smear-positive recurrent pulmonary TB
  • Adults as well as children (no age limit)
  • Able and willing to provide written informed consent
  • Added for stage 2: lives within 5 km of a health facility with a medical doctor
Exclusion Criteria
  • All patients with TB initially resistant to rifampicin on Xpert MTB/RIF testing
  • Patients transferred to a health facility not supported by the Damien Foundation
  • Patients previously enrolled in the trial, and with another episode of rifampicin-susceptible TB during the study period
  • Those with grade III elevation of liver function tests at baseline, or with clinically active liver disease at screening
  • Pregnant or breastfeeding woman
  • HIV co-infected patients requiring treatment with a protease inhibitor

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
6EHRZ6EHRZStandard of care: 6-month 6RHZE regimen with dose combination tablets (one tablet: 150 mg R + 75 mg H + 400 mg Z + 275mg E)
6EH³R3Z6EH³R³Z(Rifampicin (R)/ Isoniazid (H) / Pyrazinamide (Z)/Ethambutol (E)) 6-month high-dose treatment; New high-dose isoniazid / high-dose rifampicin retreatment regimen (6EH³R3Z) - that includes triple-dose rifampicin (R3; 30 mg/kg), and triple-dose isoniazid (H3; 15 mg/kg), complemented with pyrazinamide (Z) and ethambutol (E).
Primary Outcome Measures
NameTimeMethod
STAGE 1:number of patients with any grade 3-5 Adverse Event (AE) during treatment, assessed as probably or definitely related to TB treatment18 months
STAGE 2: Describe bacterial effectiveness18 months

for stage 2 participants of the trial

STAGE 2: Describe acquired resistance18 months

for stage 2 participants of the trial

Secondary Outcome Measures
NameTimeMethod
STAGE 2:number of participants with any TB treatment change due to drug-induced hepatoxicity18 months
STAGE 2:number of participants with any TB treatment change due to AE18 months
STAGE 2:number of participants with any grade 3-5 AE18 months
STAGE 2:number of participants with any Serious Adverse Event (SAE)18 months
STAGE 1:number of participants with any TB treatment change due to AE18 months
STAGE 1:number of participants with any grade 3-5 AE18 months
number of participants with stable (without reversion) SSM conversion2 months

conversion to 0 AFB per field, without subsequent treatment failure

STAGE 1: number of previously treated patients with H-monoresistance and H-polyresistance, rifampicin (RMP) resistance missed by Xpert Mycobacterium tuberculosis (MTB)/rifampicin (RIF)18 months

frequency of initial resistance patterns and mutations conferring resistance

STAGE 1: Programmatical effectiveness:number of participants with treatment success divided by number of participants with failure, death, or Lost to follow-up (LTFU)6 months, 18 months

6 months treatment success: A patient with smear-positive Pulmonary Tuberculosis (PTB) at the beginning of treatment who completed treatment and sputum smear microscopy (SSM) negative in the last month of treatment and on at least one previous occasion or culture-negative at 6 months. A patient with smear-positive PTB at the beginning of treatment who completed treatment without clinical evidence of failure but with no record of sputum smear or culture results in the last month of treatment (either because tests were not done or because results are unavailable) 18 months treatment success: Those who were cured or completed treatment and were evaluated at 12 months post-treatment to be a) SSM negative , or b) SSM positive but culture (CU) negative, or c) without sputum and no clinical signs of TB.

STAGE 1: Clinical effectiveness: number of participants with treatment success, divided by number of participants with failure or death6 months, 18 months

6 months treatment success: A patient with smear-positive PTB at the beginning of treatment who completed treatment and SSM negative in the last month of treatment and on at least one previous occasion or culture-negative at 6 months. A patient with smear-positive PTB at the beginning of treatment who completed treatment without clinical evidence of failure but with no record of sputum smear or culture results in the last month of treatment (either because tests were not done or because results are unavailable) 18 months treatment success: Those who were cured or completed treatment and were evaluated at 12 months post-treatment to be a) SSM negative , or b) SSM positive but CU negative, or c) without sputum and no clinical signs of TB.

STAGE 1:Bacteriological effectiveness : number of participants with treatment success, divided by number of participants with failure6 months, 18 months

6 months treatment success: A patient with smear-positive PTB at the beginning of treatment who completed treatment and SSM negative in the last month of treatment and on at least one previous occasion or culture-negative at 6 months. A patient with smear-positive PTB at the beginning of treatment who completed treatment without clinical evidence of failure but with no record of sputum smear or culture results in the last month of treatment (either because tests were not done or because results are unavailable) 18 months treatment success: Those who were cured or completed treatment and were evaluated at 12 months post-treatment to be a) SSM negative , or b) SSM positive but CU negative, or c) without sputum and no clinical signs of TB.

STAGE 2:Bacteriological effectiveness : number of participants with treatment success, divided by number of participants with failure6 months, 18 months

6 months treatment success: A patient with smear-positive PTB at the beginning of treatment who completed treatment and SSM negative in the last month of treatment and on at least one previous occasion or culture-negative at 6 months. A patient with smear-positive PTB at the beginning of treatment who completed treatment without clinical evidence of failure but with no record of sputum smear or culture results in the last month of treatment (either because tests were not done or because results are unavailable) 18 months treatment success: Those who were cured or completed treatment and were evaluated at 12 months post-treatment to be a) SSM negative , or b) SSM positive but CU negative, or c) without sputum and no clinical signs of TB.

STAGE 1:number of participants with any TB treatment change due to drug-induced hepatoxicity18 months
STAGE 2: number of participants with acquired resistance, to Isoniazid (INH) and/or RMP6 months, 18 months

In patients with recurrence, the recurrent strain will be compared with the diagnostic strain to identify possible differences in the resistance pattern. If the strain is the same, and resistance is not present in the diagnostic sample but is identified in the recurrence sample, then this resistance is considered as acquired. Resistance is defined as a) on genotypic Drug susceptibility testing (DST) (Deeplex or other): presence of a mutation in the resistance determining region for the drug with exception of generally recognized polymorphisms and silent mutations not leading to an error in the gene product. Heteroresistance at the proportion detectable by these methods will be considered at par with full-blown resistance, or b) growth at the critical concentration for the drug tested in phenotypic DST.

STAGE 1: number of participants with drug-induced hepatotoxicity18 months

hepatotoxicity due to anti-TB drug treatment was defined as the following criteria:

1. Grade 3-5 elevation of Liver function test (LFT), or grade 2 elevation of liver function tests with jaundice; AND

2. absence of serological evidence of infection with hepatitis B or C, AND

3. normalization or at least a 50% improvement in abnormal liver chemistry results after withdrawal of anti-TB drugs

STAGE 1:number of participants with any Serious Adverse Event (SAE)18 months
STAGE 2: number of participants with drug-induced hepatotoxicity18 months

hepatotoxicity due to anti-TB drug treatment was defined as the following criteria:

1. Grade 3-5 elevation of Liver function test (LFT), or grade 2 elevation of liver function tests with jaundice; AND

2. absence of serological evidence of infection with hepatitis B or C, AND

3. normalization or at least a 50% improvement in abnormal liver chemistry results after withdrawal of anti-TB drugs

Trial Locations

Locations (1)

Damien Foundation

🇳🇪

Niamey, Niger

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