A Multi-Country Prospective Multi-Drug Resistant Tuberculosis Patient Registry to Monitor Bedaquiline Safety, Utilization, and Emergence of Resistance

Study type

• Study topic:
  Human medicinal product

  Disease /health condition
• Study type:
  Non-interventional study
• Scope of the study:
  Disease epidemiology

  Drug utilisation
• Data collection methods:
  Combined primary and secondary data collection

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  SIRTURO
• Medical condition to be studied:
  Multiple-drug resistance

  Pulmonary tuberculosis

Population studied

• Short description of the study population:
  Inclusion Criteria; The study population will be drawn from the population of all patients treated for MDR-TB in multiple countries. All patients newly diagnosed with MDR-TB as well as all patients newly treated with BDQ at participating sites will be eligible for inclusion in the registry. All patients meeting registry inclusion criteria will be eligible for inclusion in the registry provided they submit the necessary informed consent for participation.; ; Exclusion Criteria; No specific exclusion criteria will be applied in this study other than those scenarios that follow from the inclusion criteria.
• Age groups:
  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Special population of interest:
  Other
• Special population of interest, other:
  Pulmonary tuberculosis patients
• Estimated number of subjects:
  6000

Study design details

• Main study objective:
  To further assess the benefits and risks of BDQ by evaluating BDQ safety, effectiveness, and emergence of resistance, BDQ drug utilization, and adherence to WHO guidance on the use of BDQ in MDR-TB treatment.
• Outcomes:
  The registry is designed to observe several aspects of MDR-TB treatment such as drug utilization characteristics, treatment outcomes (clinical and microbiologic), and safety outcomes (adverse events and mortality).
• Data analysis plan:
  Descriptive analyses will be used to describe baseline characteristics including demographics, country of origin, medical history, susceptibility of baseline MDR-TB isolates to BDQ and TB background regimen drugs, and use of medications at cohort entry. MDR-TB treatment will also be described including indication of use, dose, frequency, duration of use, usage with TB background regimen, and susceptibility of concomitant TB treatment regimens. Relative risk rates and 95% confidence intervals will be calculated and appropriate stratified analyses will be conducted for each adverse event, including death. Adjustments for imbalances between the treatment groups will be applied using accepted methods based on sample size availability and the observed overlap of key patient characteristics.

Documents

Study publications

Pai H, Ndjeka N, Mbuagbaw L, Kaniga K, et al. Bedaquiline safety, efficacy, uti…

Shim TS, Pai H, Mok J, et al. A prospective patient registry to monitor safety,…