A Prospective Patient Registry of Patients Exposed to Bedaquiline

Bedaquiline Patient Registry: Prospective Patient Registry of Patients Exposed to Bedaquiline In the United States

The purpose of this study is to describe the medical indication and utilization of expert medical consultation among participants treated with bedaquiline (BDQ), BDQ susceptibility based on minimum inhibitory concentrations (MICs) reported for baseline and subsequent isolates, BDQ drug utilization data to include dose, duration, past treatment history, past medical history, concomitant medications, and health care site of treatment, drug distribution mechanisms used in the administration of BDQ, patient outcomes (clinical and microbiologic) and adverse events among BDQ-treated participants, including deaths.

Study Overview

• Status: Completed
• Conditions: Pulmonary Multi-drug Resistant Tuberculosis
• Intervention / Treatment: Drug: No Intervention

Detailed Description

This is a prospective (observation of a population for a sufficient number of persons over a sufficient number of years to generate incidence or mortality rates subsequent to the selection of the study group) observational (clinical study in which participants may receive diagnostic, therapeutic, or other types of interventions, but the investigator does not assign participants to specific interventions) study of participants treated with BDQ. Participants will be enrolled by their healthcare provider once the healthcare provider decides to treat with BDQ or after BDQ treatment initiation but prior to observation of any participant outcomes. All participants meeting the inclusion criteria and providing informed consent for participation in the study will be enrolled in this study. The registry enrollment period will be open for 36 months with an additional 24 months BDQ treatment-free follow-up for safety assessment for the last participants enrolled during the 36 months enrollment period. Participants' follow-up data will be collected at each visit from the healthcare provider during the course of BDQ and for an additional 24 months after the last BDQ dose or until the participant is lost to follow-up. Participants' safety will be monitored throughout the study.

• Study Type: Observational
• Enrollment (Actual): 5

Contacts and Locations

Study Locations

• United States
  • North Carolina
    • Raleigh, North Carolina, United States

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Study population will be drawn from the population of all partIcipants treated with bedaquiline (BDQ) in the United States. All participants meeting registry inclusion criteria will be eligible for inclusion in the BDQ patient registry provided they submit the necessary informed consent for participation.

Description

Inclusion Criteria:

• Participants capable of providing informed consent, which must be obtained prior to collection of any patient outcomes and documented laboratory and clinical variables to meet the study objectives
• Participants who have documented enrollment in the bedaquiline patient registry (BPR) prior to the start of BDQ treatments and documented initiation of BDQ treatment after enrollment
• Participants who have documented initiation of BDQ treatment prior to enrollment and have no documented patient outcomes and documented laboratory and clinical variables to meet the objectives of the study

Exclusion Criteria:

• No specific exclusion criteria will be applied in this study other than those scenarios that follow from the inclusion criteria

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Bedaquiline Patient Registry (BPR)	Drug: No Intervention; This is an observational study and participants did not receive any intervention in this study. Participants initiating bedaquiline (BDQ) treatment as decided by healthcare provider, will be prospectively observed.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Participants with Medical Indication for Bedaquiline (BDQ) Treatment and Frequency of use of Expert Medical Consultation With BDQ	Indication for a drug refers to the use of that drug for treating a particular disease. Percentage of participants treated using expert medical consultation will be summarized. Percentage of participants with medical indication for BDQ treatment as reported by the healthcare provider and frequency of use of expert medical consultation in multi-drug resistant tuberculosis (MDR-TB) treatment with BDQ will be reported.	Baseline up to 24 months after last BDQ dose
Number of Participants With BDQ Susceptibility Based on Minimum Inhibitory Concentration (MIC)	The MIC is the lowest concentration of an antimicrobial that will inhibit the visible growth of a microorganism after overnight incubation. Number of participants whose post-enrollment isolates exhibit at least 4-fold increase in BDQ MIC in comparison to baseline MIC, provided that the post-baseline MIC is greater than 0.25 milligram per milliliter (mg/mL) based on phenotypic methods on solid or liquid media.	Baseline up to 24 months after last BDQ dose
Maximum Dose of BDQ	Maximum dose of BDQ used will be reported.	Baseline up to Month 12
Total Duration of BDQ Treatment	Exposure to BDQ will be determined by documentation by the healthcare provider. Duration of BDQ treatment will be calculated from the initiation and discontinuation dates for BDQ. The initiation date for BDQ drug for each participant will be the drug initiation date as reported by the healthcare provider. The discontinuation date will be defined as the date the drug was stopped as reported by the healthcare provider.	Baseline up to Month 12
Percentage of Participants with Different Drug Distribution Mechanisms Utilized for Drug Administration	Directly observed therapy short-course (DOTS) combines 5 elements: political commitment; microscopy services; drug supplies; surveillance and monitoring systems; and use of highly efficacious regimens with direct observation of treatment. Percentage of participants with drug distribution mechanisms used and DOT use for drug administration will be reported.	Baseline up to Month 12
Number of Participants With Clinical and Microbiologic Patient Reported Outcomes	The following treatment outcomes will be estimated separately for participants with MDR-TB and participants with non MDR cases or MDR status not documented as per World Health Organization (WHO) Treatment of tuberculosis guidelines: cured, treatment completed, treatment failed, died, lost to follow-up, not evaluated, and treatment success.	Baseline up to 24 months after last BDQ dose
Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)	An AE is any untoward medical occurrence in a clinical study participant administered a medicinal (investigational or non investigational) product and does not necessarily have a causal relationship with the treatment. An SAE is any untoward medical occurrence that meets any of the following conditions: results in death, is life threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, or is a congenital anomaly/birth defect.	Baseline up to 24 months after last BDQ dose

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Comparison of Rate of Observed Outcomes With Nationally Available Reported Rates	Treatment outcome will be evaluated on basis of following parameters; Treatment Completed:without evidence of failure BUT no record that three or more consecutive cultures taken at least 30 days apart are negative after intensive phase; Treatment Failed:Treatment terminated or need for permanent regimen change of at least two anti-tuberculosis drugs because of: Lack of conversion by the end of the intensive phase, or Bacteriological reversion in the continuation phase after conversion to negative, or Evidence of additional acquired resistance to fluoroquinolones or second-line injectable drugs, or Adverse drug reactions; Died:participant who dies for any reason during course of treatment; Lost to Follow-Up:whose treatment was interrupted for 2 consecutive months or more; Not Evaluated:whom no treatment outcome is assigned (this includes cases "transferred out" to another treatment unit and whose treatment outcome is unknown); Treatment Success:The sum of cured and treatment completed.	Baseline up to Month 36

Collaborators and Investigators

• Sponsor: Janssen Research & Development, LLC

Study record dates

Study Major Dates

• Study Start (Actual): December 24, 2013
• Primary Completion (Actual): November 20, 2018
• Study Completion (Actual): November 20, 2018

Study Registration Dates

• First Submitted: October 22, 2014
• First Submitted That Met QC Criteria: October 22, 2014
• First Posted (Estimate): October 24, 2014

Study Record Updates

• Last Update Posted (Actual): June 26, 2019
• Last Update Submitted That Met QC Criteria: June 25, 2019
• Last Verified: June 1, 2019

More Information

Terms related to this study

• Keywords: Observational; Prospective; Bedaquiline; Sirturo; Pulmonary Multi-drug Resistant Tuberculosis
• Additional Relevant MeSH Terms: Infections; Bacterial Infections; Bacterial Infections and Mycoses; Gram-Positive Bacterial Infections; Actinomycetales Infections; Mycobacterium Infections; Tuberculosis; Tuberculosis, Multidrug-Resistant
• Other Study ID Numbers: CR104620; RRA-12028 (Other Identifier: Janssen Research and Development, LLC); TMC207TBC4001 (Other Identifier: Janssen Research and Development, LLC)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes