A Prospective Patient Registry of Patients Exposed to Bedaquiline

NCT02274389 | Completed FDA Drug

• 3 other identifiers: CR104620RRA-12028TMC207TBC4001
• Study Type: observational
• Target: 5
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Pulmonary Multi-drug Resistant Tuberculosis

Keywords

Pulmonary Multi-drug Resistant Tuberculosis; Bedaquiline; Sirturo; Observational; Prospective

Outcome Measures

Primary Outcomes (7)

• 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 Outcomes (1)

• Comparison of Rate of Observed Outcomes With Nationally Available Reported Rates

  Baseline up to Month 36

Study Arms (1)

Bedaquiline Patient Registry (BPR)

Drug: No Intervention

Interventions

No Intervention DRUG

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.

Bedaquiline Patient Registry (BPR)

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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

Sponsors & Collaborators

• Janssen Research & Development, LLC: lead

Study Sites (1)

Unknown Facility

Raleigh, North Carolina, United States

Location

Study Officials

• Janssen Research & Development, LLC Clinical Trial

  Janssen Research & Development, LLC

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 22, 2014
• First Posted: October 24, 2014
• Study Start: December 24, 2013
• Primary Completion: November 20, 2018
• Study Completion: November 20, 2018
• Last Updated: June 26, 2019

Record last verified: 2019-06

Locations

US (1)