Clinical trial NCT02274389: A Prospective Patient Registry of Patients Exposed to Bedaquiline
Official title: Bedaquiline Patient Registry: Prospective Patient Registry of Patients Exposed to Bedaquiline In the United States
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Pulmonary Multi-drug Resistant Tuberculosis
Keywords: Pulmonary Multi-drug Resistant Tuberculosis
Bedaquiline
Sirturo
Observational
Prospective
Lead sponsor: Janssen Research & Development, LLC
Sponsor class: INDUSTRY
First posted: 2014-10-24
Enrollment: 5
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

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.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Percentage of Participants with Medical Indication for Bedaquiline (BDQ) Treatment and Frequency of use of Expert Medical Consultation With BDQ
timeFrame: Baseline up to 24 months after last BDQ dose
measure: Number of Participants With BDQ Susceptibility Based on Minimum Inhibitory Concentration (MIC)
timeFrame: Baseline up to 24 months after last BDQ dose
measure: Maximum Dose of BDQ
timeFrame: Baseline up to Month 12
measure: Total Duration of BDQ Treatment
timeFrame: Baseline up to Month 12
measure: Percentage of Participants with Different Drug Distribution Mechanisms Utilized for Drug Administration
timeFrame: Baseline up to Month 12
measure: Number of Participants With Clinical and Microbiologic Patient Reported Outcomes
timeFrame: Baseline up to 24 months after last BDQ dose
measure: Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: Baseline up to 24 months after last BDQ dose
Secondary outcomes:
measure: Comparison of Rate of Observed Outcomes With Nationally Available Reported Rates
timeFrame: Baseline up to Month 36
Other outcomes:
Locations:
city: Raleigh
country: United States
state: North Carolina
Study officials:
affiliation: Janssen Research & Development, LLC
name: Janssen Research & Development, LLC Clinical Trial
role: STUDY_DIRECTOR
References: