Clinical trial NCT04579211: Prospective Analysis of Urine LAM to Eliminate NTM Sputum Screening
Official title: Prospective Analysis of Urine LAM to Eliminate NTM Sputum Screening (PAINLESS Trial)
Study type: OBSERVATIONAL
Overall status: ENROLLING_BY_INVITATION
Conditions: Cystic Fibrosis
Lead sponsor: National Jewish Health
Sponsor class: OTHER
First posted: 2020-10-08
Enrollment: 220
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: 100 adult male or female participants with a diagnosis of CF and NTM culture status of negative and no known previous positive cultures for pathogenetic NTM

Brief summary: This is a prospective, single-center, nonrandomized observational study to investigate the utility of urine lipoarabinomannan (LAM) as a test to identify individuals at very low risk for having a sputum culture positive for Nontuberculous Mycobacteria (NTM). The study is designed to evaluate if a urine test can eliminate the need for obtaining a sputum specimen to screen for NTM in individuals with Cystic Fibrosis (CF). The participants will be asked to provide 3 urine samples either in person or by mail over approximately 3 years. The 3 urine sample requests will be timed to coincide with their usual clinical care and routine sputum collection.


Eligibility criteria: Inclusion Criteria:

* Informed consent obtained online or in person from the participant
* Enrolled in the CFF Patient Registry (CFFPR)
* Be willing and able to adhere to study procedures in the context of clinical care, and other protocol requirements
* Diagnosis of CF consistent with the 2017 CFF Guidelines NTM culture status of negative (defined by a review of clinical and culture data with at least 3 years of clinical data available to review and at least one negative culture within the year prior to enrollment and no known history of previous positive cultures for pathogenic NTM)

Exclusion Criteria:

1. Has any other condition that, in the opinion of the Site Investigator/designee, would preclude informed consent or assent, make study participation unsafe, complicate interpretation of study outcome data, or otherwise interfere with achieving the study objectives

Primary outcomes:
measure: To determine the ability of the urine LAM assay to predict continued negative sputum culture over the next 12 months.
timeFrame: From time enrollment to over the next 12 months
Secondary outcomes:
measure: To determine the ability of the urine LAM assay to predict new positive NTM sputum culture over the next 12 months.
timeFrame: From time of enrollment to over the next 12 months
measure: To determine the time between a positive LAM assay and a new positive NTM culture.
timeFrame: 3 years
measure: To facilitate the development of a ELISA-based assay as a possible replacement to GC/MS in the clinical setting.
timeFrame: 3 years
Other outcomes:
Locations:
city: Denver
country: United States
facility: National Jewish Health
state: Colorado
Study officials:
affiliation: National Jewish Health
name: Jerry A. Nick, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: De P, Amin AG, Graham B, Martiniano SL, Caceres SM, Poch KR, Jones MC, Saavedra MT, Malcolm KC, Nick JA, Chatterjee D. Urine lipoarabinomannan as a marker for low-risk of NTM infection in the CF airway. J Cyst Fibros. 2020 Sep;19(5):801-807. doi: 10.1016/j.jcf.2020.06.016. Epub 2020 Jul 3.