Prospective Analysis of Urine LAM to Eliminate NTM Sputum Screening

Study Overview
=================
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.

Official Title
-----------------
Prospective Analysis of Urine LAM to Eliminate NTM Sputum Screening (PAINLESS Trial)

Conditions
-----------------
Cystic Fibrosis

Intervention / Treatment
-----------------
* Diagnostic Test: urine lipoarabinomannan (LAM)


Participation Criteria
=================
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

Ages Eligible for Study
-----------------
Minimum Age: 18 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Adults with a diagnosis of CF with history of negative NTM sputum cultures<br>Male or female participants age 18 or greater at time of enrollment with diagnosis of CF consistent with the 2017 CFF Guidelines and NTM culture status of negative, as defined by a review of at least 3 year or more years of culture data and at least 3 NTM negative cultures with one of those negative cultures being within the last 3 years and no known history of previous positive cultures for pathogenic NTM by chart review. | Diagnostic Test: urine lipoarabinomannan (LAM)<br>* Investigate the utility of urine LAM as a test to identify individuals at very low risk for having a sputum culture positive for NTM.<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To determine the ability of the urine LAM assay to predict continued negative sputum culture over the next 12 months. | Correlation of a negative NTM sputum culture in the year following a negative urine LAM assay | From time enrollment to over the next 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To determine the ability of the urine LAM assay to predict new positive NTM sputum culture over the next 12 months. | Correlation of a positive NTM sputum culture in the year following a positive urine LAM assay | From time of enrollment to over the next 12 months | 
| To determine the time between a positive LAM assay and a new positive NTM culture. | Correlation of a positive NTM sputum culture up to 3 years following a positive urine LAM assay | 3 years | 
| To facilitate the development of a ELISA-based assay as a possible replacement to GC/MS in the clinical setting. | Excess urine will be used to develop and validate an alternative assay using ELISA. | 3 years |