Prospective Analysis of Urine LAM to Eliminate NTM Sputum Screening (PAINLESS)

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

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.

Study Overview

• Status: Enrolling by invitation
• Conditions: Cystic Fibrosis
• Intervention / Treatment: Diagnostic test: urine lipoarabinomannan (LAM)

• Study Type: Observational
• Enrollment (Estimated): 220

Contacts and Locations

Study Locations

• United States
  • Colorado
    • Denver, Colorado, United States, 80206
      • National Jewish Health

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• 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

Description

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

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

Adults with a diagnosis of CF with history of negative NTM sputum cultures; 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); Investigate the utility of urine LAM as a test to identify individuals at very low risk for having a sputum culture positive for NTM.

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

Collaborators and Investigators

• Sponsor: Jerry A. Nick, M.D.
• Investigators: Principal Investigator: Jerry A. Nick, MD, National Jewish Health

Publications and helpful links

General Publications

• 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.

Study record dates

Study Major Dates

• Study Start (Actual): December 10, 2020
• Primary Completion (Actual): April 1, 2025
• Study Completion (Estimated): May 1, 2026

Study Registration Dates

• First Submitted: October 1, 2020
• First Submitted That Met QC Criteria: October 1, 2020
• First Posted (Actual): October 8, 2020

Study Record Updates

• Last Update Posted (Actual): May 23, 2025
• Last Update Submitted That Met QC Criteria: May 20, 2025
• Last Verified: May 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Respiratory Tract Diseases; Digestive System Diseases; Lung Diseases; Infant, Newborn, Diseases; Pancreatic Diseases; Cystic Fibrosis
• Other Study ID Numbers: NICK20A0 20-08-402-528

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No