PATIENCE Trial: Prospective Algorithm for Treatment of NTM in Cystic Fibrosis (PATIENCE)

March 30, 2024 updated by: Jerry A. Nick, M.D., National Jewish Health

Prospective Evaluation of a Standardized Approach to Diagnosis (PREDICT) and Treatment (PATIENCE) of Nontuberculous Mycobacteria Disease in Cystic Fibrosis

Isolation of nontuberculous mycobacteria (NTM) from the sputum of individuals with CF is an increasingly common finding, and the lack of an evidenced-based approach to treatment of NTM disease has been identified as one of the greatest unmet needs within the CF community. Current evidence predicts that the prevalence of NTM will remain relatively high in the CF population. Approaches to NTM disease treatment differ widely between centers, and expected outcomes are not known. This study is observational and follows current best practices. The study will help define response to treatment, and collect relevant data associated with treatment of NTM disease to build a framework for future therapeutic trials.

Study Overview

Status

Enrolling by invitation

Conditions

Study Type

Observational

Enrollment (Estimated)

70

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35233
        • CFF Pediatric Program, University of Alabama
    • California
      • Los Angeles, California, United States
        • Children's Hospital of Los Angeles
      • San Diego, California, United States
        • University of California San Diego
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Children's Hospital Colorado
      • Denver, Colorado, United States, 80206
        • National Jewish Health
    • Florida
      • Gainesville, Florida, United States
        • University of Florida
    • Illinois
      • Chicago, Illinois, United States
        • Northwestern University
    • Maryland
      • Baltimore, Maryland, United States, 21205
        • CFF Adult Program, Johns Hopkins University
    • Massachusetts
      • Boston, Massachusetts, United States
        • Boston Children's Hospital, Brigham & Women's Hospital
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • CFF Pediatric Program University of Michigan
    • New Hampshire
      • Lebanon, New Hampshire, United States
        • Dartmouth Hitchcock Medical Center
    • New York
      • New York, New York, United States, 10032
        • CFF Pediatric Program, Columbia University
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27517
        • CFF Adult Program, University of North Carolina at Chapel Hill
    • Ohio
      • Columbus, Ohio, United States
        • Nationwide Children's Hospital
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States
        • University of Pittsburgh
    • Texas
      • Dallas, Texas, United States, 75390-8558
        • CFF Adult Program, The University of Texas Southwestern Medical Center at Dallas
    • Vermont
      • Burlington, Vermont, United States
        • University of Vermont Medical Center
    • Washington
      • Seattle, Washington, United States, 98105
        • CFF Pediatric Program Seattle Children's Hospital
      • Seattle, Washington, United States, 98195
        • CFF Adult Program, University of Washington

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 years to 99 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Male and female subjects with CF age 6 years and older with a confirmed diagnosis of NTM disease who are initiating treatment for NTM.

Description

Inclusion Criteria:

  1. Previous participation in PREDICT and diagnosis of NTM disease for the same species of NTM
  2. Intention to treat for NTM disease for M. avium complex or M. abscessus complex
  3. Written informed consent (and assent when applicable) obtained from participant or participant's legal representative
  4. Signed informed consent to participate in data submission to the CFF Patient Registry
  5. Be willing and able to initiate treatment for NTM and to adhere to study procedures in the context of clinical care, and other protocol requirements

Exclusion Criteria:

  1. Pregnant or breastfeeding
  2. 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

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Case-Only
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
CF patients
Male and female subjects with CF age 6 years and older who meet diagnostic criteria for NTM disease through participation in the PREDICT (Part A) study who are being offered NTM treatment.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Proportion who adhere to the protocol based on: number of respiratory cultures obtained per year, withdrawals or major deviations from protocol
Time Frame: 12 months following end of antibiotic treatment
12 months following end of antibiotic treatment

Secondary Outcome Measures

Outcome Measure
Time Frame
Duration and combinations of NTM treatment regimens received
Time Frame: At end of treatment interval
At end of treatment interval
Proportion requiring antibiotic course change due to intolerance or lack of microbiological conversion (i.e. eradication from sputum)
Time Frame: At end of treatment interval
At end of treatment interval
Proportion who complete ≥ 12 months of treatment since first negative culture
Time Frame: At end of treatment interval
At end of treatment interval
Proportion eradicating NTM defined by ≥ 12 months of negative cultures since end of treatment
Time Frame: 12 months following end of treatment
12 months following end of treatment
Variability between sites for NTM eradication success
Time Frame: 12 months following end of treatment
12 months following end of treatment
Time to treatment discontinuation due to treatment success
Time Frame: 12 months following end of treatment
12 months following end of treatment
Clinical outcomes (FEV1, growth parameters, CFQ-R)
Time Frame: 12 months following end of treatment
12 months following end of treatment

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

National Jewish Health

Collaborators

Children's Hospital Colorado
Cystic Fibrosis Foundation

Investigators

  • Principal Investigator: Jerry A Nick, MD, National Jewish Health
  • Principal Investigator: Stacey Martiniano, MD, Children's Hospital Colorado

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 1, 2014

Primary Completion (Estimated)

March 1, 2025

Study Completion (Estimated)

March 1, 2025

Study Registration Dates

First Submitted

December 9, 2014

First Submitted That Met QC Criteria

April 14, 2015

First Posted (Estimated)

April 17, 2015

Study Record Updates

Last Update Posted (Actual)

April 2, 2024

Last Update Submitted That Met QC Criteria

March 30, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NTM-OB-17 (PATIENCE PART B)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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