PATIENCE Trial: Prospective Algorithm for Treatment of NTM in Cystic Fibrosis
PATIENCE
Prospective Evaluation of a Standardized Approach to Diagnosis (PREDICT) and Treatment (PATIENCE) of Nontuberculous Mycobacteria Disease in Cystic Fibrosis
1 other identifier
observational
70
1 country
19
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for all trials
Started Jan 2014
Longer than P75 for all trials
19 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
January 1, 2014
CompletedFirst Submitted
Initial submission to the registry
December 9, 2014
CompletedFirst Posted
Study publicly available on registry
April 17, 2015
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 1, 2025
CompletedStudy Completion
Last participant's last visit for all outcomes
March 1, 2025
CompletedApril 2, 2024
March 1, 2024
11.2 years
December 9, 2014
March 30, 2024
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Proportion who adhere to the protocol based on: number of respiratory cultures obtained per year, withdrawals or major deviations from protocol
12 months following end of antibiotic treatment
Secondary Outcomes (7)
Duration and combinations of NTM treatment regimens received
At end of treatment interval
Proportion requiring antibiotic course change due to intolerance or lack of microbiological conversion (i.e. eradication from sputum)
At end of treatment interval
Proportion who complete ≥ 12 months of treatment since first negative culture
At end of treatment interval
Proportion eradicating NTM defined by ≥ 12 months of negative cultures since end of treatment
12 months following end of treatment
Variability between sites for NTM eradication success
12 months following end of treatment
- +2 more secondary outcomes
Study Arms (1)
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.
Eligibility Criteria
Male and female subjects with CF age 6 years and older with a confirmed diagnosis of NTM disease who are initiating treatment for NTM.
You may qualify if:
- Previous participation in PREDICT and diagnosis of NTM disease for the same species of NTM
- Intention to treat for NTM disease for M. avium complex or M. abscessus complex
- Written informed consent (and assent when applicable) obtained from participant or participant's legal representative
- Signed informed consent to participate in data submission to the CFF Patient Registry
- 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
You may not qualify if:
- Pregnant or breastfeeding
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- National Jewish Healthlead
- Cystic Fibrosis Foundationcollaborator
- Children's Hospital Coloradocollaborator
Study Sites (19)
CFF Pediatric Program, University of Alabama
Birmingham, Alabama, 35233, United States
Children's Hospital of Los Angeles
Los Angeles, California, United States
University of California San Diego
San Diego, California, United States
Children's Hospital Colorado
Aurora, Colorado, 80045, United States
National Jewish Health
Denver, Colorado, 80206, United States
University of Florida
Gainesville, Florida, United States
Northwestern University
Chicago, Illinois, United States
CFF Adult Program, Johns Hopkins University
Baltimore, Maryland, 21205, United States
Boston Children's Hospital, Brigham & Women's Hospital
Boston, Massachusetts, United States
CFF Pediatric Program University of Michigan
Ann Arbor, Michigan, 48109, United States
Dartmouth Hitchcock Medical Center
Lebanon, New Hampshire, United States
CFF Pediatric Program, Columbia University
New York, New York, 10032, United States
CFF Adult Program, University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27517, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
University of Pittsburgh
Pittsburgh, Pennsylvania, United States
CFF Adult Program, The University of Texas Southwestern Medical Center at Dallas
Dallas, Texas, 75390-8558, United States
University of Vermont Medical Center
Burlington, Vermont, United States
CFF Pediatric Program Seattle Children's Hospital
Seattle, Washington, 98105, United States
CFF Adult Program, University of Washington
Seattle, Washington, 98195, United States
Biospecimen
Samples collected may include serum, sputum, whole blood, breath, urine, and NTM isolates.
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Jerry A Nick, MD
National Jewish Health
- PRINCIPAL INVESTIGATOR
Stacey Martiniano, MD
Children's Hospital Colorado
Study Design
- Study Type
- observational
- Observational Model
- CASE ONLY
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- Professor of Medicine
Study Record Dates
First Submitted
December 9, 2014
First Posted
April 17, 2015
Study Start
January 1, 2014
Primary Completion
March 1, 2025
Study Completion
March 1, 2025
Last Updated
April 2, 2024
Record last verified: 2024-03