PATIENCE Trial: Prospective Algorithm for Treatment of NTM in Cystic Fibrosis
- Conditions
- Cystic Fibrosis
- Registration Number
- NCT02419989
- Lead Sponsor
- National Jewish Health
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ENROLLING_BY_INVITATION
- Sex
- All
- Target Recruitment
- 70
- 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
- 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
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 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 Outcome Measures
Name Time Method Variability between sites for NTM eradication success 12 months following end of treatment 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 Clinical outcomes (FEV1, growth parameters, CFQ-R) 12 months following end of treatment Duration and combinations of NTM treatment regimens received 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 Time to treatment discontinuation due to treatment success 12 months following end of treatment
Trial Locations
- Locations (19)
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
University of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
Northwestern University
🇺🇸Chicago, Illinois, United States
CFF Pediatric Program Seattle Children's Hospital
🇺🇸Seattle, Washington, United States
CFF Adult Program, University of Washington
🇺🇸Seattle, Washington, United States
CFF Pediatric Program, University of Alabama
🇺🇸Birmingham, Alabama, United States
University of California San Diego
🇺🇸San Diego, California, United States
National Jewish Health
🇺🇸Denver, Colorado, United States
Children's Hospital Colorado
🇺🇸Aurora, Colorado, United States
CFF Adult Program, Johns Hopkins University
🇺🇸Baltimore, Maryland, United States
CFF Pediatric Program, Columbia University
🇺🇸New York, New York, United States
University of Vermont Medical Center
🇺🇸Burlington, Vermont, United States
CFF Adult Program, The University of Texas Southwestern Medical Center at Dallas
🇺🇸Dallas, Texas, United States
CFF Pediatric Program University of Michigan
🇺🇸Ann Arbor, Michigan, United States
Children's Hospital of Los Angeles
🇺🇸Los Angeles, California, United States
Boston Children's Hospital, Brigham & Women's Hospital
🇺🇸Boston, Massachusetts, United States
University of Florida
🇺🇸Gainesville, Florida, United States
Dartmouth Hitchcock Medical Center
🇺🇸Lebanon, New Hampshire, United States
CFF Adult Program, University of North Carolina at Chapel Hill
🇺🇸Chapel Hill, North Carolina, United States