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

Study Timeline

• Study Start: 2014-01
• Study First Submitted: 2014-12-09
• Study First Submitted QC: 2015-04-14
• Study First Posted: 2015-04-17
• Status Verified: 2024-03
• Last Update Submitted: 2024-03-30
• Last Update Posted: 2024-04-02
• Primary Completion: 2025-03
• Study Completion: 2025-03

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 70

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 & 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)

CFF Pediatric Program, University of Alabama; 🇺🇸 Birmingham, Alabama, 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, United States

National Jewish Health; 🇺🇸 Denver, Colorado, United States

University of Florida; 🇺🇸 Gainesville, Florida, United States

Northwestern University; 🇺🇸 Chicago, Illinois, United States

CFF Adult Program, Johns Hopkins University; 🇺🇸 Baltimore, Maryland, United States

Boston Children's Hospital, Brigham & Women's Hospital; 🇺🇸 Boston, Massachusetts, United States

CFF Pediatric Program University of Michigan; 🇺🇸 Ann Arbor, Michigan, United States

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