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

Study Overview
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Brief Summary
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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.

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

Conditions
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Cystic Fibrosis

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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 Exclusion Criteria: 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

Ages Eligible for Study
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Minimum Age: 6 Years
Maximum Age: 99 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| CF patients<br>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?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 
| Time to treatment discontinuation due to treatment success |  | 12 months following end of treatment | 
| Clinical outcomes (FEV1, growth parameters, CFQ-R) |  | 12 months following end of treatment | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Nontuberculous Mycobacteria