Cystic Fibrosis
Conditions
Keywords
Nontuberculous Mycobacteria
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.
Interventions
None listed
Sponsors
Cystic Fibrosis Foundation
Children's Hospital Colorado
National Jewish Health
Study design
Observational model
CASE_ONLY
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
6 Years to 99 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | 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
| Measure | Time frame |
|---|---|
| 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 |
| Duration and combinations of NTM treatment regimens received | At end of treatment interval |
| 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 |
| Variability between sites for NTM eradication success | 12 months following end of treatment |
Countries
United States
Outcome results
None listed