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

Launched by NATIONAL JEWISH HEALTH · Apr 14, 2015

Keywords

ClinConnect Summary

This is an observational study called PATIENCE, which follows people with cystic fibrosis (CF) who have a lung infection caused by non-tuberculous mycobacteria (NTM). It does not test a new drug; instead, researchers watch how patients are treated in real-world care after they start NTM treatment and collect data to learn what happens in typical practice. The goal is to understand responses to treatment and to gather information that can guide future trials. The study is happening at many CF centers across the United States and plans to enroll about 70 participants.

Who can participate? People aged 6 and older with CF who have a confirmed NTM lung infection and are starting treatment for NTM, and who have previously taken part in the PREDICT study. Participants must give consent to take part and to share their data. They will be followed for roughly 12 months after the end of treatment to see how well they stick to the plan, what treatment regimens are used, whether they tolerate the drugs, whether the infection clears from sputum, and how their lung function, growth (for children), and quality of life change. The study may collect various samples (blood, sputum, breath, urine, and NTM isolates) to build a detailed picture of NTM care in CF and to help plan future research.

Gender

ALL

Eligibility criteria

• 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