Prospective Evaluation of a Standardized Approach to Diagnosis (PREDICT) and Treatment (PATIENCE) of Nontuberculous Mycobacteria Disease in Cystic Fibrosis
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 入组人数
- 70
- 试验地点
- 19
- 主要终点
- Proportion who adhere to the protocol based on: number of respiratory cultures obtained per year, withdrawals or major deviations from protocol
研究概览
简要总结
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 年龄范围
- 6 Years 至 99 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
结局指标
主要结局
Proportion who adhere to the protocol based on: number of respiratory cultures obtained per year, withdrawals or major deviations from protocol
时间窗: 12 months following end of antibiotic treatment
次要结局
- 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)
研究者
Jerry A. Nick, M.D.
Professor of Medicine
National Jewish Health