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
- 200
- 试验地点
- 19
- 主要终点
- Proportion meeting the diagnosis of NTM disease
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 the diagnosis 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 diagnosis differ widely between centers. This study is observational and follows current best practices. The study will help standardize the diagnosis and collect relevant data associated with the diagnosis 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
- •Written informed consent (and assent when applicable) obtained from participant or participant's legal representative
- •Enrolled in the CFF Patient Registry (CFF PR)
- •Be willing and able to adhere to study procedures in the context of clinical care, and other protocol requirements
- •Male or female participant ≥ 6 years of age at enrollment
- •Diagnosis of CF consistent with the 2017 CFF Guidelines
- •NTM positive for a species or sub-species in the 2 years prior to enrollment that has never been treated.
- •Willing to discontinue chronic azithromycin use for the duration of the study
排除标准
- •Prior or ongoing antibiotic treatment of the same NTM species or sub-species for which the patient is being considered for this study
- •History of solid organ or hematological transplantation
- •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 meeting the diagnosis of NTM disease
时间窗: 12 months intervals following study enrollment
次要结局
- Adherence to PREDICT protocol: number of respiratory cultures obtained per participant per year, withdrawals and major deviations from protocol(12 months intervals following study enrollment)
- Clinical parameters and outcomes (FEV1, growth parameters, CFQ-R)(12 months intervals following study enrollment)
- Incidence and prevalence of NTM species/subspecies by geographical region(12 months intervals following study enrollment)
- Proportion of PREDICT participants with NTM disease diagnosis who enroll in Part B PATIENCE(12 months intervals following study enrollment)
- Proportion of eligible NTM positive individuals with CF enrolling in PREDICT by site(12 months intervals following study enrollment)
研究者
Jerry A. Nick, M.D.
Professor of Medicine
National Jewish Health