Canadian Observational Study Evaluating the Long-term IMPACT of CFTR (Can-IMPACT CF)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 500
- 试验地点
- 2
- 主要终点
- To establish a bio-repository to enable further investigations of the effectiveness of CFTR modulator therapies on biological markers and predictors of response.
研究概览
简要总结
This observational study intends to investigate health trends and data in cystic fibrosis patients all across Canada that are receiving modulator treatment so researchers can determine if CFTR treatments are effective over a long period of time and if so, which treatments work best for each individual. The study will collect clinical data from routine standard of care, patient reported outcomes via survey data and samples for a biobank.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Prescribed a CFTR modulator therapy and planning to commence modulator therapy within 30 days OR planning to switch modulator therapies within 30 days
- •Participates in the Canadian Cystic Fibrosis Registry (CCFR)
- •Informed consent by participant, or parent/legal guardian or assent
排除标准
- •Known contraindications to CFTR modulator therapy
研究组 & 干预措施
Pre-Therapy Participants
All CF people with CF who do not have any known contraindications to CFTR modulator therapy and will be initiated on CFTR modulator therapy by their treating physician as part of clinical care are eligible and will be asked to participate in this study.
干预措施: Trikafta (Drug)
结局指标
主要结局
To establish a bio-repository to enable further investigations of the effectiveness of CFTR modulator therapies on biological markers and predictors of response.
时间窗: 5 years
To establish the benefits of CFTR modulators compared to propensity-score matched historic CF populations followed in the Canadian CF registry.
时间窗: 5 years
To examine the long-term pulmonary and nutritional effects of CFTR modulators.
时间窗: 5 years
To assess the long-term impact of CFTR modulators on patient-reported outcome measures including quality of life, disease burden, and physical activity.
时间窗: 5 years
次要结局
未报告次要终点
研究者
Jonathan Rayment
Principle Investigator
University of British Columbia