Innovative Approach for Self-management and Social Welfare of Cystic Fibrosis Patients in Europe: Development, Validation and Implementation of a Telematics Tool. WP6.2: Impact Assessment Through a European Multicentre Clinical Trial: Validation of MyCyFAPP as a Portable System for Self-management in Children With CF
试验速览
- 阶段
- 不适用
- 入组人数
- 200
- 主要终点
- change in Modified PedsQL GI
研究概览
简要总结
Interventional trial to study the influence of the use of MyCyFAPP (mobile application) on the gastro-intestinal related quality of life.
This mobile APP has been developed during previous workpackages of the Horizon2020 Project and contains several modules:
- mathematical prediction model to calculate the needed dose for pancreatic enzyme replacement therapy
- educational games and other educational material
- communication with doctor/dietician through professional webtool
- diary to register symptoms and data on nutrition.
The app will be introduced and used during 6 months. Primary outcome parameter will be change in modified PedsQL GI after 3 months. PedsQL GI is an existing questionnaire that evaluates gastro-intestinal related quality of life in children. We validated it for use in cystic fibrosis in a previous observational study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of CF as evidenced by one or more clinical feature consistent with the CF phenotype or positive CF newborn screen AND one or more of the following criteria:
- •A documented sweat chloride ≥ 60 mEq/L by quantitative pilocarpine iontophoresis (QPIT)
- •A documented genotype with two disease-causing mutations in the CFTR gene
- •Having pancreatic insufficiency (stool elastase < 200 mcg/g stool) and using PERT
- •Age ≥ 24 months and < 18 years at screening visit
- •Informed consent by parent or legal guardian; assent for children from age 12 years on
- •Inclusion visit coincides with scheduled routine clinic visit
- •Ability and willingness to comply with APP use and evaluations at time of routine clinic visits as judged by the site investigator
- •Availability of wifi connection at home so that connection to the internet is feasible at home at least weekly.
排除标准
- •Acute infection associated with decreased appetite or fever at time of run-in visit
- •Acute abdominal pain necessitating an intervention at time of run-in visit
- •Physical findings that would compromise the safety of the participant or the quality of the study data as determined by site investigator
- •Investigational drug use within 30 days prior to run-in visit
- •Started with CFTR modulator treatment less than 3 months before start of run-in visit
- •Inability to use the APP due to patient specific factors such as language or learning difficulties
研究组 & 干预措施
use of MyCyFAPP
use of MyCyFAPP during 6 months
干预措施: MyCyFAPP (Device)
结局指标
主要结局
change in Modified PedsQL GI
时间窗: 3 months
Modified PedsQL GI will be assessed at month 0 and month 3 by applying questionnaires to children and their parents
次要结局
- change in Modified PedsQL GI(6 months)
- change in lung function(3 and 6 months)
- change in CFQ-R(3 months and 6 months)
- change in VAS(3 months and 6 months)
研究者
Mieke Boon, MD PhD
Mieke Boon
Universitaire Ziekenhuizen KU Leuven