Gastrointestinal Outcome Measures Before and After Orkambi Therapy in Cystic Fibrosis (CF) Patients Carrying the F508del Mutation on Both Alleles
试验速览
- 阶段
- 不适用
- 发起方
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Concentration of fecal calprotectin
研究概览
简要总结
Ivacaftor caused a significance increase in weight in patients carrying the G551D mutation and the etiology of this has largely remained unknown but may be due to improved function of the gastrointestinal tract. The combination therapy of Orkambi has been recently approved for subjects with Cystic Fibrosis homozygous for F508del mutation. This provides an opportunity to examine if there are any improvements in gastrointestinal function. The investigators aim to investigate various aspects of gastrointestinal and pancreatic function before and 6 months after the commencement of Orkambi therapy.
详细描述
To examine the entire intestinal mucosa via capsule endoscopy before and 6 months after Orkambi therapy to ascertain if the inflammatory changes in the intestine have improved. A marker of intestinal inflammation measured in the stool, Calprotectin, will be examined before and 6 months after Orkambi treatment. The investigators hypothesize that the result will be reduced on therapy.
A marker of pancreatic exocrine function, pancreatic elastase, will be examined before and 6 months after therapy to examine if the result has increased indicating improvement of exocrine pancreatic function
Study Population All subjects with CF homozygous for the F508del mutation in Sweden eligible for Orkambi therapy, i.e. above 12 years of age, in total 145 patients in Sweden of which 60 are taken care of at Stockholm CF Center; the investigators aim to examine 20 patients.
Study Duration The duration will be 6 months for each patient.
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
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •CF patients F508del homozygote
- •>12 years of age
- •eligible for Orkambi therapy.
排除标准
- •Patients who the patency capsule does not pass within 48 hrs
- •FEV1<30%
- •Pregnancy and breastfeeding women
- •Liver function blood tests (AST, ALT, Gamma-GT, ALP) >3 xULN
- •Bilirubin >2 xULN
- •AST or ALT alone >5 xULN
- •Previous lung transplant
结局指标
主要结局
Concentration of fecal calprotectin
时间窗: Change from baseline at 6 months after commencing treatment with Orkambi
Is a marker of intestinal inflammation measured in the stool
Change in small bowel capsule endoscopy (SBCE)
时间窗: Change from baseline at 6 months after commencing treatment with Orkambi
The method of SBCE has been well established as a descriptive diagnostic tool for intestinal inflammation and has been used as an outcome measure in clinical trials. Erythema, petechiae, mucosal erosions and ulcerations will be assessed according to the Maiden criteria
Concentration of fecal elastase-1
时间窗: Change from baseline at 6 months after commencing treatment with Orkambi
Is a test of pancreatic function measured in the stool.
次要结局
- Change in CRP(Change from baseline at 6 months after commencing treatment with Orkambi)
- Change in sedimentation rate(Change from baseline at 6 months after commencing treatment with Orkambi)
- Change in liver function tests(Change from baseline at 6 months after commencing treatment with Orkambi)
- Change in bilirubin(Change from baseline at 6 months after commencing treatment with Orkambi)
- Concentration of serum electrophoresis.(Change from baseline, 6 months after commencing treatment with Orkambi)
研究者
Isabelle de Monestrol
Director of Stockholm CF Center
Karolinska University Hospital