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Report for Clinical Trial NCT03859531

Developed by Shray Alag, 2020.
SNP Clinical Trial Gene

Gastrointestinal Outcome Measures Before and After Orkambi Therapy in Cystic Fibrosis (CF) Patients Carrying the F508del Mutation on Both Alleles

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.

NCT03859531 Cystic Fibrosis
MeSH:Cystic Fibrosis Fibrosis


Primary Outcomes

Description: Is a marker of intestinal inflammation measured in the stool

Measure: Concentration of fecal calprotectin

Time: Change from baseline at 6 months after commencing treatment with Orkambi

Description: Is a test of pancreatic function measured in the stool.

Measure: Concentration of fecal elastase-1

Time: Change from baseline at 6 months after commencing treatment with Orkambi

Description: 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

Measure: Change in small bowel capsule endoscopy (SBCE)

Time: Change from baseline at 6 months after commencing treatment with Orkambi

Secondary Outcomes

Description: Inflammatory marker, unit mg/L.

Measure: Change in CRP

Time: Change from baseline at 6 months after commencing treatment with Orkambi

Description: Inflammatory marker, unit mm.

Measure: Change in sedimentation rate

Time: Change from baseline at 6 months after commencing treatment with Orkambi

Description: Inflammatory markers: alpha-1-antitrypsin, haptoglobin, orosomucoid, immunoglobulin A, M and G.

Measure: Concentration of serum electrophoresis.

Time: Change from baseline, 6 months after commencing treatment with Orkambi

Description: ALT, AST, ALP, gamma-GT. Unit: mikrokat/L

Measure: Change in liver function tests

Time: Change from baseline at 6 months after commencing treatment with Orkambi

Description: Bilirubin. Unit: mikromol/L

Measure: Change in bilirubin

Time: Change from baseline at 6 months after commencing treatment with Orkambi

Time Perspective: Prospective

Case-Only


There is one SNP

SNPs


1 G551D

Gastrointestinal Study at Orkambi Therapy in CF Patients 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. --- G551D ---



HPO Nodes