An observational study in Cystic Fibrosis, sponsored by Karolinska University Hospital. Status unknown at 1 site in Sweden. Open to participants aged 12 Years and older. Per ClinicalTrials.gov, last updated 2019-03-04.
Sponsored by Karolinska University Hospital · Observational
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.
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.
1,581 studies on the registry are indexed under Cystic Fibrosis; 190 are open to participants now.
This study's planned enrollment of 20 is below the median of 85 across 482 observational studies indexed under Cystic Fibrosis.
Browse Cystic Fibrosis studies →Karolinska University Hospital is the lead sponsor of 275 studies on the registry; 54 are open to participants now.
Counted across the registry records on this site, refreshed daily.
CF patients, F508del homozygote, >12 years of age, eligible and planned for Orkambi therapy in Clinical routine.
Exclusion Criteria:
CF patients carrying the F508del mutation on both alleles planned to receive Orkambi therapy
Concentration of fecal calprotectin
Is a marker of intestinal inflammation measured in the stool
Time frame: Change from baseline at 6 months after commencing treatment with Orkambi
Concentration of fecal elastase-1
Is a test of pancreatic function measured in the stool.
Time frame: Change from baseline at 6 months after commencing treatment with Orkambi
Change in small bowel capsule endoscopy (SBCE)
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
Time frame: Change from baseline at 6 months after commencing treatment with Orkambi
Change in CRP
Inflammatory marker, unit mg/L.
Time frame: Change from baseline at 6 months after commencing treatment with Orkambi
Change in sedimentation rate
Inflammatory marker, unit mm.
Time frame: Change from baseline at 6 months after commencing treatment with Orkambi
Concentration of serum electrophoresis.
Inflammatory markers: alpha-1-antitrypsin, haptoglobin, orosomucoid, immunoglobulin A, M and G.
Time frame: Change from baseline, 6 months after commencing treatment with Orkambi
Change in liver function tests
ALT, AST, ALP, gamma-GT. Unit: mikrokat/L
Time frame: Change from baseline at 6 months after commencing treatment with Orkambi
Change in bilirubin
Bilirubin. Unit: mikromol/L
Time frame: Change from baseline at 6 months after commencing treatment with Orkambi
Documents are hosted by the registry — open the source record to download them.
Plan to share: No — Because this would expose the patients too much
This study is status unknown, as verified in Feb 2019. You cannot join it, but the record below documents what was studied.
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Karolinska University Hospital