Gastrointestinal Study at Orkambi Therapy in CF Patients

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Total Enrollment

20 participants

Study Classification

OBSERVATIONAL

Study Start Date

2019-02-27

Study Completion Date

2020-06-30

Brief Summary

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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.

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Detailed Description

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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.

Conditions

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Cystic Fibrosis

Study Design

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Observational Model Type

CASE_ONLY

Study Time Perspective

PROSPECTIVE

Study Groups

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CF patients planned to receive Orkambi

CF patients carrying the F508del mutation on both alleles planned to receive Orkambi therapy

No interventions assigned to this group

Eligibility Criteria

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Inclusion Criteria

* CF patients F508del homozygote
* \>12 years of age
* eligible for Orkambi therapy.

Exclusion Criteria

* 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

Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No