Gastrointestinal Study at Orkambi Therapy in CF Patients
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| ClinicalTrials.gov Identifier: NCT03859531 |
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Recruitment Status :
Recruiting
First Posted : March 1, 2019
Last Update Posted : March 4, 2019
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| Condition or disease |
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| Cystic Fibrosis |
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.
| Study Type : | Observational |
| Estimated Enrollment : | 20 participants |
| Observational Model: | Case-Only |
| Time Perspective: | Prospective |
| Official Title: | Gastrointestinal Outcome Measures Before and After Orkambi Therapy in Cystic Fibrosis (CF) Patients Carrying the F508del Mutation on Both Alleles |
| Actual Study Start Date : | February 27, 2019 |
| Estimated Primary Completion Date : | June 30, 2020 |
| Estimated Study Completion Date : | June 30, 2020 |
| Group/Cohort |
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CF patients planned to receive Orkambi
CF patients carrying the F508del mutation on both alleles planned to receive Orkambi therapy
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- Concentration of fecal calprotectin [ Time Frame: Change from baseline at 6 months after commencing treatment with Orkambi ]Is a marker of intestinal inflammation measured in the stool
- Concentration of fecal elastase-1 [ Time Frame: Change from baseline at 6 months after commencing treatment with Orkambi ]Is a test of pancreatic function measured in the stool.
- Change in small bowel capsule endoscopy (SBCE) [ Time Frame: 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
- Change in CRP [ Time Frame: Change from baseline at 6 months after commencing treatment with Orkambi ]Inflammatory marker, unit mg/L.
- Change in sedimentation rate [ Time Frame: Change from baseline at 6 months after commencing treatment with Orkambi ]Inflammatory marker, unit mm.
- Concentration of serum electrophoresis. [ Time Frame: Change from baseline, 6 months after commencing treatment with Orkambi ]Inflammatory markers: alpha-1-antitrypsin, haptoglobin, orosomucoid, immunoglobulin A, M and G.
- Change in liver function tests [ Time Frame: Change from baseline at 6 months after commencing treatment with Orkambi ]ALT, AST, ALP, gamma-GT. Unit: mikrokat/L
- Change in bilirubin [ Time Frame: Change from baseline at 6 months after commencing treatment with Orkambi ]Bilirubin. Unit: mikromol/L
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| Ages Eligible for Study: | 12 Years and older (Child, Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Non-Probability Sample |
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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03859531
| Sweden | |
| Stockholm CF Center | Recruiting |
| Stockholm, Sweden, 14186 | |
| Contact: Isabelle de Monestrol, MD PhD +468 58580000 isabelle.demonestrol@ki.se | |
| Principal Investigator: | Isabelle de Monestrol, MD PhD | Stockholm CF Center |
Documents provided by Isabelle de Monestrol, Karolinska University Hospital:
| Responsible Party: | Isabelle de Monestrol, Director of Stockholm CF Center, Karolinska University Hospital |
| ClinicalTrials.gov Identifier: | NCT03859531 |
| Other Study ID Numbers: |
CF-GI-001 |
| First Posted: | March 1, 2019 Key Record Dates |
| Last Update Posted: | March 4, 2019 |
| Last Verified: | February 2019 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Plan Description: | Because this would expose the patients too much |
| Studies a U.S. FDA-regulated Drug Product: | No |
| Studies a U.S. FDA-regulated Device Product: | No |
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Cystic Fibrosis Fibrosis Pathologic Processes Pancreatic Diseases Digestive System Diseases |
Lung Diseases Respiratory Tract Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases |