CI

At a glance

ClinicalIndex Comparison Record
N/ACompleted· 28 enrolled
Drug / intervention
Orkambidrug
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT03795363
NCT03795363N/ACompleted
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Nutritional Impact of Orkambi Treatment in 2 to 5 Year Old Children Homozygous for F508del Mutations

Children's Hospital of Philadelphia·observational·Posted Jan 7, 2019·Updated Dec 2, 2021

In Brief

An observational study evaluating Orkambi for Cystic Fibrosis. Completed, enrolled 28 participants across 1 site.

Detailed Summary

The purpose of this observational research study is to determine the effects of clinically prescribed Orkambi treatment on 2 to 5 year old children homozygous for the F508del Mutations in the Cystic fibrosis transmembrane conductance regulator (CFTR) gene on sleeping energy expenditure, growth status and gut health and function.

Study Details

Study Typeobservational
Allocation--
Masking--
Primary Purpose--
ConditionsCystic Fibrosis
CountriesUnited States
CollaboratorsVertex Pharmaceuticals Incorporated

Timeline

N/ACompletedFinished
20192020202120222023202420252026
First PostedJan 7, 2019
Enrollment StartApr 10, 2019
Primary CompletionJun 16, 2021
TodayJul 2, 2026
Enrollment to primary: 2.2 yearsPosted 7.5 years ago

Interventions

Orkambidrug

Orkambi is a novel approved therapy for use in people homozygous for the F508del mutation in the CFTR gene. It is a combination of lumacaftor (VX-809) and ivacaftor( VX-770) that addresses both the processing and gating defects of the F508del mutation. The small-molecule corrector lumacaftor corrects the F508del processing defect and increases epithelial delivery of CFTR protein1. Ivacaftor is a CFTR potentiator that increases the channel open probability in F508del-mutant CFTRs that undergo epithelial delivery in vitro and has an additive effect with lumacaftor on chloride transport (2,3,4,5).