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Orkambi Treatment in 2 to 5 Year Old Children With CF

NCT03795363 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 28

Last updated 2021-12-02

No results posted yet for this study

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.

Conditions

Interventions

DRUG

Orkambi

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).

Sponsors & Collaborators

Principal Investigators

  • Virginia Stallings, MD · Children's Hospital of Philadelphia

Eligibility

Min Age
2 Years
Max Age
5 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-04-10
Primary Completion
2021-06-16
Completion
2021-06-16
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03795363 on ClinicalTrials.gov