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Ivacaftor Treatment in 4 Month to 2 Year Old CF Subjects

NCT03783286 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 15

Last updated 2022-03-11

No results posted yet for this study

Summary

The purpose of this research study is to determine the effects of clinically prescribed ivacaftor treatment on 4-24 month old children with CF and gating mutations on sleeping energy expenditure, growth status and gut health and function.

Conditions

Interventions

DRUG

Ivacaftor

Ivacaftor (Kalydeco®, Vertex Pharmaceuticals Inc.) was the first of a new class of drugs that improved CFTR gating dysfunction (2, 3, 4). For such mutations (G551D, G178R, S549N, S549R, G551S, G970R, G1244E, S1251N, S1255P, G1349D) that permit CFTR expression at the cell membrane but compromise its activity.

Sponsors & Collaborators

Principal Investigators

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

Eligibility

Min Age
4 Months
Max Age
2 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-02-06
Primary Completion
2021-12-30
Completion
2021-12-30
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 NCT03783286 on ClinicalTrials.gov