MedTrace Logo

Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young Children

NCT04509050 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 210

Last updated 2024-08-12

No results posted yet for this study

Summary

This is a two-part, multi-center, prospective longitudinal, exploratory study of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators and their impact on children with cystic fibrosis (CF).

Conditions

Interventions

DRUG

Ivacaftor or elexacaftor/tezacaftor/ivacaftor

In Part B, approved CFTR modulator as prescribed at the discretion of the treating physician -not dictated by the BEGIN investigators

Sponsors & Collaborators

  • Cystic Fibrosis Foundation

    collaborator OTHER

  • University of Washington

    collaborator OTHER

  • University of Alabama at Birmingham

    collaborator OTHER

  • Sonya Heltshe

    lead OTHER

Principal Investigators

  • Bonnie Ramsey, MD · Seattle Children's Hospital

  • Lucas Hoffman, MD PhD · University of Washington/Seattle Children's

  • Katie Larson Ode, MD · University of Iowa

Eligibility

Max Age
10 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-11-18
Primary Completion
2029-12-01
Completion
2029-12-01

Countries

  • United States

Study Locations

More Related Trials

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 NCT04509050 on ClinicalTrials.gov