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Testing Drug Efficacy in Cystic Fibrosis Through N-of-1 Trials

NCT04580368 · Status: RECRUITING · Phase: NA · Type: INTERVENTIONAL · Enrollment: 50

Last updated 2026-01-07

No results posted yet for this study

Summary

The purpose of this study is to validate and utilize a personalized medicine approach to identify potential treatments with current FDA approved CFTR modifiers for non-approved CF gene mutations. The study will perform ex vivo testing of CFTR function and current marketed CFTR modulating drugs on expanded nasal cells at Cincinnati Children's Human Nasal Epithelium (HNE) Core Laboratory. The results will be confirmed and translated into bedside care through an N of 1 trial to determine effectiveness of treatment.

Conditions

Interventions

DRUG

CFTR Modulators

Participants with rare mutations will receive active therapy in N-of-1 design with participants serving as their own control

Sponsors & Collaborators

  • Children's Hospital Medical Center, Cincinnati

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-05-01
Primary Completion
2028-12-31
Completion
2030-01-31

Countries

  • United States

Study Locations

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Entities

Diseases

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 NCT04580368 on ClinicalTrials.gov