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

Part of paid clinical trials in Cincinnati, Ohio.

Sponsor
Children's Hospital Medical Center, Cincinnati
Study ID
NCT04580368
Status
Recruiting
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Conditions

  • Cystic Fibrosis

Eligibility Criteria

Sex
ALL
Age
6 Years - N/A
Healthy Volunteers
Not accepted

Interventions

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

Study Details

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.

Key Dates

Start date
May 1, 2021
Status verified
Jan 2026
Primary completion
Dec 31, 2028
Completion
Jan 31, 2030

Study Design

Enrollment
50 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: CFTR modulator or other therapies
    CFTR modulator or active therapy

Primary Outcome Measure

ppFEV1 [ Time Frame: 16 weeks ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
CCHMCCincinnatiOhio45203
John Brewington
[email protected]
5138031548

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By research site
CCHMC· Cincinnati, OH

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