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CFTR Modifiers

CFTR Modulators for Cystic Fibrosis (Nof1 Trial)

N/A
Recruiting
Research Sponsored by Children's Hospital Medical Center, Cincinnati
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Ivacaftor: ≥4 months old
Tezacaftor/Ivacaftor: 12 years old
Must not have
End stage renal disease
FEV1 ≤ 50% predicted for age
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 16 weeks
Awards & highlights
No Placebo-Only Group

Summary

This trial will test if current CF treatments can be effective for new, non-approved CF gene mutations.

Who is the study for?
This trial is for individuals with Cystic Fibrosis who have rare CFTR gene mutations and are at least 6 years old, meeting age-specific criteria for different modulator drugs. They must not have had changes in their chronic CF therapies in the last 28 days, have an FEV1 > 50% predicted, and no history of organ transplantation or conditions that could risk their safety.
What is being tested?
The study tests FDA-approved CFTR modulators on nasal cells to see if they work for non-approved CF gene mutations. It involves lab testing followed by personalized 'N-of-1' trials where each participant's response to treatment is closely monitored to assess effectiveness.
What are the potential side effects?
While specific side effects aren't listed here, generally CFTR modulators can cause chest tightness, shortness of breath, stomach issues (like pain or discomfort), elevated liver enzymes which may indicate liver injury, and potential drug interactions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am at least 4 months old.
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I am at least 12 years old.
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I have two mutations in my CFTR gene.
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My lung function is more than half of what is expected for my age.
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I am 12 years old or older.
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I have a rare CF gene mutation.
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I am prescribed a CFTR modulator by my doctor.
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I am at least 2 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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My kidneys are in the final stage of failure.
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My lung function is less than half of what's expected for my age.
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My diabetes is not well-managed (HbA1c > 8.5).
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I have had an organ transplant.
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My BMI is below the healthy range for my age.
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I am not taking strong medications like voriconazole or high-dose prednisone.
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My kidney function, measured by a specific test, is reduced.
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I have advanced liver disease due to cystic fibrosis.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~16 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and 16 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
ppFEV1

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: CFTR modulator or other therapiesExperimental Treatment1 Intervention
CFTR modulator or active therapy

Find a Location

Who is running the clinical trial?

Children's Hospital Medical Center, CincinnatiLead Sponsor
837 Previous Clinical Trials
6,565,287 Total Patients Enrolled
22 Trials studying Cystic Fibrosis
1,135 Patients Enrolled for Cystic Fibrosis

Media Library

CFTR Modulators (CFTR Modifiers) Clinical Trial Eligibility Overview. Trial Name: NCT04580368 — N/A
Cystic Fibrosis Research Study Groups: CFTR modulator or other therapies
Cystic Fibrosis Clinical Trial 2023: CFTR Modulators Highlights & Side Effects. Trial Name: NCT04580368 — N/A
CFTR Modulators (CFTR Modifiers) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04580368 — N/A
~19 spots leftby Dec 2026
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