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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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 16 weeks
Awards & highlights
Nof1 Trial 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.Check my eligibility
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.See study design
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.
Nof1 Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
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.
Nof1 Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 16 weeks
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~16 weeks
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
ppFEV1
Nof1 Trial Design
1Treatment groups
Experimental Treatment
Group I: CFTR modulator or other therapiesExperimental Treatment1 Intervention
CFTR modulator or active therapy
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Who is running the clinical trial?
Children's Hospital Medical Center, CincinnatiLead Sponsor
815 Previous Clinical Trials
6,531,541 Total Patients Enrolled
22 Trials studying Cystic Fibrosis
1,135 Patients Enrolled for Cystic Fibrosis
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I am at least 4 months old.Your blood clotting time is more than 1.5 times the normal level.Your white blood cell count is very high or your absolute neutrophil count is very low.Your platelet count is less than 50,000 per microliter of blood.My kidneys are in the final stage of failure.My lung function is less than half of what's expected for my age.I am 6 years or older and eligible for the drug according to FDA guidelines.I am at least 12 years old.I have been diagnosed with cystic fibrosis based on symptoms and tests.I have two mutations in my CFTR gene.Your sweat chloride level is higher than 60mmol/L.My lung function is more than half of what is expected for my age.Complete Blood CountYour total or direct bilirubin levels are more than twice the normal limit.I have no health issues preventing me from starting the treatment.I am 12 years old or older.I have had a lung infection with specific bacteria in the last 6 months.I have a rare CF gene mutation.You have a test that shows unusual results and supports a diagnosis of cystic fibrosis.My diabetes is not well-managed (HbA1c > 8.5).You have a high level of glucose in your urine.I have had an organ transplant.My BMI is below the healthy range for my age.I am not taking strong medications like voriconazole or high-dose prednisone.I am prescribed a CFTR modulator by my doctor.My kidney function, measured by a specific test, is reduced.You need to have tests done to check the health of your liver and blood clotting.You are pregnant.My cystic fibrosis treatments have been stable for over a month.You have important health problems as determined by the doctor.Your lab test results at the screening visit are not normal.Your hemoglobin level is less than 10 grams per deciliter.Your AST levels are more than three times the normal limit.Your gamma-glutamyl transpeptidase levels are more than three times the upper limit of normal.I have advanced liver disease due to cystic fibrosis.I am at least 2 years old.Your alanine aminotransferase (ALT) levels are more than three times the upper limit of normal.This criterion likely refers to the requirement for certain levels of chemicals in the blood, such as electrolytes, glucose, and liver function tests, to be within a specified range for participation in the clinical trial.
Research Study Groups:
This trial has the following groups:- Group 1: CFTR modulator or other therapies
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
What is the scale of this research endeavor?
"Affirmative. The information shared on clinicaltrials.gov confirms that this research is still recruiting participants, with the original post dating back to May 1st 2021 and updates as recently as April 26th 2022. 50 subjects must be sourced through a single medical centre for successful completion of the trial."
Answered by AI
Are there any opportunities to participate in this clinical experiment still available?
"Affirmative. Information available on clinicaltrials.gov affirms that this medical trial is presently seeking participants; the study first posted in May 2021 and has most recently been modified in April 2022. A total of 50 patients are being recruited from a single test site."
Answered by AI
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