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Tests treatment safety and results for Cystic Fibrosis
Official title Testing Drug Efficacy in Cystic Fibrosis Through N-of-1 Trials
ClinicalTrials.gov ID: NCT04580368
What this study is testing
What is CFTR Modulators?
CFTR Modulators is an investigational medicine, being studied as a potential treatment for cystic fibrosis.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- You might receive a placebo (an inactive treatment) instead of the study drug. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 6 and older
You may be able to join if
- Signed informed consent (and assent when applicable)
- Willing and able to adhere to the study visit schedule and protocol requirements
- Male or Female ≥6 years old and within the FDA-approved range for the proposed modulator drug
- Ivacaftor: ≥4 months old
- Lumacaftor/Ivacaftor: 2 years old
You likely can't join if
- Presence of any condition or abnormality that, in the opinion of the Investigator, would compromise the safety of the patient and/or quality of the...
- For women of child bearing potential:
- Positive pregnancy test or known pregnancy at Visit 1
- Lactating
- Unwilling to practice a medically acceptable form of contraception (acceptable forms include abstinence, hormonal birth control, intrauterine device...
- BMI \< 10th percentile for age (if \<18 years old) or \< 20kg/m2 (if ≥18 years old)
See the full eligibility criteria
- Signed informed consent (and assent when applicable)
- Willing and able to adhere to the study visit schedule and protocol requirements
- Male or Female ≥6 years old and within the FDA-approved range for the proposed modulator drug
- Ivacaftor: ≥4 months old
- Lumacaftor/Ivacaftor: 2 years old
- Tezacaftor/Ivacaftor: 12 years old
- Elexacaftor/Tezacaftor/Ivacaftor: ≥12 years old
- At least one rare CFTR variant (incidence of \<5% of the CF population)
- Documentation of a CF diagnosis as evidenced by one or more clinical features of CF plus at least one of the following:
- Sweat Chloride ≥60mmol/L by quantitative pilocarpine iontophoresis
- Two mutations in the CFTR gene
- Abnormal nasal potential difference (NPD) testing supportive of a CF diagnosis
- FEV1 \> 50% predicted for age
- Stable chronic CF therapies with no changes in \>28 days (except for chronic cycled inhaled antibiotics such as tobramycin)
- Prescribed CFTR modulator by a licensed physician
- No contraindication to treatment with the selected drug at the time of treatment initiation
- Presence of any condition or abnormality that, in the opinion of the Investigator, would compromise the safety of the patient and/or quality of the data
- For women of child bearing potential:
- Positive pregnancy test or known pregnancy at Visit 1
- Lactating
- Unwilling to practice a medically acceptable form of contraception (acceptable forms include abstinence, hormonal birth control, intrauterine device, or barrier method plus a spermicidal agent), unless surgically...
- BMI \< 10th percentile for age (if \<18 years old) or \< 20kg/m2 (if ≥18 years old)
- FEV1 ≤ 50% predicted for age
- Growth of CF pathogens from sputum cultures that are associated with unstable disease (e.g., nontuberculous mycobacteria, Burkholderia spp) within six months of enrollment
- Concomitant use of CYP3A inducers or inhibitors (e.g., voriconazole, fluconazole, rifampin) or prednisone (\>20mg daily)
- Concomitant conditions:
- Poorly controlled diabetes mellitus (HbA1c \>8.5 or glucosuria as noted below)
- Advanced CF liver disease (cirrhosis with portal hypertension, ascites, or abnormal liver laboratory testing as noted below)
- End stage renal disease
- History of organ transplantation
- Additional medical conditions that in the opinion of the Investigator place the patient at risk of participation or may impact the patient's ability to complete the trial (e.g., uncontrolled depression, anxiety...
- Any of the following abnormal laboratory values at the Screening Visit:
- WBC \>15,000 K/mcL or ANC \<1,500 K/mcL
- Hemoglobin \<10 gm/dL
- Platelets \<50,000 K/mcL
- Chemistries
- \>2+ Glucosuria
- Clinically significant abnormalities as assessed by the Investigator
- Glomerular filtration rate ≤50 mL/min/1.73 m2 (calculated by the Counahan-Barratt equation)
- Hepatic Function Testing / Coagulation Testing
- ≥3 × upper limit of normal (ULN) aspartate aminotransferase (AST)
- ≥3 × ULN alanine aminotransferase (ALT)
- ≥3 × ULN gamma-glutamyl transpeptidase
- Total or direct bilirubin \>2 × ULN
- INR \> 1.5 x ULN
- Positive pregnancy test
The study team makes the final eligibility decision.
Where it's taking place
- Cincinnati, Ohio, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 6 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Cincinnati, Ohio, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.