Status:
COMPLETED
Evaluation of Ivacaftor in Patients Using Ataluren for Nonsense Mutations
Lead Sponsor:
University of Alabama at Birmingham
Conditions:
Cystic Fibrosis
Eligibility:
All Genders
19+ years
Phase:
PHASE4
Brief Summary
The purpose of this study is to explore the combination of Ataluren and ivacaftor as a treatment for patients with a specific cystic fibrosis mutation
Detailed Description
In about 10% of patients with CF, the defect in the gene is known as a stop mutation. This mutation truncates the cystic fibrosis transductive regulator (CFTR) protein production by introducing a prem...
Eligibility Criteria
Inclusion
- Evidence of signed and dated informed consent/assent document(s) indicating that the subject (and/or his parent/legal guardian) has been informed of all pertinent aspects of the trial.
- Age ≥19 years
- Body weight ≥16 kg
- Diagnosis of cystic fibrosis and documentation of the presence of a nonsense mutations of the CFTR gene, as determined by historical genotyping
- Ability to perform a valid, reproducible spirometry with demonstration of a forced expiratory volume in 1second (FEV1) ≥30% of predicted for age, gender, and height.
- If the subject is sexually active, willingness to abstain from sexual intercourse or employ a barrier or medical method of contraception during the study drug administration
- Willingness and ability to comply with all study procedures and assessments.
- Currently receiving Ataluren for nonsense mutations through other clinical trial access.
Exclusion
- Any change (initiation, change in type of drug, dose modification, schedule modification, interruption, discontinuation, or re-initiation) in a chronic treatment/prophylaxis regimen for CF or for CF-related conditions within 2 weeks prior to screening.
- Evidence of pulmonary exacerbation or acute upper or lower respiratory tract infection (including viral illnesses) within 2 weeks prior to screening.
- Ongoing immunosuppressive therapy (other than corticosteroids up to 10mg/d equivalent of prednisone)
- Ongoing warfarin, phenytoin, or tolbutamide therapy.
- History of solid organ or hematological transplantation.
- A history of positive hepatitis B surface antigen test, hepatitis C antibody test, or human immunodeficiency
- Major complications of lung disease (including massive hemoptysis, pneumothorax, or pleural effusion) within 4 weeks prior to screening.
- Pregnancy or breast-feeding.
- Current smoker or a smoking history of ≥10 pack-years (number of cigarette packs/day × number of years smoked).
- Prior or ongoing medical condition (eg, renal failure, alcoholism, drug abuse, psychiatric condition), medical history, physical findings, ECG findings, or laboratory abnormality that, in the investigator's opinion, could adversely affect the safety of the subject, makes it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results.
- \-
Key Trial Info
Start Date :
March 17 2017
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
February 16 2018
Estimated Enrollment :
1 Patients enrolled
Trial Details
Trial ID
NCT03256799
Start Date
March 17 2017
End Date
February 16 2018
Last Update
July 19 2019
Active Locations (1)
Enter a location and click search to find clinical trials sorted by distance.
1
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35233