Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
Register:
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ClinicalTrials.gov |
Last refreshed on:
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12 December 2020 |
Main ID: |
NCT03256799 |
Date of registration:
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18/08/2017 |
Prospective Registration:
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No |
Primary sponsor: |
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Public title:
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Evaluation of Ivacaftor in Patients Using Ataluren for Nonsense Mutations
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Scientific title:
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An Open Label Study to Investigate the Role of Ivacaftor for the Treatment of Cystic Fibrosis in Combination With Ataluren (PTC124) in Cystic Fibrosis Patients Using Ataluren for Nonsense Mutations |
Date of first enrolment:
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March 17, 2017 |
Target sample size:
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1 |
Recruitment status: |
Completed |
URL:
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https://clinicaltrials.gov/show/NCT03256799 |
Study type:
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Interventional |
Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 4
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Countries of recruitment
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United States
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Contacts
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Name:
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Steven M Rowe, MD |
Address:
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Telephone:
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Email:
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Affiliation:
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University of Alabama at Birmingham |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
1. 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.
2. Age =19 years
3. Body weight =16 kg
4. Diagnosis of cystic fibrosis and documentation of the presence of a nonsense mutations
of the CFTR gene, as determined by historical genotyping
5. 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.
6. 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
7. Willingness and ability to comply with all study procedures and assessments.
8. Currently receiving Ataluren for nonsense mutations through other clinical trial
access.
Exclusion Criteria:
1. 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.
2. Evidence of pulmonary exacerbation or acute upper or lower respiratory tract infection
(including viral illnesses) within 2 weeks prior to screening.
3. Ongoing immunosuppressive therapy (other than corticosteroids up to 10mg/d equivalent
of prednisone)
4. Ongoing warfarin, phenytoin, or tolbutamide therapy.
5. History of solid organ or hematological transplantation.
6. A history of positive hepatitis B surface antigen test, hepatitis C antibody test, or
human immunodeficiency
7. Major complications of lung disease (including massive hemoptysis, pneumothorax, or
pleural effusion) within 4 weeks prior to screening.
8. Pregnancy or breast-feeding.
9. Current smoker or a smoking history of =10 pack-years (number of cigarette packs/day ×
number of years smoked).
10. 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.
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Age minimum:
19 Years
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Cystic Fibrosis
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Intervention(s)
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Drug: Ivacaftor/Ataluren
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Primary Outcome(s)
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Lung Function
[Time Frame: Baseline through 48 weeks]
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Secondary ID(s)
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F161208009
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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