Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)
Status: | Unknown status |
---|---|
Conditions: | Pulmonary |
Therapuetic Areas: | Pulmonary / Respiratory Diseases |
Healthy: | No |
Age Range: | 6 - Any |
Updated: | 12/9/2017 |
Start Date: | June 2014 |
End Date: | November 2016 |
A Phase 3 Efficacy and Safety Study of Ataluren (PTC124®) in Patients With Nonsense Mutation Cystic Fibrosis
This is a Phase 3, international, multicenter, randomized, double-blind, placebo-controlled,
efficacy and safety study of ataluren in patients with nonsense mutation cystic fibrosis
(nmCF) not receiving chronic inhaled aminoglycosides.
efficacy and safety study of ataluren in patients with nonsense mutation cystic fibrosis
(nmCF) not receiving chronic inhaled aminoglycosides.
Inclusion Criteria:
- 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 >=6 years.
- Body weight >=16 kg.
- Sweat chloride >60 mEq/L
- Documentation of the presence of a nonsense mutation in at least 1 allele of the CFTR
gene, as determined by genotyping performed at a laboratory certified by the College
of American Pathologists (CAP), or under the Clinical Laboratory Improvement
Act/Amendment (CLIA), or by an equivalent organization
- Verification that a blood sample has been drawn for sequencing of the CFTR gene
- Ability to perform a valid, reproducible spirometry test using the study-specific
spirometer with demonstration of an FEV1 >=40% and <=90% of predicted
- Demonstration at Visit 2 of a valid %-predicted FEV1 within 15% of the Screening %
predicted FEV1 value
- Resting oxygen saturation (as measured by pulse oximetry) >=92% on room air.
- Confirmed screening laboratory values within pre-specified ranges
- In subjects who are sexually active, willingness to abstain from sexual intercourse or
employ a barrier or medical method of contraception during the study drug
administration and 60-day follow-up period
- Willingness and ability to comply with all study procedures and assessments, including
scheduled visits, drug administration plan, study procedures, laboratory tests, and
study restrictions
Exclusion Criteria:
- Known hypersensitivity to any of the ingredients or excipients of the study drug
- Previous participation in the Phase 3 trial of ataluren (PTC124-GD-009-CF).
- 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 4 weeks prior
to screening
- Chronic use of inhaled aminoglycosides (eg, tobramycin) or use of inhaled
aminoglycosides within 4 months prior to screening.
- Exposure to another investigational drug within 4 weeks prior to screening
- Ongoing participation in any other therapeutic clinical trial
- Evidence of pulmonary exacerbation or acute upper or lower respiratory tract infection
(including viral illnesses) within 3 weeks prior to screening
- Treatment with intravenous antibiotics within 3 weeks prior to screening
- Ongoing immunosuppressive therapy (other than corticosteroids)
- Ongoing warfarin, phenytoin, or tolbutamide therapy
- History of solid organ or hematological transplantation
- Major complications of lung disease (including massive hemoptysis, pneumothorax, or
pleural effusion) within 8 weeks prior to screening
- Known portal hypertension
- Positive hepatitis B surface antigen, hepatitis C antibody test, or human
immunodeficiency virus (HIV) test
- Pregnancy or breast-feeding
- Current smoker or a smoking history of >=10 pack-years (number of cigarette packs/day
x number of years smoked).
- Prior or ongoing medical condition (eg, concomitant illness, 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
We found this trial at
31
sites
University of Alabama at Birmingham The University of Alabama at Birmingham (UAB) traces its roots...
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Rainbow Babies and Children's Hospital UH Rainbow Babies & Children’s Hospital is a 244-bed, full-service...
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Indiana University INDIANA UNIVERSITY is a major multi-campus public research institution, grounded in the liberal...
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Nemours Children's Clinic At Nemours Children’s Clinic, Jacksonville, we've treated every child as we would...
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Childrens Hospital Los Angeles Children's Hospital Los Angeles is a 501(c)(3) nonprofit hospital for pediatric...
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Miami Children's Hospital Welcome to Miami Children
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University of Miami A private research university with more than 15,000 students from around the...
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Beth Israel Med Ctr The physicians and staff of Mount Sinai Beth Israel's Heart Institute...
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747 52nd St
Oakland, California 94609
Oakland, California 94609
(510) 428-3000
Children's Hospital and Research Center Oakland For nearly 100 years, Children's Hospital & Research Center...
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Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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Children's Hospital Colorado At Children's Hospital Colorado, we see more, treat more and heal more...
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225 E Chicago Ave
Chicago, Illinois 60611
Chicago, Illinois 60611
(312) 227-4000
Ann & Robert H. Lurie Children's Hospital of Chicago Ann & Robert H. Lurie Children
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University of Cincinnati The University of Cincinnati offers students a balance of educational excellence and...
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500 University Dr
Hershey, Pennsylvania 17033
Hershey, Pennsylvania 17033
(717) 531-6955
Penn State Milton S. Hershey Medical Center Penn State Milton S. Hershey Medical Center, Penn...
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Texas Children's Hospital Texas Children's Hospital, located in Houston, Texas, is a not-for-profit organization whose...
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9000 W Wisconsin Ave #270
Milwaukee, Wisconsin 53226
Milwaukee, Wisconsin 53226
(414) 266-2000
Children's Hospital of Wisconsin Nothing matters more than our children. At Children's Hospital of Wisconsin,...
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New York University Langone Medical Center NYU NYU Langone Medical Center, a world-class, patient-centered, integrated,...
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Columbia University Medical Center Situated on a 20-acre campus in Northern Manhattan and accounting for...
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2900 W Queen Ln
Philadelphia, Pennsylvania 19129
Philadelphia, Pennsylvania 19129
(215) 991-8100
Drexel University College of Medicine Drexel University College of Medicine represents the consolidation of two...
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All Children's Hospital All Children's Hospital provides expert pediatric care for children and families from...
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