Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)

Completed · Phase 3 · Has a placebo group

Conditions studied: Cystic Fibrosis

In brief

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.

Key facts

Study ID

NCT02139306

Run by

PTC Therapeutics

People needed

279

Starts

2014-08-01

Expected to finish

2016-11-01

Last updated by the study team

2020-05-14

Who can join

Age: 6 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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 milliequivalent per liter (mEq/L)
• Documentation of the presence of a nonsense mutation in at least 1 allele of the cystic fibrosis transmembrane conductance regulator (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

You may not qualify if…

• 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 Cystic Fibrosis (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 weeks 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, electrocardiogram (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

Where it is running

• Pulmonary Associates of Mobile PC — Mobile, Alabama, United States
• Miller Children's Hospital Long Beach — Long Beach, California, United States
• Children's Hospital Los Angeles — Los Angeles, California, United States
• Children's Hospital and Research Center at Oakland — Oakland, California, United States
• Stanford University-Children's Hospital — Palo Alto, California, United States
• Children's Hospital Colorado — Aurora, Colorado, United States
• Nemours Children's Clinic — Jacksonville, Florida, United States
• University of Miami — Miami, Florida, United States
• Miami Children's Hospital — Miami, Florida, United States
• All Children's Hospital — St. Petersburg, Florida, United States
• Ann and Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
• Indiana University — Indianapolis, Indiana, United States
• Children's Hospital Boston — Boston, Massachusetts, United States
• Washington University — St Louis, Missouri, United States
• Monmouth Medical Center — Long Branch, New Jersey, United States
• Morristown Medical Center — Morristown, New Jersey, United States
• Beth Israel Medical Center — New York, New York, United States
• New York University Langone Medical Center — New York, New York, United States
• Columbia University Medical Center — New York, New York, United States
• SUNY Upstate Medical University — Syracuse, New York, United States
• University of Cincinnati — Cincinnati, Ohio, United States
• Rainbow Babies & Children's Hospital — Cleveland, Ohio, United States
• Santiago Reyes, MD — Oklahoma City, Oklahoma, United States
• Penn State Milton S. Hershey Medical Center — Hershey, Pennsylvania, United States
• University of Alabama at Birmingham — Birmingham, Alabama, United States

Full record on ClinicalTrials.gov

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