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

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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.


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
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1720 2nd Ave S
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425 University Blvd.
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807 Childrens Way
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4650 Sunset Blvd
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3100 SW 62nd Ave
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South 34th Street
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225 E Chicago Ave
Chicago, Illinois 60611
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2600 Clifton Ave
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500 University Dr
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