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Completed Phase 3 Interventional Results available

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

ClinicalTrials.gov ID: NCT02139306

Public ClinicalTrials.gov record NCT02139306. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Oct 28, 2026, 5:24 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

A Phase 3 Efficacy and Safety Study of Ataluren (PTC124®) in Patients With Nonsense Mutation Cystic Fibrosis

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

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.

Study identification

NCT ID
NCT02139306
Recruitment status
Completed
Study type
Interventional
Phase
Phase 3
Lead sponsor
PTC Therapeutics
Industry
Enrollment
279 participants

Conditions and interventions

Interventions

Drug

Eligibility (public fields only)

Age range
6 Years and older
Sex
All
Healthy volunteers
Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Aug 2014 (Actual)
Primary completion
Nov 2016 (Actual)
Completion
Nov 2016 (Actual)
Last update posted
May 14, 2020

2014 – 2016

United States locations

U.S. sites
30
U.S. states
15
U.S. cities
26
Facility City State ZIP Site status
University of Alabama at Birmingham Birmingham Alabama 35233 —
Pulmonary Associates of Mobile PC Mobile Alabama 36608 —
Miller Children's Hospital Long Beach Long Beach California 90806 —
Children's Hospital Los Angeles Los Angeles California 90027 —
Children's Hospital and Research Center at Oakland Oakland California 94609 —
Stanford University-Children's Hospital Palo Alto California 94304 —
Children's Hospital Colorado Aurora Colorado 80045 —
Nemours Children's Clinic Jacksonville Florida 32207 —
University of Miami Miami Florida 33136 —
Miami Children's Hospital Miami Florida 33155 —
All Children's Hospital St. Petersburg Florida 33701 —
Ann and Robert H. Lurie Children's Hospital of Chicago Chicago Illinois 60611 —
Indiana University Indianapolis Indiana 46202 —
Children's Hospital Boston Boston Massachusetts 02115 —
Washington University St Louis Missouri 63110 —
Monmouth Medical Center Long Branch New Jersey 07740 —
Morristown Medical Center Morristown New Jersey 07960 —
Beth Israel Medical Center New York New York 10003 —
New York University Langone Medical Center New York New York 10016 —
Columbia University Medical Center New York New York 10032 —
SUNY Upstate Medical University Syracuse New York 13210 —
University of Cincinnati Cincinnati Ohio 45221 —
Rainbow Babies & Children's Hospital Cleveland Ohio 44106 —
Santiago Reyes, MD Oklahoma City Oklahoma 73112 —
Penn State Milton S. Hershey Medical Center Hershey Pennsylvania 17033 —
Children's Hospital of Philadelphia Philadelphia Pennsylvania 19104 —
Drexel University College of Medicine Philadelphia Pennsylvania 19129 —
Texas Children's Hospital Houston Texas 77094 —
University of Texas Health Science Center Tyler Texas 75708 —
Childrens Hospital of Wisconsin Milwaukee Wisconsin 53226 —

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

Non-U.S. locations

This page focuses on the U.S. directory. The official record also lists 58 non-U.S. sites.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT02139306, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted May 14, 2020 · Synced Oct 28, 2026

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Open the official record

The complete public registry record, listed eligibility criteria, and available contact information for NCT02139306 live on ClinicalTrials.gov.

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