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NCT02139306 · ClinicalTrials.gov registry record · Phase 3

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

A Phase 3 study of Cystic Fibrosis, sponsored by PTC Therapeutics.

Completed
Registry status
Phase 3
Development phase
279
Enrollment target
20
Study locations

NCT02139306: Completed Phase 3 study of Cystic Fibrosis, sponsored by PTC Therapeutics.

NCT02139306 is a Phase 3 study of Cystic Fibrosis that has completed, run by PTC Therapeutics. The registered enrollment target is 279 participants, above the 178-participant average among 169 other Cystic Fibrosis trials with a reported enrollment target (57% higher). The trial reports 20 study locations across 10 states. According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT02139306, a Phase 3 study of Cystic Fibrosis, has completed, sponsored by PTC Therapeutics.

COMPLETED
Registry status
Phase 3
Development phase
279 participants
Enrollment target
20
Study locations

Study Summary

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.

Primary Outcome

The FEV1 is the volume of air forcibly exhaled in one second and is measured using forced expiratory air spirometry. Change in ppFEV1 at Week 48 was defined as the average between the change from baseline at Week 40 and that at Week 48. Baseline for ppFEV1 was defined as an average of ppFEV1 at Screening (Weeks -4 to -1) and Baseline (Day 1) visits.

Conditions Studied

Interventions

  • DRUG Placebo
  • DRUG Ataluren (PTC124®)

Study Locations (20)

California

  • Miller Children's Hospital Long Beach - Long Beach
  • Children's Hospital Los Angeles - Los Angeles
  • Children's Hospital and Research Center at Oakland - Oakland
  • Stanford University-Children's Hospital - Palo Alto

Florida

  • Nemours Children's Clinic - Jacksonville
  • University of Miami - Miami
  • Miami Children's Hospital - Miami
  • All Children's Hospital - St. Petersburg

New York

  • Beth Israel Medical Center - New York
  • New York University Langone Medical Center - New York
  • Columbia University Medical Center - New York

Alabama

  • University of Alabama at Birmingham - Birmingham
  • Pulmonary Associates of Mobile PC - Mobile

New Jersey

  • Monmouth Medical Center - Long Branch
  • Morristown Medical Center - Morristown

Colorado

  • Children's Hospital Colorado - Aurora

Illinois

  • Ann and Robert H. Lurie Children's Hospital of Chicago - Chicago

Indiana

  • Indiana University - Indianapolis

Trial Details

FieldValue
Enrollment Target 279 participants
Start Date 2014-08
Est. Completion 2016-11
Phase Phase 3
PTC Therapeutics

52 total trials

What the finished NCT02139306 record still lists

NCT02139306 is an interventional study that assigns participants to a tested intervention. The registered 279 participants enrollment target is mid-sized for trials with a published cap, above the 178-participant average among 169 other Cystic Fibrosis trials with a reported enrollment target (57% higher).

The record links to 1 condition, with Cystic Fibrosis appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.

NCT02139306 names 20 study sites across 10 states, led by California, Florida, New York.

Frequently Asked Questions

What is clinical trial NCT02139306 about?

NCT02139306 is a clinical study titled "Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)". 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.

What is the current status of trial NCT02139306?

This trial is currently completed. It is a Phase 3 study. The enrollment target is 279 participants. The study started on 2014-08. Estimated completion is 2016-11.

What conditions does trial NCT02139306 study?

This clinical trial studies the following conditions: Cystic Fibrosis.

What interventions are being tested in trial NCT02139306?

The interventions under investigation include: Placebo (DRUG), Ataluren (PTC124®) (DRUG).

Who is sponsoring clinical trial NCT02139306?

This trial is sponsored by PTC Therapeutics, which has 52 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT02139306 being conducted?

This trial has 20 study locations across Alabama, California, Colorado, Florida, Illinois. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

Similar trials for Cystic Fibrosis

Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.

Where NCT02139306's enrollment target sits among peer trials

279 24th of 169 higher than 146 of 169 other Cystic Fibrosis trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other Cystic Fibrosis trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.

Source ClinicalTrials.gov registry export · 2026-08-08

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Source: ClinicalTrials.gov NCT02139306, the US trial registry maintained by the National Library of Medicine. NCT02139306 (mid enrollment · wide site footprint · completed) retrieved and formatted by PlainTrial, see methodology.