NCT02139306

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

Completed Phase 3 Results posted Last updated 4 May 2020

What this trial tests

Phase 3 trial testing Ataluren (PTC124®) in Cystic Fibrosis in 279 participants. Completed in 1 November 2016.

Timeline

1 August 2014

Primary endpoint
1 November 2016

1 November 2016

Quick facts

Lead sponsor	PTC Therapeutics
Phase	Phase 3
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	quadruple
Primary purpose	treatment
Enrollment	279
Start date	1 August 2014
Primary completion	1 November 2016
Estimated completion	1 November 2016
Sites	88 locations across United States, Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, France

Drugs / interventions tested

• Ataluren (PTC124®) — full drug profile →
• Placebo

Conditions studied

• Cystic Fibrosis — all drugs for Cystic Fibrosis →

Sponsor

PTC Therapeutics — full company profile →

Who can join

6 and older, any sex, with Cystic Fibrosis. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Absolute Change From Baseline in Percent-predicted Forced Expiratory Volume in One Second (ppFEV1) at Week 48
  Time frame: From Baseline to Week 48
  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.

Sponsor's own description

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.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. CFTR Modulators: The Changing Face of Cystic Fibrosis in the Era of Precision Medicine.
   Lopes-Pacheco M. · · 2019 · cited 349× · PMID 32153386 · DOI 10.3389/fphar.2019.01662
2. Engineered transfer RNAs for suppression of premature termination codons.
   Lueck JD, Yoon JS, Perales-Puchalt A, Mackey AL, et al · · 2019 · cited 128× · PMID 30778053 · DOI 10.1038/s41467-019-08329-4
3. Advances in therapeutic use of a drug-stimulated translational readthrough of premature termination codons.
   Dabrowski M, Bukowy-Bieryllo Z, Zietkiewicz E. · · 2018 · cited 122× · PMID 30134808 · DOI 10.1186/s10020-018-0024-7
4. CFTR Modulators: Shedding Light on Precision Medicine for Cystic Fibrosis.
   Lopes-Pacheco M. · · 2016 · cited 108× · PMID 27656143 · DOI 10.3389/fphar.2016.00275
5. New and emerging targeted therapies for cystic fibrosis.
   Quon BS, Rowe SM. · · 2016 · cited 85× · PMID 27030675 · DOI 10.1136/bmj.i859
6. Nonsense suppression therapies in human genetic diseases.
   Martins-Dias P, Romão L. · · 2021 · cited 70× · PMID 33751142 · DOI 10.1007/s00018-021-03809-7
7. Efficacy and safety of ataluren in patients with nonsense-mutation cystic fibrosis not receiving chronic inhaled aminoglycosides: The international, randomized, double-blind, placebo-controlled Ataluren Confirmatory Trial in Cystic Fibrosis (ACT CF).
   Konstan MW, VanDevanter DR, Rowe SM, Wilschanski M, et al · · 2020 · cited 49× · PMID 31983658 · DOI 10.1016/j.jcf.2020.01.007
8. Strategies for the etiological therapy of cystic fibrosis.
   Maiuri L, Raia V, Kroemer G. · · 2017 · cited 39× · PMID 28937684 · DOI 10.1038/cdd.2017.126