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Contact Information:

Study Location:

St James University Hospital

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Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis


Open to: All Genders

Age: 6 Years - N/A

Medical Conditions

Cystic Fibrosis

Study summary

This is an open-label extension study for participants who completed a Phase 3, placebo-controlled study of ataluren in participants with nonsense mutation cystic fibrosis (nmCF) not receiving chronic inhaled aminoglycosides.

The primary objective of this Phase 3 extension study will be to obtain long-term safety data to augment the overall safety database. The secondary objectives will be to augment the efficacy data collected in the double-blind study (PTC124-GD-021-CF; NCT02139306).

Key dates

Start dates may differ between countries and research sites. The research team are responsible for keeping the information up-to-date.  

The recruitment start and end dates are as follows:

Aug 2015

Jun 2017

Study type


Intervention Type : Drug
Intervention Name : Ataluren
Intervention Description : Ataluren will be provided as a vanilla-flavored powder to be mixed with water or milk.

Intervention Arm Group : Ataluren

Who can take part?

You can take part if:

Inclusion Criteria:

- Completion of study treatment (placebo or active) in the previous Phase 3, double-blind study protocol (Protocol PTC124-GD-021-CF)

- Evidence of signed and dated informed consent/assent document(s) indicating that the participant (and/or the participant's parent/legal guardian) has been informed of all pertinent aspects of the trial.

Exclusion Criteria:

- Known hypersensitivity to any of the ingredients or excipients of the study drug.

- Ongoing participation in any other therapeutic clinical trial.

You may not be able to take part if:

This is in the inclusion criteria above

Where can I take part?

Below are the locations for where you can take part in the trial. Please note that not all sites may be open.

  • St James University Hospital
    LS9 7TF
  • Royal Brompton Hospital
    SW3 6NP


The study is sponsored by PTC Therapeutics .

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for Trial ID: NCT02456103

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