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Study of Ataluren (PTC124) in Cystic Fibrosis

NCT02107859 · Status: TERMINATED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 61

Last updated 2020-03-25

Study results available
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Summary

The primary objective of this study is to determine the long-term safety and tolerability of ataluren in participants with nonsense mutation cystic fibrosis (nmCF) who completed participation in the double-blind study PTC124-GD-009-CF (NCT00803205), as assessed by adverse events and laboratory abnormalities. The secondary objective of this study includes the assessment of the efficacy of ataluren, as measured by forced expiratory volume in 1 second (FEV1) and pulmonary exacerbation rate, and other safety parameters (for example, 12-lead electrocardiogram \[ECG\] measurements, vital signs).

Conditions

Interventions

DRUG

Ataluren

Ataluren will be administered per dose and schedule specified in the arm.

Sponsors & Collaborators

Principal Investigators

  • Joseph McIntosh, MD · PTC Therapeutics, Inc.

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-05-23
Primary Completion
2017-06-05
Completion
2017-06-05

Countries

  • United States
  • Belgium
  • France
  • Israel
  • Italy
  • Spain
  • Sweden

Study Locations

More Related Trials

Entities

Drugs
Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02107859 on ClinicalTrials.gov