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Study of Ataluren in Patients With Aniridia (STAR)

This study is currently recruiting participants. (see Contacts and Locations)
Verified December 2016 by PTC Therapeutics
Information provided by (Responsible Party):
PTC Therapeutics Identifier:
First received: December 29, 2015
Last updated: December 13, 2016
Last verified: December 2016
This is a Phase 2, randomized, double-masked, placebo-controlled study of ataluren in patients with aniridia caused by a nonsense mutation. Patients will receive masked study drug for 48 weeks followed by open-label ataluren for another 48 weeks. Safety and efficacy will be assessed.

Condition Intervention Phase
Drug: Ataluren
Drug: Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double Blind (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: STAR: A Phase 2, Multicenter, Randomized, Double Masked, Placebo Controlled Study of the Safety and Efficacy of Ataluren (PTC124) for the Treatment of Nonsense Mutation Aniridia

Resource links provided by NLM:

Further study details as provided by PTC Therapeutics:

Primary Outcome Measures:
  • Safety as assessed by systemic and ocular adverse events [ Time Frame: 96 weeks ]

Secondary Outcome Measures:
  • Visual acuity as assessed by the Early Treatment Diabetic Retinopathy Study (ETDRS) chart [ Time Frame: 48 weeks ]
  • Severity of corneal keratopathy as assessed by slit lamp examination and macro photography [ Time Frame: 48 weeks ]
  • Iris area [ Time Frame: 48 weeks ]

Estimated Enrollment: 36
Study Start Date: January 2016
Estimated Study Completion Date: June 2018
Estimated Primary Completion Date: June 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Ataluren
Ataluren taken orally at a dose of 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening.
Drug: Ataluren
Other Names:
  • PTC124
  • Translarna
Placebo Comparator: Placebo
Matching placebo taken orally in the morning, at midday, and in the evening.
Drug: Placebo


Ages Eligible for Study:   2 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Age ≥2 years
  • Body weight ≥12 kg
  • Documentation of the presence of a nonsense mutation in 1 allele of the PAX6 gene
  • Clinical diagnosis of aniridia

Exclusion Criteria:

  • 20/200 or worse visual acuity in the better eye with best correction.
  • Subjects who are monocular.
  • Subjects with a history of complicated ocular surgery
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT02647359

Contact: Mary Frances Harmon

United States, Indiana
Price Vision Center Not yet recruiting
Indianapolis, Indiana, United States, 46260
Contact: Francis Price         
Principal Investigator: Francis Price, MD         
United States, Oregon
Casey Eye Institute, Oregon Health & Science University Not yet recruiting
Portland, Oregon, United States, 97239
Contact: Brenda Purvis         
Principal Investigator: Winston Chamberlain, MD         
United States, Virginia
University of Virginia Recruiting
Charlottesville, Virginia, United States, 22908
Contact: Asima Bajwa         
Principal Investigator: Peter Netland, MD         
Canada, British Columbia
University of British Columbia Recruiting
Vancouver, British Columbia, Canada, V5Z3N9
Contact: Aleksandra Kuzmanovic         
Principal Investigator: Kevin Gregory-Evans, MD, PhD         
Sponsors and Collaborators
PTC Therapeutics
Study Director: Quintus Ngumah, OD, PhD PTC Therapeutics
  More Information

Responsible Party: PTC Therapeutics Identifier: NCT02647359     History of Changes
Other Study ID Numbers: PTC124-GD-028 ANI
Study First Received: December 29, 2015
Last Updated: December 13, 2016

Additional relevant MeSH terms:
Eye Abnormalities
Eye Diseases
Eye Diseases, Hereditary
Iris Diseases
Uveal Diseases
Congenital Abnormalities
Genetic Diseases, Inborn processed this record on April 21, 2017