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Study of Ataluren in Patients With Aniridia (STAR)

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02647359
First Posted: January 6, 2016
Last Update Posted: August 3, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
PTC Therapeutics
  Purpose
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
Aniridia Drug: Ataluren Drug: Placebo Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (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 ]

Enrollment: 39
Actual Study Start Date: January 2016
Estimated Study Completion Date: December 2019
Estimated Primary Completion Date: December 2019 (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

  Eligibility

Information from the National Library of Medicine

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, Learn About Clinical Studies.


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

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
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02647359


Locations
United States, Oregon
Casey Eye Institute, Oregon Health & Science University
Portland, Oregon, United States, 97239
United States, Virginia
University of Virginia
Charlottesville, Virginia, United States, 22908
Canada, British Columbia
University of British Columbia
Vancouver, British Columbia, Canada, V5Z3N9
Sponsors and Collaborators
PTC Therapeutics
Investigators
Study Director: Quintus Ngumah, OD, PhD PTC Therapeutics
  More Information

Responsible Party: PTC Therapeutics
ClinicalTrials.gov Identifier: NCT02647359     History of Changes
Other Study ID Numbers: PTC124-GD-028 ANI
First Submitted: December 29, 2015
First Posted: January 6, 2016
Last Update Posted: August 3, 2017
Last Verified: August 2017

Additional relevant MeSH terms:
Aniridia
Eye Abnormalities
Eye Diseases
Eye Diseases, Hereditary
Iris Diseases
Uveal Diseases
Congenital Abnormalities
Genetic Diseases, Inborn