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Study of Ataluren in Participants With Nonsense Mutation Aniridia (STAR)

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ClinicalTrials.gov Identifier: NCT02647359
Recruitment Status : Active, not recruiting
First Posted : January 6, 2016
Last Update Posted : June 12, 2019
Sponsor:
Information provided by (Responsible Party):
PTC Therapeutics

Brief Summary:
This study is designed to characterize the systemic and ocular safety profile of ataluren when administered chronically in participants with nonsense mutation aniridia. This study involves a 4-week screening period, a 144-week treatment period (Stage 1: Weeks 1 to 48 [double-masked treatment] and Stage 2: Weeks 49 to 144 [open label treatment]), an optional 96-week open label extension sub-study, and a 4-week post-treatment follow-up period (either study completion or early termination). Participants that choose not to participate in the sub-study will be required to complete the post-treatment follow-up visit at the end of the Stage 2 open-label extension.

Condition or disease Intervention/treatment Phase
Aniridia Drug: Ataluren Drug: Placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 39 participants
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
Actual Study Start Date : January 31, 2016
Estimated Primary Completion Date : February 21, 2022
Estimated Study Completion Date : February 21, 2022

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Ataluren
Participants will receive ataluren orally 3 times a day (TID) at a dose of 10 milligrams per kilogram (mg/kg) in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening for 48 weeks in Stage 1 (double-masked period) and for additional 96 weeks in Stage 2 (open-label extension period). Participants, who complete Stage 2 and agree to continue in open-label sub-study, will continue to receive ataluren treatment at same dose as mentioned above, for 96 weeks or until commercial availability of ataluren for this indication, whichever is first, or until a positive risk-benefit assessment in this indication is not demonstrated.
Drug: Ataluren
Ataluren oral suspension will be administered as per the dose and schedule specified in the respective arms.
Other Names:
  • PTC124
  • Translarna

Placebo Comparator: Placebo
Participants will receive placebo matching to ataluren TID orally in the morning, at midday, and in the evening for 48 weeks in Stage 1 (double-masked period) and ataluren orally TID at a dose of 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening for additional 96 weeks in Stage 2 (open-label extension period). Participants, who complete Stage 2 and agree to continue in open-label sub-study, will continue to receive ataluren treatment at same dose as mentioned above, for 96 weeks or until commercial availability of ataluren for this indication, whichever is first, or until a positive risk-benefit assessment in this indication is not demonstrated.
Drug: Ataluren
Ataluren oral suspension will be administered as per the dose and schedule specified in the respective arms.
Other Names:
  • PTC124
  • Translarna

Drug: Placebo
Placebo will be administered as per the schedule specified in the respective arm.




Primary Outcome Measures :
  1. Number of Participants With Treatment-Emergent Adverse Events (TEAEs) [ Time Frame: Day 1 to Week 240 ]

Secondary Outcome Measures :
  1. Change From Baseline in Visual Acuity at Week 48 [ Time Frame: Baseline, Week 48 ]
  2. Change From Baseline in Severity of Corneal Keratopathy at Week 48 [ Time Frame: Baseline, Week 48 ]
  3. Change From Baseline in Iris Area at Week 48 [ Time Frame: Baseline, Week 48 ]
  4. Change From Baseline in Visual Acuity at Week 240 (Sub-Study End of Treatment Visit) [ Time Frame: Baseline, Week 240 ]
    This endpoint will be assessed only for the cohort of participants who enroll into the sub-study.



Information from the National Library of Medicine

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

Inclusion Criteria:

  • Evidence of signed and dated informed consent document(s) indicating that the study candidate (and/or a parent/legal guardian) has been informed of all pertinent aspects of the study. Note: If the study candidate is considered a child under local regulation, a parent or legal guardian must provide written consent prior to initiation of study screening procedures and the study candidate may be required to provide written assent. The rules of the responsible institutional review board/independent ethics committee (IRB/IEC) regarding whether one or both parents must provide consent and the appropriate ages for obtaining consent and assent from the participant should be followed.
  • Body weight greater than or equal to (>=) 12 kilograms (kg).
  • Documentation of the presence of a nonsense mutation in 1 allele of the PAX6 gene as determined by genotyping performed at a laboratory certified by the College of American Pathologists (CAP), or under the Clinical Laboratory Improvement Act/Amendment (CLIA), or by an equivalent organization.
  • Clinical diagnosis of aniridia.
  • Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, and study restrictions.
  • Good general health, as determined at Visit 1 (Screening) by medical history and physical examination (including vital sign measurements).
  • No clinically significant abnormality based upon laboratory assessments at Visit 1 (Screening), in the opinion of the investigator.
  • Female participants of childbearing potential are eligible for the study but must be willing to use adequate (at least 1 form of) contraceptive methods as described below during the study treatment period (starting from the day of first dose of study drug and ending 60 days after the last dose of study drug). Childbearing potential is defined as participants who have experienced menarche and who are neither postmenopausal or have been permanently sterilized.

    1. Hormonal methods of contraception (including oral and transdermal contraceptives, injectable progesterone, progestin subdermal implants, progesterone-releasing intrauterine devices [IUDs]) initiated at least 14 days prior to the first dose of study drug
    2. Abstinence
    3. Placement of a copper-containing IUD
    4. Condom with spermicidal foam/gel/film/cream/suppository
    5. Postmenopausal at least 12 months prior to first dose of study drug or permanently sterilized (for example, tubal occlusion, hysterectomy, bilateral salpingectomy)
    6. Male partner who has had a vasectomy for at least 3 months prior to the first dose of study drug
  • Male participants with partners of childbearing potential must agree to use adequate (at least 1 form of) contraception as described below during the study treatment period (starting from the day of first dose of study drug and ending 60 days after the last dose of study drug).

    1. Abstinence
    2. Vasectomy for at least 3 months prior to first dose of study drug or surgically sterile
    3. Without a vasectomy, must use a condom with spermicidal foam/gel/film/cream suppository

Exclusion Criteria:

  • Participants participating in any drug or device clinical investigation within 90 days prior to Visit 1 (Screening) or who anticipate participating in any other drug or device clinical investigation within the duration of this study.
  • Exposure to ataluren within 90 days prior to Visit 1 (Screening).
  • Surgery within 30 days prior to enrollment.
  • Female participants who are pregnant or breastfeeding. Female participants of childbearing potential must have a negative pregnancy test (beta-human chorionic gonadotropin [beta-HCG]) at screening and must use adequate (at least 1 form of) contraceptive methods.
  • Active ocular infection or inflammation.
  • Prior or ongoing medical condition (for example, concomitant illness, alcoholism, drug abuse, psychiatric condition), medical history, physical findings, or laboratory abnormality that, in the investigator's opinion, could adversely affect the safety of the participant, makes it unlikely that the course of study drug administration or follow-up would be completed, or could impair the assessment of study results.
  • Participants with a positive result for hepatitis B, hepatitis C, or human immunodeficiency virus at Visit 1 (Screening).
  • Ongoing warfarin, phenytoin, or tolbutamide therapy.
  • Ongoing intravenous (IV) aminoglycoside or IV vancomycin use.
  • Ongoing systemic cyclosporine therapy. Note: Topical cyclosporine therapy is permitted.
  • Known hypersensitivity to any of the ingredients or excipients of the study drug (polydextrose, polyethylene glycol 3350, poloxamer 407, mannitol 25C, crospovidone XL10, hydroxyethyl cellulose, vanilla, colloidal silica, or magnesium stearate).
  • 20/200 or worse visual acuity in the better eye with best correction.
  • Participants who are monocular.
  • Participants with a history of complications due to ocular surgery that could interfere with the study procedures or assessment of study endpoints.
  • Participants with any other significant ocular or systemic disease that the Investigator determines could interfere with the study.

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
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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
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Study Director: Quintus Ngumah, OD, PhD PTC Therapeutics

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Responsible Party: PTC Therapeutics
ClinicalTrials.gov Identifier: NCT02647359     History of Changes
Other Study ID Numbers: PTC124-GD-028 ANI
First Posted: January 6, 2016    Key Record Dates
Last Update Posted: June 12, 2019
Last Verified: May 2019

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