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A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia

Primary Purpose

Aniridia

Status
Withdrawn
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
Ataluren
Sponsored by
PTC Therapeutics
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Aniridia

Eligibility Criteria

2 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria

Participants who will be selected for this study must meet the following criteria:

  1. 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.
  2. Must have participated in and exited from Study PTC124-GD-028 ANI
  3. Age ≥2 years and of either gender.
  4. Body weight ≥12 kg.
  5. Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, and study restrictions.
  6. Good general health.
  7. 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.

    • Hormonal methods of contraception (including oral and transdermal contraceptives, injectable progesterone, progestin subdermal implants, progesterone-releasing intrauterine contraceptive devices [IUDs]) initiated at least 14 days prior to the first dose of study drug
    • Abstinence
    • Placement of a copper-containing IUD
    • Condom with spermicidal foam/gel/film/cream/suppository
    • Postmenopausal at least 12 months prior to first dose of study drug or permanently sterilized (for example, tubal occlusion, hysterectomy, bilateral salpingectomy)
    • Male partner who has had a vasectomy for at least 3 months prior to the first dose of study drug
  8. Male participants with partners of childbearing potential must agree to use the following adequate (at least 1 form of) contraception 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):

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

Exclusion Criteria

The presence of any of the following conditions will exclude a participant from study enrollment:

General exclusion criteria

  1. Participation in any drug or device clinical investigation (other than Study PTC124-GD-028 ANI) within 90 days prior to Visit 1 (Screening) or anticipation of participating in any other drug or device clinical investigation during this study.
  2. Surgery within 30 days prior to enrollment.
  3. Female participants who are pregnant or breastfeeding. Female participants of childbearing potential must have a negative pregnancy test (beta-human chorionic gonadotropin [β-HCG]) at screening and must use adequate (at least 1 form of) contraceptive methods.
  4. Active ocular infection or inflammation.
  5. 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.
  6. Participants with a positive result for hepatitis B, hepatitis C, or human immunodeficiency virus at Visit 1 (Screening).

Sites / Locations

    Arms of the Study

    Arm 1

    Arm Type

    Experimental

    Arm Label

    Ataluren

    Arm Description

    Ataluren Oral suspension taken 3 times per day (10 mg/kg in the morning, 10 mg/kg at mid-day, and 20 mg/kg in the evening).

    Outcomes

    Primary Outcome Measures

    Number of Participants With Treatment-Emergent Adverse Events (TEAEs)

    Secondary Outcome Measures

    Change From Baseline in Visual Acuity at Week 104
    Change From Baseline in Severity of Corneal Keratopathy at Week 104

    Full Information

    First Posted
    September 19, 2019
    Last Updated
    October 3, 2019
    Sponsor
    PTC Therapeutics
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    1. Study Identification

    Unique Protocol Identification Number
    NCT04117880
    Brief Title
    A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia
    Official Title
    Nonsense Mutation Aniridia: An Ataluren (PTC124) Phase 2 Extension Study
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    September 2019
    Overall Recruitment Status
    Withdrawn
    Why Stopped
    Study cancelled before protocol was implemented.
    Study Start Date
    December 31, 2018 (Anticipated)
    Primary Completion Date
    January 31, 2021 (Anticipated)
    Study Completion Date
    January 31, 2021 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    PTC Therapeutics

    4. Oversight

    Studies a U.S. FDA-regulated Drug Product
    Yes
    Studies a U.S. FDA-regulated Device Product
    No

    5. Study Description

    Brief Summary
    This is a Phase 2, multicenter, open-label study evaluating the overall systemic and ocular safety profile of ataluren in nonsense mutation aniridia as determined by the incidences of treatment-emergent adverse events (TEAEs) as well as abnormal findings on laboratory assessments, vital signs, physical examinations, ophthalmoscopy, and slit-lamp examination. Participants who complete PTC124-GD-028 ANI (NCT02647359) meet all inclusion and none of the exclusion criteria will be enrolled into this study.
    Detailed Description
    This study was a planned extension study of PTC124-GD-028 ANI (NCT02647359) and was never initiated.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Aniridia

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 2
    Interventional Study Model
    Single Group Assignment
    Masking
    None (Open Label)
    Allocation
    N/A
    Enrollment
    0 (Actual)

    8. Arms, Groups, and Interventions

    Arm Title
    Ataluren
    Arm Type
    Experimental
    Arm Description
    Ataluren Oral suspension taken 3 times per day (10 mg/kg in the morning, 10 mg/kg at mid-day, and 20 mg/kg in the evening).
    Intervention Type
    Drug
    Intervention Name(s)
    Ataluren
    Other Intervention Name(s)
    PTC124
    Intervention Description
    Oral
    Primary Outcome Measure Information:
    Title
    Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
    Time Frame
    104 weeks
    Secondary Outcome Measure Information:
    Title
    Change From Baseline in Visual Acuity at Week 104
    Time Frame
    104 weeks
    Title
    Change From Baseline in Severity of Corneal Keratopathy at Week 104
    Time Frame
    104 weeks

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    2 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    Inclusion Criteria Participants who will be selected for this study must meet the following 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. Must have participated in and exited from Study PTC124-GD-028 ANI Age ≥2 years and of either gender. Body weight ≥12 kg. Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, and study restrictions. Good general health. 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. Hormonal methods of contraception (including oral and transdermal contraceptives, injectable progesterone, progestin subdermal implants, progesterone-releasing intrauterine contraceptive devices [IUDs]) initiated at least 14 days prior to the first dose of study drug Abstinence Placement of a copper-containing IUD Condom with spermicidal foam/gel/film/cream/suppository Postmenopausal at least 12 months prior to first dose of study drug or permanently sterilized (for example, tubal occlusion, hysterectomy, bilateral salpingectomy) 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 the following adequate (at least 1 form of) contraception 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): Abstinence Vasectomy for at least 3 months prior to first dose of study drug or surgically sterile Without a vasectomy, must use a condom with spermicidal foam/gel/film/cream suppository Exclusion Criteria The presence of any of the following conditions will exclude a participant from study enrollment: General exclusion criteria Participation in any drug or device clinical investigation (other than Study PTC124-GD-028 ANI) within 90 days prior to Visit 1 (Screening) or anticipation of participating in any other drug or device clinical investigation during this study. 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 [β-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).

    12. IPD Sharing Statement

    Learn more about this trial

    A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia

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