An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis Pigmentosa
Condition: Retinitis Pigmentosa; Usher Syndrome Type 2
MeSH terms: Usher Syndromes; Retinitis; Retinitis Pigmentosa
Intervention: RNA antisense oligonucleotide for intravitreal injection
Intervention description: QR-421a will be first administered to the fellow eye (as defined in the preceding study), and will be repeated every 6 months.
Treatment of the study eye (as defined in the preceding study) can commence 3 months (9 months for subjects from study PQ-421a-001) after the treatment of the fellow eye has been initiated and will be repeated every 6 months as well.
Continued subject treatment in this study will be pursued provided that the benefit-risk balance is positive for the individual subject.
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Intervention model description: Eligible participants from previous QR-421a study can be enrolled into this extension study to receive continuous dosing of QR-421a for 24 months or until treatment becomes available (whichever is longer). Subject who receive follow up only, will be in this study for 12 months.
Masking: None (Open Label)
Enrollment: 21.0
Sex: All
Minimum age: 12 Years
Healthy volunteers: No
Keywords: QR-421a; Retinitis Pigmentosa; Usher Syndrome; Exon 13 Mutation; USH2A gene; Night Blindness; Usherin protein; Deaf Blind; Retinal Disease; Eye Disease; Vision Disorders; Congenital, Familial and Genetic Disorders; PQ-421a-002

Summary:

      PQ-421a-002 (Helia) is an open-label, extension study to evaluate the safety, tolerability
      and efficacy of QR 421a (ultevursen) administered via intravitreal (IVT) injection in one or
      both eyes, in subjects ≥ 12 years of age with RP due to mutations in exon 13 of the USH2A
      gene, for an anticipated period of 24 months, or until provision of continued treatment by
      other means is available, provided the subject's benefit-risk determination remains positive.
    

Eligibility criteria:

        Principal Inclusion Criteria:

          1. Subjects who have participated in a preceding QR-421a study and who may derive benefit
             from continued treatment with QR 421a, and/or continued follow up, as assessed by the
             Investigator, in consultation and agreement with the Medical Monitor

          2. An adult (≥ 18 years) willing and able to provide informed consent for participation
             prior to performing any study related procedures, and suitable verbal, auditory,
             written and/or tactile sign language communication as to allow informed consent to be
             obtained, in the opinion of the Investigator.

        OR A minor (12 to < 18 years) able to provide age-appropriate assent for study
        participation, and with a parent or legal guardian willing and able to provide written
        permission for the subject's participation prior to performing any study related
        procedures.

        Principal Exclusion Criteria:

          1. Presence of any significant ocular or non-ocular disease/disorder (or medication
             and/or laboratory test abnormalities) which, in the opinion of the Investigator and
             with concurrence of the Medical Monitor, may either put the subject at risk because of
             participation in the study, may influence the results of the study, or the subject's
             ability to participate in the study. This includes but is not limited to a subject who
             has uncontrolled cystoid macular edema (CME) in the treatment eye. CME is permissible
             if stable for 3 months (with or without treatment). Past CME is permissible if
             resolved for more than 1 month.

          2. Receipt within 3 months prior to Screening of any intraocular or periocular surgery
             (including refractive surgery), or an IVT injection or planned intraocular surgery or
             procedure during the course of the study.

          3. Safety issue during preceding QR-421a study that may compromise subject safety when
             continued dosing, as determined by the Investigator, and in consultation with the
             Medical Monitor.