An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis Pigmentosa (HELIA)
Retinitis Pigmentosa, Usher Syndrome Type 2
About this trial
This is an interventional treatment trial for Retinitis Pigmentosa focused on measuring 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
Eligibility Criteria
Principal Inclusion Criteria:
- 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
- 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:
- 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.
- 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.
- 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.
Sites / Locations
- Center for Clinical Research Operations, Massachusetts Eye and Ear
- University of Michigan, Kellogg Eye Center
- Casey Eye Institute, Oregon Health & Science University
- Retina Foundation of the Southwest
- Centre for Innovative Medicine, Department of Paediatric Surgery, Montreal Children's Hospital at the McGill University Health Centre
- Hôpital Gui de Chauliac - CHRU de Montpellier - Maladies Sensorielles Génétique
- Centre de maladies rares CHNO des Quinze Vingts
Arms of the Study
Arm 1
Other
QR421a RNA antisense oligonucleotide for intravitreal injection
There is only one treatment arm in the PQ-421a-002 study; All participants that are eligible to be dosed will receive QR-421a in an open label fashion.