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This study will evaluate the safety and efficacy of a recombinant adeno-associated virus vector (rAAV2tYF-GRK1-RPGR) in patients with X-linked retinitis pigmentosa caused by RPGR mutations.
Full description
Approximately 12 participants, who were not part of the Phase 1/2 (HORIZON) study, will be enrolled into the dose expansion portion of the study. These participants will be randomized in a 1:1 ratio to 1 of 2 treatment groups (i.e., Group 1 [low dose] and Group 2 [high dose]). Each participant will receive the assigned dose of AGTC-501 in one eye on a single occasion.
Enrollment
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Inclusion criteria
Exclusion criteria
Have other known disease-causing mutations documented in the subject's medical history or identified through a retinal dystrophy gene panel, that in the opinion of the investigator would interfere with the potential therapeutic effect of the study agent or the quality of the assessments.
For subjects with herpes simplex virus (HSV):
Have complicating systemic diseases (e.g., medical conditions causing immunosuppression, active systemic infection) that would preclude the gene transfer or ocular surgery.
Have known sensitivity or allergy to systemic corticosteroids or other immunosuppressive medications.
Have used anti-coagulant agents that may alter coagulation
Have received any vaccination/immunization within 28 days prior to screening and/or during screening with the exception of the influenza vaccine, which is only exclusionary if they have received the influenza vaccine within 28 days prior to randomization.
Have used systemic corticosteroids or other immunosuppressive medications within 3 months prior to screening and/or intend to use during screening.
Have previously received any AAV gene therapy product, stem cell therapy, cell-based therapy, or similar biologics.
Ocular Exclusion Criteria (Either Eye):
Primary purpose
Allocation
Interventional model
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14 participants in 2 patient groups
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Data sourced from clinicaltrials.gov
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