Summary
A Phase 1/2, open-label, non-randomized, dose-escalation study, where a minimum of 9 and a maximum of 18 eligible patients with Stargardt Disease will be enrolled sequentially in up to 3 dose cohorts of RTx-021. Enrolled patients will receive a single, unilateral intravitreal injection of RTx-021 in the study eye and be followed for 5 years.
Detailed Description
This Phase 1/2, open-label, non-randomized, sequential assignment, first-in-human dose-escalation study is to evaluate the safety and preliminary efficacy of a single, uniocular intravitreal injection of an investigational optogenetic gene therapy, RTx-021, in patients with Stargardt Disease. Up to 3 dose cohorts of RTx-021 are planned, and each cohort will consist initially of 3 patients. The study is a traditional dose escalation design with the potential for cohort expansion and is intended to support dose selection for further clinical development. Patients will receive a single, unilateral intravitreal injection of RTx-021 in the study eye and be followed for 5 years with visits being more frequent in the initial 12 months after treatment.
Eligibility Criteria
Age Range: 16 years to No maximum
Inclusion Criteria:
* Male and female patients \>= 16 years of age
* Able to comply with the study visit schedule and all protocol assessments
* Diagnosis of Stargardt Disease (genetic testing required)
* Study eye and fellow eye Best Corrected Visual Acuity meeting study criteria
* Presence of retinal ganglion cells and/or retinal nerve fiber layer on Spectral Domain Optical Coherence Tomography testing at Screening confirmed by central image reading center
* Adequate organ function and general good health
Exclusion Criteria:
* Participation in a clinical study (ocular or non-ocular) with an investigational drug, agent, or therapy in the past six months
* Concurrent participation in another interventional clinical ocular study
* Prior receipt of any gene therapy (ocular or other), retinal implant, or ocular cell therapy
* Pre-existing eye conditions in either eye that would preclude the planned treatment or are significant enough to interfere with the interpretation of study endpoints or procedural complications
* Known steroid responders if their intraocular pressure was not able to be managed effectively with topical pressure-lowering medications after prior use of steroid medications
* Complicating systemic diseases including those in which the disease itself, or the treatment of the disease, can alter ocular and/or central nervous system function (e.g. radiation treatment of the orbit; leukemia with optic nerve involvement)
* Any immunological response dysfunction including, immuno-compromising diseases or use of immunosuppressive medications, among others
* Cataract or other ocular (including refractive) surgery, intraocular and/or peri-ocular injection in either eye within the prior four months (i.e. 120 days) prior to screening
* Prior vitrectomy or aphakia in the study eye
* Known sensitivity to any component of the study treatment or contraindication to medications planned for use in the peri-procedural period (e.g. povidone-iodine to prep for intravitreal injection)
* Known contraindication to prophylactic steroid regimen
* Current pregnancy or breastfeeding
* Any other condition that would not allow the patient to complete follow-up examinations during the study