Stargardt Disease
Conditions
Keywords
AURORA, RTx-021
Brief 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.
Interventions
GENETICRTx-021
Optogenetic gene therapy
Sponsors
Ray Therapeutics, Inc.
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
16 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of Treatment-Emergent Adverse Events | 6 Months | The number of patients in each cohort with treatment-emergent adverse events categorized using MedDRA v24.0 or higher |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Best Corrected Visual Acuity (BCVA) | 6 Months | Change from Baseline to Month 6 after injection with RTx-021 in BCVA |
| Low Luminance Visual Acuity (LLVA) | 6 Months | Change from Baseline to Month 6 after injection with RTx-021 in LLVA |
| MNREAD Reading Assessment | 6 Months | Change from Baseline to Month 6 after injection with RTx-021 in reading acuity and reading speed. |
| Contrast Sensitivity | 6 Months | Change from Baseline to Month 6 after injection with RTx-021 in contrast sensitivity |
| Visual Field | 6 Months | Change from Baseline to Month 6 after injection with RTx-021 in the total area in which objects can be seen |
| Low Vision Quality of Life (VA LV VFQ-48) | 6 Months | Change from Baseline to Month 6 after injection with RTx-021 in low vision quality of life questionnaire VA LV VFQ-48. A total score will be calculated with higher scores indicating an improvement. |
Countries
United States
Contacts
CONTACTExecutive Clinical Director
Outcome results
None listed