RECRUITINGPhase 1 / Phase 2INTERVENTIONAL
Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety of a Single Intravitreal Injection of RTx-021 in Patients With Stargardt Disease
Phase 1/2, Open-Label, Dose-Escalation Study to Evaluate Safety of a Single Intravitreal Injection of RTx-021 in Patients With Stargardt Disease (AURORA)
About This Trial
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.
Who May Be Eligible (Plain English)
Who May Qualify:
- 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
- your organs (liver, kidneys, etc.) are working well enough based on blood tests and general good health
Who Should NOT Join This Trial:
- 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
...See full criteria on ClinicalTrials.gov
Always talk to your doctor about whether this trial is right for you.
Original Eligibility Criteria
View original clinical language
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
Treatments Being Tested
GENETIC
RTx-021
Optogenetic gene therapy
Locations (2)
RayTx Clinical Site
Bakersfield, California, United States
RayTx Clinical Site
Bellaire, Texas, United States