Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety of a Single Intravitreal Injection of RTx-021 in Patients With Stargardt Disease
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: RTx-021.
- Who it may be relevant to
- Registry conditions: Stargardt Disease. Basic parameters: from 16 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
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)
Overview
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
- Genetic RTx-021
Optogenetic gene therapy
Primary outcome measures
- Incidence of Treatment-Emergent Adverse Events [Time frame: 6 Months]
Secondary outcome measures (6)
- Best Corrected Visual Acuity (BCVA) [Time frame: 6 Months]
- Low Luminance Visual Acuity (LLVA) [Time frame: 6 Months]
- MNREAD Reading Assessment [Time frame: 6 Months]
- Contrast Sensitivity [Time frame: 6 Months]
- Visual Field [Time frame: 6 Months]
- Low Vision Quality of Life (VA LV VFQ-48) [Time frame: 6 Months]
Eligibility criteria
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Non-randomized
- Model
- Sequential
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 3 centers
- RayTx Clinical Site — Bakersfield
- UPMC Vision Institute — Pittsburgh
- RayTx Clinical Site — Bellaire
Identifiers
NCT: NCT07439887 · RTx-021-CP-101