Retinitis PigmentosaPhase 1NCT06460844
Study to Evaluate Safety of RTx-015 Injection in Retinitis Pigmentosa or Choroideremia Patients (ENVISION)
A Phase 1 study testing RTx-015 in people with Retinitis Pigmentosa and Choroideremia, run by Ray Therapeutics, Inc. at 4 sites in United States.
Trial summary
A Phase 1, open-label, non-randomized, dose-escalation study, where approximately 18 eligible patients with retinitis pigmentosa or choroideremia will be enrolled sequentially in up to 4 dose cohorts of RTx-015. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Retinitis Pigmentosa, Choroideremia
- What is being tested?
- RTx-015
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 4 sitesUnited States
- Study dates
- 27 Aug 2024to Oct 2030
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Adults ≥18 years of age
- Informed consent obtained from the patient
- Clinical diagnosis of Retinitis Pigmentosa independent of causative mutation OR Clinical diagnosis of Choroideremia with known causative mutation
- 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
Who cannot join
- 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, in the opinion of the Investigator, 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; complicating systemic diseases include those in which the disease itself, or the treatment for the disease, can alter ocular and/or Central Nervous System (CNS) function (e.g., radiation treatment of the orbit; leukemia with CNS/optic nerve involvement)
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- 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
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Retina Consultants of Texas Research Centers
Bellaire, Texas, 77401
Retina Vitreous Associates Medical Group
Beverly Hills, California, 90211
UCI Alpha Clinic
Orange, California, 92868
UPMC Vision Institute
Pittsburgh, Pennsylvania, 15221
About this study
Official title: Phase 1, Open-Label, Dose-Escalation Study to Evaluate Safety of a Single Intravitreal Injection of RTx-015 in Patients With Retinitis Pigmentosa or Choroideremia (ENVISION)
A Phase 1, open-label, non-randomized, dose-escalation study, where approximately 18 eligible patients with retinitis pigmentosa or choroideremia will be enrolled sequentially in up to 4 dose cohorts of RTx-015. Enrolled patients will receive a single, unilateral intravitreal injection of RTx-015 in the study eye at Visit 3 (Day 0) and be followed for a total of 5 years.
Read the detailed description
This Phase 1, open-label, non-randomized, multicenter clinical trial is to evaluate the safety and preliminary efficacy of a single, uniocular intravitreal injection of an investigational optogenetic gene therapy, RTx-015, in patients with retinitis pigmentosa or choroideremia. Up to 4 dose cohorts are planned, and each cohort will consist initially of 3 patients. Eligible patients will be assigned to a dose cohort by sequential enrollment.
- Conditions
- Retinitis Pigmentosa, Choroideremia
- Being tested
- RTx-015
- Medical subject headings
- Retinitis Pigmentosa, Choroideremia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RTx-015Genetic
Intravitreal injection using gene therapy to deliver an optogenetic gene to the eye. Cells in the retina use this Optogenetic gene to make a protein that responds to light.
| Study group | Role | What they receive |
|---|---|---|
| Low Dose RTx-015 | Experimental | Single intravitreal injection of low dose RTx-015 Receives: Genetic: RTx-015 |
| Middle Dose RTx-015 | Experimental | Single intravitreal injection of middle dose RTx-015 Receives: Genetic: RTx-015 |
| High Dose RTx-015 | Experimental | Single intravitreal injection of high dose RTx-015 Receives: Genetic: RTx-015 |
| Higher Dose RTx-015 | Experimental | Single intravitreal injection of higher dose RTx-015 Receives: Genetic: RTx-015 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment-Emergent Adverse Events
The number of patients in each cohort with treatment-emergent adverse events categorized using MedDRA v24.0 or higher
Measured: 12 months
Secondary outcomes (6)
Best Corrected Visual Acuity (BCVA) at Month 6 and Month 12
Change from baseline to Month 6 and Month 12 after injection with RTx-015 in BCVA
Measured: 6 and 12 Months
Low Luminance Visual Acuity (LLVA) at Month 6 and Month 12
Change from baseline to Month 6 and Month 12 after injection with RTx-015 in LLVA
Measured: 6 and 12 Months
Multi luminance mobility at Month 6 and Month 12
Change from baseline to Month 6 and Month 12 after injection with RTx-015 in Visual Change from baseline to Month 6 and 12 in mobility performance at multiple luminance levels
Measured: 6 and 12 Months
Contrast Sensitivity at Month 6 and Month 12
Change from baseline to Month 6 and Month 12 after injection with RTx-015 in Contrast Sensitivity
Measured: 6 and 12 Months
Full-field static visual field testing at Month 6 and Month 12
Change from baseline to Month 6 and Month 12 after injection with RTx-015 in the total area in which objects can be seen
Measured: 6 and 12 Months
Low Vision Quality of Life at Month 6 and Month 12
Change from baseline in low vision quality of life at Month 6 and Month 12. An algorithm will be applied to assess a total score. Higher scores are a worsening of the condition.
Measured: 6 and 12 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jun 2024
- Study start27 Aug 2024
- Primary completionOct 2030 (estimated)
- Study completionOct 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Retinitis PigmentosaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ray Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06460844
- Sponsor's protocol ID
- RTx-015-CP-101
- Status last verified
- Feb 2026
- First submitted
- 10 Jun 2024
- Last updated
- 20 Feb 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1, Open-Label, Dose-Escalation Study to Evaluate Safety of a Single Intravitreal Injection of RTx-015 in Patients With Retinitis Pigmentosa or Choroideremia (ENVISION)
- ClinicalTrials.gov ID
- NCT06460844
- Acronym / sponsor ID
- RTx-015-CP-101
- Conditions
- Retinitis Pigmentosa, Choroideremia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Ray Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 27 Aug 2024 (actual)
- Primary completion
- Oct 2030 (estimated)
- Study completion
- Oct 2030 (estimated)
- First posted
- 14 Jun 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A Phase 1, open-label, non-randomized, dose-escalation study, where approximately 18 eligible patients with retinitis pigmentosa or choroideremia will be enrolled sequentially in up to 4 dose cohorts of RTx-015. Enrolled patients will receive a single, unilateral intravitreal injection of RTx-015 in the study eye at Visit 3 (Day 0) and be followed for a total of 5 years.
Detailed description
This Phase 1, open-label, non-randomized, multicenter clinical trial is to evaluate the safety and preliminary efficacy of a single, uniocular intravitreal injection of an investigational optogenetic gene therapy, RTx-015, in patients with retinitis pigmentosa or choroideremia. Up to 4 dose cohorts are planned, and each cohort will consist initially of 3 patients. Eligible patients will be assigned to a dose cohort by sequential enrollment.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Low Dose RTx-015 | Experimental | Genetic: RTx-015Single intravitreal injection of low dose RTx-015 |
| Middle Dose RTx-015 | Experimental | Genetic: RTx-015Single intravitreal injection of middle dose RTx-015 |
| High Dose RTx-015 | Experimental | Genetic: RTx-015Single intravitreal injection of high dose RTx-015 |
| Higher Dose RTx-015 | Experimental | Genetic: RTx-015Single intravitreal injection of higher dose RTx-015 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Adults ≥18 years of age
- Informed consent obtained from the patient
- Clinical diagnosis of Retinitis Pigmentosa independent of causative mutation OR Clinical diagnosis of Choroideremia with known causative mutation
- 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
Key exclusion criteria (13 listed)
- 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, in the opinion of the Investigator, 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RTx-015Intravitreal injection using gene therapy to deliver an optogenetic gene to the eye. Cells in the retina use this Optogenetic gene to make a protein that responds to light. | Genetic | High Dose RTx-015, Higher Dose RTx-015, Low Dose RTx-015, Middle Dose RTx-015 |
Primary outcome
Incidence of Treatment-Emergent Adverse Events
Time frame: 12 months
The number of patients in each cohort with treatment-emergent adverse events categorized using MedDRA v24.0 or higher
6 secondary outcomes
Best Corrected Visual Acuity (BCVA) at Month 6 and Month 12
Time frame: 6 and 12 Months
Low Luminance Visual Acuity (LLVA) at Month 6 and Month 12
Time frame: 6 and 12 Months
Multi luminance mobility at Month 6 and Month 12
Time frame: 6 and 12 Months
Contrast Sensitivity at Month 6 and Month 12
Time frame: 6 and 12 Months
Full-field static visual field testing at Month 6 and Month 12
Time frame: 6 and 12 Months
Low Vision Quality of Life at Month 6 and Month 12
Time frame: 6 and 12 Months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Feb 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing RTx-015 in people with Retinitis Pigmentosa and Choroideremia, run by Ray Therapeutics, Inc. at 4 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 20 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Treatment-Emergent Adverse Events (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Feb 2026.