RTx-015 for Retinitis Pigmentosa

Recruiting at 3 trial locations

Overseen By David Boyer, MD

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Ray Therapeutics, Inc.

Must not be taking: Immunosuppressives, Steroids

Disqualifiers: Gene therapy, Retinal implant, Others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 13

Duration: 1 day

18 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to explore the safety and effectiveness of different doses of RTx-015 for treating retinitis pigmentosa and choroideremia, eye conditions that can lead to vision loss. Participants will receive a single eye injection of RTx-015 and be monitored over five years. It suits those diagnosed with retinitis pigmentosa or choroideremia who are in good general health and have not participated in another clinical study in the past six months.

As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the chance to be among the first to receive this new treatment.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, if you are using immunosuppressive medications or have complicating systemic diseases, you may not be eligible to participate.

Is there any evidence suggesting that RTx-015 is likely to be safe for humans?

Research has shown that RTx-015 is being tested for safety in individuals with retinitis pigmentosa and choroideremia. In similar studies, patients received a single injection directly into the eye. As these studies are in the early stages, researchers are closely monitoring the treatment's safety.

As a Phase 1 trial, this marks the first time RTx-015 is tested in humans. This phase focuses on assessing how well participants tolerate the treatment and identifying any side effects. Early trials like this often have limited safety information, but they are crucial to ensure treatments are safe before advancing to the next stages.

Participants in similar studies have generally tolerated these injections, though some have experienced mild side effects like eye discomfort. These side effects are usually manageable and expected in such trials. With RTx-015 being a new treatment, safety remains the top priority in these early tests. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about RTx-015 for retinitis pigmentosa because it offers a promising new approach compared to existing treatments like vitamin A supplements, gene therapy, or retinal implants. Unlike these options, RTx-015 is administered as a single intravitreal injection, which means it's delivered directly into the eye, potentially enhancing its effectiveness and reducing the need for frequent treatments. This treatment explores different dosages—high, middle, low, and higher—allowing researchers to fine-tune its impact on slowing the progression of vision loss. By targeting the root causes of retinitis pigmentosa more precisely, RTx-015 could offer a more convenient and potentially more effective solution for patients.

What evidence suggests that this trial's treatments could be effective for retinitis pigmentosa?

Research has shown that gene therapy, such as RTx-015, might improve vision in people with retinitis pigmentosa. In similar treatments, some patients have experienced better vision. This suggests that RTx-015 could potentially slow vision loss or even enhance sight for some individuals with this condition. Although more studies are needed, early results are promising and offer hope for those affected by retinitis pigmentosa. ^{1 2 4 6 7}

Are You a Good Fit for This Trial?

This trial is for about 9 people with retinitis pigmentosa, a degenerative eye condition. Participants will receive one injection in the affected eye and be monitored for a year. Details on specific inclusion or exclusion criteria are not provided.

Inclusion Criteria

Informed consent obtained from the patient

Study eye and fellow eye Best Corrected Visual Acuity meeting study criteria

My organs are functioning well and I am in 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

I do not have any serious eye conditions that could affect my treatment.

Concurrent participation in another interventional clinical ocular study

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single, unilateral intravitreal injection of RTx-015 in the study eye

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 months

Multiple visits over 12 months

What Are the Treatments Tested in This Trial?

Interventions

• RTx-015

Trial Overview The study tests RTx-015, given as a single intravitreal (into the eye) injection to see if it's safe at different doses. It's an early-phase trial without randomization, focusing on dose safety over 12 months.

How Is the Trial Designed?

Group I: Middle Dose RTx-015 Experimental Treatment 1 Intervention

Single intravitreal injection of middle dose RTx-015

Group II: Low Dose RTx-015 Experimental Treatment 1 Intervention

Single intravitreal injection of low dose RTx-015

Group III: Higher Dose RTx-015 Experimental Treatment 1 Intervention

Single intravitreal injection of higher dose RTx-015

Group IV: High Dose RTx-015 Experimental Treatment 1 Intervention

Single intravitreal injection of high dose RTx-015

Who Is Running the Clinical Trial?

Ray Therapeutics, Inc.

Lead Sponsor

Trials

2

Recruited

140+

Published Research Related to This Trial

In a study of 9 patients with a specific Rhodopsin variant associated with sector retinitis pigmentosa, a wide range of disease phenotypes was observed, from sector-like to pericentral RP, indicating significant variability in how the disease manifests among individuals.

The longest follow-up of a patient with this condition showed that while central visual acuity remains good over time, there can be significant progression affecting peripheral vision, highlighting the need for regular monitoring through imaging and electroretinography.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Beyond Sector Retinitis Pigmentosa: Expanding the Phenotype and Natural History of the Rhodopsin Gene Codon 106 Mutation (Gly-to-Arg) in Autosomal Dominant Retinitis Pigmentosa. Ballios, BG., Place, EM., Martinez-Velazquez, L., et al.[2022]

The study followed 46 individuals with PRPF31-related retinitis pigmentosa (RP11) and found that the median age of disease onset was 10 years, with a median follow-up of 8 years, highlighting the early onset and long-term progression of the disease.

Visual field areas deteriorated significantly faster than visual acuity, with legal blindness typically occurring at a median age of 57 years, emphasizing the need for careful monitoring of visual field changes in patients with this condition.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Disease progression of retinitis pigmentosa caused by PRPF31 variants in a Nordic population: a retrospective study with up to 36 years follow-up. Lisbjerg, K., Bertelsen, M., Lyng Forman, J., et al.[2023]

In a study of 106 patients with retinitis pigmentosa (RP) over 1 to 5 years, various imaging techniques (SD-OCT, FAF, and MP) were found to reliably measure disease progression, showing strong correlations between structural and functional outcomes.

The annual rates of decline in visual acuity and other parameters were significant, with visual acuity decreasing by an average of 2.3 letters per year, indicating a measurable progression of RP that can be tracked using these imaging methods.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Progression of retinitis pigmentosa on multimodal imaging: The PREP-1 study. Iftikhar, M., Usmani, B., Sanyal, A., et al.[2020]

The first-in-human phase 1/2 clinical trial of retinal gene therapy for X-linked retinitis pigmentosa (RP) involving 18 patients showed that the treatment was generally safe, with only mild steroid-responsive inflammation noted at higher doses.

Significant visual field improvements were observed in six patients starting from one month after treatment and lasting through the 6-month follow-up, indicating potential efficacy of the gene therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Initial results from a first-in-human gene therapy trial on X-linked retinitis pigmentosa caused by mutations in RPGR. Cehajic-Kapetanovic, J., Xue, K., Martinez-Fernandez de la Camara, C., et al.[2023]

A new classification system for retinitis pigmentosa (RP) was developed, allowing for easy categorization of disease severity based on visual acuity, visual field width, and ellipsoid zone width, using a scoring system from 0 to 15.

In a study of 93 patients, the classification system showed strong correlations between scores and disease severity grades, which were also linked to age and disease duration, making it a useful tool for clinicians to assess and compare patient conditions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Classification of disease severity in retinitis pigmentosa. Iftikhar, M., Lemus, M., Usmani, B., et al.[2020]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06460844

Study to Evaluate Safety of RTx-015 Injection in Retinitis ... This Phase 1, open-label, non-randomized, multicenter clinical trial is to evaluate the safety and preliminary efficacy of a single, uniocular intravitreal ...

2. scge.mcw.edu scge.mcw.edu/platform/data/clinicalTrials/report/NCT06460844

Study to Evaluate Safety of RTx-015 Injection in Retinitis ... A Phase 1, open-label, non-randomized, dose-escalation study, where approximately 15 eligible patients with retinitis pigmentosa or ...

3. withpower.com withpower.com/trial/rtx-015-for-retinitis-pigmentosa-4b540

RTx-015 for Retinitis Pigmentosa A study on a similar gene therapy for retinitis pigmentosa showed that visual field improvements were observed in some patients, suggesting that gene therapy ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT06460844?cond=Choroideremia%20OR%20%22CHM%22%20OR%20%22%20progressive%20choroidal%20atrophy%22%20OR%20%22%20Tapetochoroidal%20dystrophy%22&aggFilters=status:not%20rec&viewType=Table&rank=1

Study to Evaluate Safety of RTx-015 Injection in Retinitis ... This Phase 1, open-label, non-randomized, multicenter clinical trial is to evaluate the safety and preliminary efficacy of a single, uniocular intravitreal ...

5. clinicaltrials.ucbraid.org clinicaltrials.ucbraid.org/retinitis-pigmentosa

Retinitis Pigmentosa clinical trials at University of California ... This study evaluates the safety of a single injection of jCell (famzeretcel) comprising 8.8 million (8.8M) retinal progenitor cells over a six- ...

6. ucihealth.org ucihealth.org/clinical-trials/ascc-24-05

Evaluate Safety of a Single Intravitreal Injection of RTx-015 ... In this Phase 1, first-in-human, open-label, non-randomized, sequential assignment, dose-escalation study,. approximately 9 eligible patients with RP will ...

7. app.trialscreen.org app.trialscreen.org/trials/phase-1-to-evaluate-safety-rtx-015-injection-retinitis-pigmentosa-patients-trial-nct06460844

Study to Evaluate Safety of RTx-015 Injection in Retinitis ... A Phase 1 interventional study of RTx-015 in retinitis pigmentosa. A study to evaluate the safety of a gene therapy treatment (RTx-015) for retinitis pigmentosa.