18 Participants Needed

RTx-015 for Retinitis Pigmentosa

Recruiting at 3 trial locations
EM
DB
Overseen ByDavid Boyer, MD
Age: 18+
Sex: Any
Trial Phase: Phase 1
Sponsor: Ray Therapeutics, Inc.
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

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.

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.

What data supports the effectiveness of the treatment 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 can be promising for treating this condition.12345

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
My organs are functioning well and I am in good health.
Study eye and fellow eye Best Corrected Visual Acuity meeting study criteria
See 2 more

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
See 10 more

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?
4Treatment groups
Experimental Treatment
Group I: Middle Dose RTx-015Experimental Treatment1 Intervention
Group II: Low Dose RTx-015Experimental Treatment1 Intervention
Group III: Higher Dose RTx-015Experimental Treatment1 Intervention
Group IV: High Dose RTx-015Experimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

Ray Therapeutics, Inc.

Lead Sponsor

Trials
2
Recruited
140+

Published Research Related to This Trial

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

Citations

Initial results from a first-in-human gene therapy trial on X-linked retinitis pigmentosa caused by mutations in RPGR. [2023]
Progression of retinitis pigmentosa on multimodal imaging: The PREP-1 study. [2020]
Disease progression of retinitis pigmentosa caused by PRPF31 variants in a Nordic population: a retrospective study with up to 36 years follow-up. [2023]
Classification of disease severity in retinitis pigmentosa. [2020]
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. [2022]
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top
Terms of ServiceยทPrivacy PolicyยทCookiesยทSecurity