RTx-015 for Retinitis Pigmentosa

Recruiting at 2 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

15 Participants Needed

Trial Summary

What is the purpose of this trial?

A Phase 1, open-label, non-randomized, dose-escalation study, where approximately 15 eligible patients with retinitis pigmentosa or choroideremia will be enrolled sequentially in up to 3 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 12 months.

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. ^{1 2 3 4 5}

Eligibility Criteria

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

I am 18 years old or older.

Informed consent obtained from the patient

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

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

Treatment Details

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.

Participant Groups

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: 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+

Findings from Research

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]

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 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]

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]

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]

References

1. United States 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. [2023]

2. Australia pubmed.ncbi.nlm.nih.gov

Progression of retinitis pigmentosa on multimodal imaging: The PREP-1 study. [2020]

3. England 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. [2023]

4. England pubmed.ncbi.nlm.nih.gov

Classification of disease severity in retinitis pigmentosa. [2020]

5. Switzerland 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. [2022]