OCT-980 for Retinitis Pigmentosa

Recruiting at 2 trial locations

Overseen ByOctant adRP Clinical Operations

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Octant, Inc.

Disqualifiers: Pregnancy, Breastfeeding, Ocular surgery, others

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?

This trial tests a new treatment called OCT-980 for individuals with Rhodopsin-associated, autosomal dominant Retinitis Pigmentosa (RHO-adRP). The main goal is to assess the treatment's safety and its effects on the body. Participants will take OCT-980 tablets once daily. Those with this genetic eye condition who are generally healthy may be suitable candidates. As a Phase 1 trial, participants will be among the first to receive this treatment, aiding researchers in understanding its effects in humans.

Is there any evidence suggesting that OCT-980 is likely to be safe for humans?

Studies have shown that OCT-980 aims to help with retinitis pigmentosa by stabilizing certain proteins in the eye, which should help the eye function properly. Research indicates that safety remains a key focus for this treatment. Although specific information on side effects isn't available yet, the treatment is in early trials, where it is still being tested for safety in humans.

Early trials like this one generally assess how well a treatment is tolerated and what side effects might occur. If OCT-980 were unsafe, it likely wouldn't have reached this stage. However, it's important to remember that all medicines can have side effects. Anyone considering joining a trial should consult their doctor to understand the risks and benefits.¹ ² ³ ⁴ ⁵

Why do researchers think this study treatment might be promising for retinitis pigmentosa?

OCT-980 is unique because it offers a new approach to treating Retinitis Pigmentosa, a condition that traditionally relies on vitamin A supplements, retinal implants, or gene therapy. Unlike these existing options, OCT-980 is a tablet taken orally once daily, which could simplify treatment regimens and improve patient adherence. Researchers are excited because it may introduce a novel mechanism of action, potentially targeting pathways not addressed by current therapies, which could lead to improved outcomes for patients with this challenging vision disorder.

What evidence suggests that OCT-980 might be an effective treatment for Retinitis Pigmentosa?

Research on OCT-980 for retinitis pigmentosa remains in the early stages, so clinical data is limited. However, studies on similar treatments have shown promising results. For instance, one study on gene therapy in blind mice demonstrated that about 10% of the eye's light-sensitive cells were preserved, improving their ability to detect light and enhancing overall vision. This suggests that altering certain genes might help restore some vision. While OCT-980 is not identical, it aims to protect or restore eye cells in a similar manner. More research is needed to fully understand its potential effectiveness in humans. Participants in this trial will take OCT-980 tablets orally once daily in different cohorts to evaluate its potential benefits.⁶ ⁷ ⁸ ⁹ ¹⁰

Are You a Good Fit for This Trial?

This trial is for healthy adults aged 18-65 and people with a specific genetic form of retinitis pigmentosa (RHO-adRP), confirmed by genetic testing. Participants must be in good general health, not pregnant or breastfeeding, and able to provide blood samples.

Inclusion Criteria

I have veins suitable for regular blood draws.

I am between 18 and 65 years old and in good general health.

I am able and willing to eat a full high-fat meal as required by the study.

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Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Phase 1a

Completed study in healthy volunteers to assess safety and tolerability

Completed

Phase 1b/2

Open-label, multi-center multiple ascending dose escalation to evaluate safety, tolerability, PK, PD, and efficacy of OCT-980 for up to 48 weeks

48 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4-8 weeks

What Are the Treatments Tested in This Trial?

Interventions

OCT-980

Trial Overview

The study is testing different doses of an oral drug called OCT-980 tablet in both healthy volunteers and people with RHO-adRP to see how safe it is, how the body processes it, and if it helps vision.