New pill could slow blindness in inherited eye disease
NCT ID NCT07408232
First seen Jun 27, 2026 · Last updated Sep 18, 2026 · Updated 2 times
Summary
This early-stage trial tests a new oral drug called OCT-980 for people with a specific inherited eye disease (autosomal dominant retinitis pigmentosa) that causes gradual vision loss and blindness. The study first gives a single dose to healthy volunteers to check safety, then multiple doses to patients. The goal is to see if the drug is safe and can slow the disease.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- OCT-980 (oral capsule or tablet)
- What this could lead to
- If successful, this could point toward a treatment that slows or stops vision loss in people with a specific genetic form of retinitis pigmentosa.
- What could go wrong
- This is a very early, first-in-human trial with only 32 participants. The drug may not be safe or effective, and results may not apply to other forms of retinitis pigmentosa.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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About 18 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Mar 2026
- Expected to finish
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Nov 2028
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Phase 1b/2 is active. Inclusion Phase 1b/2 (Multiple Ascending Dose Escalation in RHO-adRP Patients) Inclusion: * Participants must have a diagnosis of Rhodopsin-associated, autosomal dominant Retinitis Pigmentosa confirmed by a genetic test that is implemented in accordance with the relevant national requirements * Male or female greater than or equal to age 18 (inclusive) at the time of signing the informed consent form * Have a body weight \>50kg, with a body mass index (BMI) from 18.0 to 35.0 kg/m\^2 (inclusive) at screening * Best Corrected Visual Acuity (BCVA) using the Early Treatment Diabetic Retinopathy Study (ETDRS) \> 55 * Other than their diagnosis of RHO-adRP, be in good general health, as determined by the Investigator * Have sufficient venous access to allow blood sampling for pharmacokinetic analysis and safety laboratory samples Phase 1b/2 (Multiple Ascending Dose Escalation in RHO-adRP Patients) Exclusion: * Females who are pregnant or breastfeeding * Evidence of any other significant ocular (other than RHO-adRP) and non-ocular disease/ disorder * Lifetime history of ocular surgery * Any prior or current ophthalmologic gene therapy * Has a history of major surgery, severe trauma, or bone fracture within 12 weeks before the first dose of the investigational product or planned surgery within 4 weeks after the end of the study
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Retina Foundation of the Southwest
RECRUITINGDallas, Texas, 75231-5080, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a single eye injection restore sight in genetic blindness?
- A sharper look at the retina: new imaging technology may spot eye disease earlier
- Stem cell eye transplant aims to halt blindness from rare genetic disease
- Could stem cells restore sight in damaged eyes?
- Gene therapy injection aims to restore sight in rare blindness
- Ground-Level vision test could help retinitis pigmentosa patients walk better