Retinitis PigmentosaPhase 1/2NCT07408232
A Phase 1/2 Study in Healthy Volunteers and Participants With Autosomal Dominant Retinitis Pigmentosa (RHO-adRP)
A Phase 1/2 study testing Phase 1b/2: Cohort 1 - OCT-980 Tablet, Phase 1b/2: Cohort 2 - OCT-980 Tablet and Phase 1b/2: Cohort 3 - OCT-980 Tablet in people with Retinitis Pigmentosa, run by Octant, Inc. at 2 sites in United States.
Trial summary
This integrated Ph1/2 clinical study is to assess the safety, tolerability and pharmacokinetics (PK), pharmacodynamics (PD) and efficacy of OCT-980 in healthy volunteers and participants with RHO-adRP. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Retinitis Pigmentosa
- What is being tested?
- Phase 1b/2: Cohort 1 - OCT-980 Tablet, Phase 1b/2: Cohort 2 - OCT-980 Tablet and Phase 1b/2: Cohort 3 - OCT-980 Tablet
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 2 sitesUnited States
- Study dates
- 10 Mar 2026to 1 Nov 2028
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States2 sites, 2 recruiting
- Recruiting
Retina Foundation of the Southwest
Dallas, Texas, 75231-5080
Charlene Nwajei, cnwajei@retinafoundation.org
Jennifer Herrera, jherrera@retinafoundation.org
- Recruiting
Casey Eye Institute
Portland, Oregon, 97239
Brian Heimdahl, BA, 503-494-9771, heimdahb@ohsu.edu
Aidan Flieger, flieger@ohsu.edu
About this study
Official title: A Phase 1/2, First-in-Human Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of OCT-980 in Healthy Volunteers and Participants With Autosomal Dominant Retinitis Pigmentosa
This integrated Ph1/2 clinical study is to assess the safety, tolerability and pharmacokinetics (PK), pharmacodynamics (PD) and efficacy of OCT-980 in healthy volunteers and participants with RHO-adRP.
Read the detailed description
Octant, Inc. is evaluating OCT-980, an oral small molecule corrector for the treatment of patients with rhodopsin (RHO)-associated autosomal dominant retinitis pigmentosa (RHO-adRP). There is no known cure and no currently approved therapy for RHO-adRP, a disease of the retina characterized by progressive loss of vision.
This study is comprised of a Phase 1a in healthy volunteers, which was conducted in Australia and is now complete.
The phase 1b/2 study is an open-label, multi-center multiple ascending dose escalation (MAD) to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of multiple-ascending doses of OCT-980 for up to 48 weeks, in participants with a genetic diagnosis of Rhodopsin-associated, autosomal dominant Retinitis Pigmentosa (RP).
RHO-adRP participants are currently being recruited for the phase 1b/2 study in the US.
- Conditions
- Retinitis Pigmentosa
- Being tested
- Phase 1b/2: Cohort 1 - OCT-980 Tablet, Phase 1b/2: Cohort 2 - OCT-980 Tablet, Phase 1b/2: Cohort 3 - OCT-980 Tablet, Phase 1b/2: Cohort 4 - OCT-980 Tablet, Phase 1b/2: Cohort 5 - OCT-980 Tablet
- Keywords
- Rhodopsin-associated autosomal dominant Retinitis Pigmentosa, RHO-adRP, Retinitis Pigmentosa, Inherited Retinal Disease, autosomal dominant Retinitis Pigmentosa, Retinitis Pigmentosa 4, RP4, RP, Eye Diseases
- Medical subject headings
- Retinitis Pigmentosa, Retinitis Pigmentosa 4, Eye Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Phase 1b/2: Cohort 1 - OCT-980 TabletDrug
OCT-980 tablets administered orally once daily at dose levels specified in the protocol
Phase 1b/2: Cohort 2 - OCT-980 TabletDrug
OCT-980 tablets administered orally once daily at dose levels specified in the protocol
Phase 1b/2: Cohort 3 - OCT-980 TabletDrug
OCT-980 tablets administered orally once daily at dose levels specified in the protocol
Phase 1b/2: Cohort 4 - OCT-980 TabletDrug
OCT-980 tablets administered orally once daily at dose levels specified in the protocol
Phase 1b/2: Cohort 5 - OCT-980 TabletDrug
OCT-980 tablets administered orally once daily at dose levels specified in the protocol
| Study group | Role | What they receive |
|---|---|---|
| Phase 1b/2: OCT-980 Cohort 1 | Experimental | OCT-980 tablets administered orally once daily. Receives: Drug: Phase 1b/2: Cohort 1 - OCT-980 Tablet |
| Phase 1b/2: OCT-980 Cohort 2 | Experimental | OCT-980 tablets administered orally once daily. Receives: Drug: Phase 1b/2: Cohort 2 - OCT-980 Tablet |
| Phase 1b/2: OCT-980 Cohort 3 | Experimental | OCT-980 tablets administered orally once daily. Receives: Drug: Phase 1b/2: Cohort 3 - OCT-980 Tablet |
| Phase 1b/2: OCT-980 Cohort 4 | Experimental | OCT-980 tablets administered orally once daily. Receives: Drug: Phase 1b/2: Cohort 4 - OCT-980 Tablet |
| Phase 1b/2: OCT-980 Cohort 5 | Experimental | OCT-980 tablets administered orally once daily. Receives: Drug: Phase 1b/2: Cohort 5 - OCT-980 Tablet |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability of multiple doses (Phase 1b/2) of OCT-980 in participants with a genetic diagnosis of RHO-adRP.
Measured: Time Frame: From Baseline to Week 60 (Ph 1b/2)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Feb 2026
- Study start10 Mar 2026
- Primary completion1 Nov 2028 (estimated)
- Study completion1 Nov 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Retinitis PigmentosaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- Phase 1b/2 in RHO-adRP participants is being conducted in the US.
- Masking
- None (open label)
- Enrolment
- 18 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Octant, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07408232
- Sponsor's protocol ID
- OCT-980-101
- Status last verified
- Sep 2026
- First submitted
- 6 Feb 2026
- Last updated
- 24 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 24 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2, First-in-Human Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of OCT-980 in Healthy Volunteers and Participants With Autosomal Dominant Retinitis Pigmentosa
- ClinicalTrials.gov ID
- NCT07408232
- Acronym / sponsor ID
- OCT-980-101
- Conditions
- Retinitis Pigmentosa
- Keywords
- Rhodopsin-associated autosomal dominant Retinitis Pigmentosa, RHO-adRP, Retinitis Pigmentosa, Inherited Retinal Disease, autosomal dominant Retinitis Pigmentosa, Retinitis Pigmentosa 4, RP4, RP
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- Octant, Inc.Industry (pharma, biotech, devices)
- Study start
- 10 Mar 2026 (actual)
- Primary completion
- 1 Nov 2028 (estimated)
- Study completion
- 1 Nov 2028 (estimated)
- First posted
- 13 Feb 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This integrated Ph1/2 clinical study is to assess the safety, tolerability and pharmacokinetics (PK), pharmacodynamics (PD) and efficacy of OCT-980 in healthy volunteers and participants with RHO-adRP.
Detailed description
Octant, Inc. is evaluating OCT-980, an oral small molecule corrector for the treatment of patients with rhodopsin (RHO)-associated autosomal dominant retinitis pigmentosa (RHO-adRP). There is no known cure and no currently approved therapy for RHO-adRP, a disease of the retina characterized by progressive loss of vision.
This study is comprised of a Phase 1a in healthy volunteers, which was conducted in Australia and is now complete.
The phase 1b/2 study is an open-label, multi-center multiple ascending dose escalation (MAD) to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of multiple-ascending doses of OCT-980 for up to 48 weeks, in participants with a genetic diagnosis of Rhodopsin-associated, autosomal dominant Retinitis Pigmentosa (RP).
RHO-adRP participants are currently being recruited for the phase 1b/2 study in the US.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 5
- Model description
- Phase 1b/2 in RHO-adRP participants is being conducted in the US.
| Arm | Type | Receives |
|---|---|---|
| Phase 1b/2: OCT-980 Cohort 1 | Experimental | Drug: Phase 1b/2: Cohort 1 - OCT-980 TabletOCT-980 tablets administered orally once daily. |
| Phase 1b/2: OCT-980 Cohort 2 | Experimental | Drug: Phase 1b/2: Cohort 2 - OCT-980 TabletOCT-980 tablets administered orally once daily. |
| Phase 1b/2: OCT-980 Cohort 3 | Experimental | Drug: Phase 1b/2: Cohort 3 - OCT-980 TabletOCT-980 tablets administered orally once daily. |
| Phase 1b/2: OCT-980 Cohort 4 | Experimental | Drug: Phase 1b/2: Cohort 4 - OCT-980 TabletOCT-980 tablets administered orally once daily. |
| Phase 1b/2: OCT-980 Cohort 5 | Experimental | Drug: Phase 1b/2: Cohort 5 - OCT-980 TabletOCT-980 tablets administered orally once daily. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Phase 1b/2: Cohort 1 - OCT-980 TabletOCT-980 tablets administered orally once daily at dose levels specified in the protocol | Drug | Phase 1b/2: OCT-980 Cohort 1 |
| Phase 1b/2: Cohort 2 - OCT-980 TabletOCT-980 tablets administered orally once daily at dose levels specified in the protocol | Drug | Phase 1b/2: OCT-980 Cohort 2 |
| Phase 1b/2: Cohort 3 - OCT-980 TabletOCT-980 tablets administered orally once daily at dose levels specified in the protocol | Drug | Phase 1b/2: OCT-980 Cohort 3 |
| Phase 1b/2: Cohort 4 - OCT-980 TabletOCT-980 tablets administered orally once daily at dose levels specified in the protocol | Drug | Phase 1b/2: OCT-980 Cohort 4 |
| Phase 1b/2: Cohort 5 - OCT-980 TabletOCT-980 tablets administered orally once daily at dose levels specified in the protocol | Drug | Phase 1b/2: OCT-980 Cohort 5 |
Primary outcome
Safety and tolerability of multiple doses (Phase 1b/2) of OCT-980 in participants with a genetic diagnosis of RHO-adRP.
Time frame: Time Frame: From Baseline to Week 60 (Ph 1b/2)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Sep 2026.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Phase 1b/2: Cohort 1 - OCT-980 Tablet, Phase 1b/2: Cohort 2 - OCT-980 Tablet and Phase 1b/2: Cohort 3 - OCT-980 Tablet in people with Retinitis Pigmentosa, run by Octant, Inc. at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 24 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Octant adRP Clinical Operations (+1-510-500-5956, adrpclinicaltrials@octant.bio), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and tolerability of multiple doses (Phase 1b/2) of OCT-980 in participants with a genetic diagnosis of RHO-adRP. (time frame: Time Frame: From Baseline to Week 60 (Ph 1b/2)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Sep 2026.