Retinitis PigmentosaPhase 3liMeliGhTNCT06388200
A Phase 3 Study Of OCU400 Gene Therapy for the Treatment Of Retinitis Pigmentosa
A Phase 3 study testing Sub-Retinal Administration of OCU400-301 in people with Retinitis Pigmentosa, run by Ocugen at 17 sites in 2 countries.
Trial summary
This is a Phase 3 study to Assess the Efficacy, Safety and Tolerability of OCU400 in patients with retinitis pigmentosa (RP) associated with RHO mutations and patients with any other RP associated mutation with a clinical phenotype of RP. Read more about this study
- Who is it for?
- Ages 3 years and older
- Conditions the study is for
- Retinitis Pigmentosa
- What is being tested?
- Sub-Retinal Administration of OCU400-301
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 140 participants
- Where
- 17 sitesin 2 countries
- Study dates
- 18 Jun 2024to 12 Feb 2027
- Phase
- Phase 3
- 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 3 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Males or females ≥ 3 years of age
- Confirmed genetic diagnosis of autosomal dominant RHO mutation with clinical diagnosis of RP
- Clinical Diagnosis of Syndromic or Non-Syndromic RP with/without confirmed genetic diagnosis of any other RP associated mutation (except AD-NR2E3)
- BCVA ≤ 80 letters and ≥25 letters as measured by an ETDRS chart
- Visual field of >5° in any meridian as measured by a III4e isopter or equivalent
- Able to perform a Luminance LDNA at certain light intensity at the Screening visit
- Presence of photoreceptors as determined by SD-OCT
Who cannot join
- Subject lacks evidence of outer nuclear layer
- Previous treatment with a gene-therapy or cell therapy product or treatment with any investigational drug or ocular device within one year.
- History of any corticosteroid contraindication, corticosteroid related IOP spikes or uncontrolled glaucoma.
- Cataract surgery within 3 months. YAG capsulotomy within 1 month. Any other intraocular surgery within 6 months.
- Active ocular/intraocular infection, any history of rhegmatogenous retinal detachment or Current retinal detachment or retinal implant.
- Breast-feeding, pregnancy, sperm donation or inability to practice strict contraception
Trial locations and contacts
17 sites in 2 countries.
17 sites in 2 countries
United States12 sites
Retina Consultants of Texas
Bellaire, Texas, 77401
Retina Foundation of the Southwest
Dallas, Texas, 75231
Advanced Research, LLC.
Deerfield Beach, Florida, 33064
Erie Retina Research LLC
Erie, Pennsylvania, 16507
Baylor College of Medicine
Houston, Texas, 77030
Gundersen Health System
La Crosse, Wisconsin, 54601
University of Southern Califormia
La Jolla, California, 92093
University of Southern California, Roski Eye Insitute
Los Angeles, California, 90033
Valley Retina Institute
McAllen, Texas, 78503
Bascom Palmer Eye Institute, University of Miami, Miller School of Medicine
Miami, Florida, 33136
Vanderbilt Eye Institute
Nashville, Tennessee, 37232
Associated Retina Consultants
Phoenix, Arizona, 85020
Canada5 sites
Calgary Retina Consultants
Calgary, Alberta, T2H OC8
University of Alberta
Edmonton, Alberta, T6G 3E1
Research Institute of the McGill University Health Centre
Montreal, Quebec, H2W 1L4
Sunnybrook Health Sciences Centre
Toronto, Ontario, M4N 3M5
The University of British Columbia
Vancouver, British Columbia, V5Z 3N9
About this study
Official title: A Phase 3, Multi-Center, Randomized Study to Assess The Efficacy, Safety and Tolerability of Subretinal OCU400 Gene Therapy for the Treatment of Retinitis Pigmentosa
This is a Phase 3 study to Assess the Efficacy, Safety and Tolerability of OCU400 in patients with retinitis pigmentosa (RP) associated with RHO mutations and patients with any other RP associated mutation with a clinical phenotype of RP.
This is a multicenter, assessor blinded and randomized study which will enroll 140 subjects. Study has completed enrollment of all 140 subjects.
Read the detailed description
A total of one hundred and forty (140) RP participants will be enrolled in this study into RHO arm or Gene agnostic arm. RHO arm will only enroll participants with confirmed genetic diagnosis of mutation in RHO gene; whereas Gene Agnostic arm will enroll RP Participants based on clinical diagnosis of RP and a confirmed genetic diagnosis with a gene associated with RP.
Subjects in each arm will be randomized into treatment and control groups with a 2:1 ratio. Subjects in the treatment group will receive a sequential, bilateral sub-retinal injection of OCU400 if both eyes meet inclusion criteria. Control or untreated group subjects will receive OCU400 subretinal injection after completion of 12-month follow-up.
- Conditions
- Retinitis Pigmentosa
- Being tested
- Sub-Retinal Administration of OCU400-301
- Keywords
- Modified Gene therapy, Retinitis Pigmentosa, RHO, RP, NR2E3, Subretinal Injection
- Medical subject headings
- Retinitis Pigmentosa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Sub-Retinal Administration of OCU400-301Genetic
Sub-Retinal Administration of OCU400-301
| Study group | Role | What they receive |
|---|---|---|
| RHO Arm | Experimental | Subjects will receive a sub-retinal injection of OCU400-301 modifier gene therapy product with a concentration of 2.5 x 10^10 vg/eye Receives: Genetic: Sub-Retinal Administration of OCU400-301 |
| Gene Agnostic Arm | Experimental | Subjects will receive a sub-retinal injection of OCU400-301 modifier gene therapy product with a concentration of 2.5 x 10^10 vg/eye Receives: Genetic: Sub-Retinal Administration of OCU400-301 |
| Control for RHO Arm | No intervention | Will not receive any active study intervention |
| Control for Gene Agnostic Arm | No intervention | Will not receive any active study intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in functional vision from baseline to week 52 in pooled analysis when study eyes in treatment group were compared to study eyes in untreated control
Change in functional vision from baseline to week 52 in pooled analysis from retinitis pigmentosa subjects when study eyes in treatment group were compared to study eyes in untreated control, as measured by the ability of a study participant to navigate through a maze in Luminance Dependent Navigation Assessment (LDNA)
Measured: 52 weeks
Secondary outcomes (4)
Change in functional vision from baseline to week 52 in all the treated eyes from RP subjects (study eyes + fellow treated eyes) when compared to all the eyes (study eyes and fellow eyes) in untreated control group
Change in functional vision from baseline to week 52 in all the treated eyes from RP subjects (study eyes + fellow treated eyes) when compared to all the eyes (study eyes and fellow eyes) in untreated control group, as measured by the ability of a study participant to navigate through a maze in Luminance Dependent Navigation Assessment (LDNA).
Measured: 52 weeks
Change from Baseline in visual function in patients with retinitis pigmentosa when treatment group were compared to untreated controls
Change from Baseline in visual function in patients with retinitis pigmentosa when treatment group were compared to untreated controls in all RP subjects, as assessed by binocular low luminance visual acuity when using the Early Treatment Diabetic Retinopathy Study (ETDRS) chart letter score at Week 52.
Measured: 52 weeks
Change from baseline in LDNA Lux-level results in treated subjects when compared to the untreated controls
Change from baseline in LDNA Lux-level results in all RP subjects in treated subjects when compared to the untreated controls at weeks 12, 24, 36, and 52
Measured: 52 weeks
Number of participants with change in visual function in Gene Agnostic Arm as assessed by LLVA letter scores
Change in LLVA letter scores in Gene Agnostic Arm subjects will be compared to controls
Measured: 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Apr 2024
- Study start18 Jun 2024
- Primary completion12 Feb 2027 (estimated)
- Study completion12 Feb 2027 (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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 140 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ocugen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jennifer Henrick, RN, MS, Study director, OcugenMohamed Genead, MD, Study chair, Ocugen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06388200
- Sponsor's protocol ID
- OCU400-301
- Acronym
- liMeliGhT
- Status last verified
- Jul 2026
- First submitted
- 9 Apr 2024
- Last updated
- 31 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
- Expanded access
- Available
Study at a glance
From the registry record, last updated by the sponsor on 31 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Multi-Center, Randomized Study to Assess The Efficacy, Safety and Tolerability of Subretinal OCU400 Gene Therapy for the Treatment of Retinitis Pigmentosa
- ClinicalTrials.gov ID
- NCT06388200
- Acronym / sponsor ID
- liMeliGhT / OCU400-301
- Conditions
- Retinitis Pigmentosa
- Keywords
- Modified Gene therapy, Retinitis Pigmentosa, RHO, RP, NR2E3, Subretinal Injection
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 140 participants (actual)
- Lead sponsor
- OcugenIndustry (pharma, biotech, devices)
- Study start
- 18 Jun 2024 (actual)
- Primary completion
- 12 Feb 2027 (estimated)
- Study completion
- 12 Feb 2027 (estimated)
- First posted
- 29 Apr 2024 (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 is a Phase 3 study to Assess the Efficacy, Safety and Tolerability of OCU400 in patients with retinitis pigmentosa (RP) associated with RHO mutations and patients with any other RP associated mutation with a clinical phenotype of RP.
This is a multicenter, assessor blinded and randomized study which will enroll 140 subjects. Study has completed enrollment of all 140 subjects.
Detailed description
A total of one hundred and forty (140) RP participants will be enrolled in this study into RHO arm or Gene agnostic arm. RHO arm will only enroll participants with confirmed genetic diagnosis of mutation in RHO gene; whereas Gene Agnostic arm will enroll RP Participants based on clinical diagnosis of RP and a confirmed genetic diagnosis with a gene associated with RP.
Subjects in each arm will be randomized into treatment and control groups with a 2:1 ratio. Subjects in the treatment group will receive a sequential, bilateral sub-retinal injection of OCU400 if both eyes meet inclusion criteria. Control or untreated group subjects will receive OCU400 subretinal injection after completion of 12-month follow-up.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| RHO Arm | Experimental | Genetic: Sub-Retinal Administration of OCU400-301Subjects will receive a sub-retinal injection of OCU400-301 modifier gene therapy product with a concentration of 2.5 x 10^10 vg/eye |
| Gene Agnostic Arm | Experimental | Genetic: Sub-Retinal Administration of OCU400-301Subjects will receive a sub-retinal injection of OCU400-301 modifier gene therapy product with a concentration of 2.5 x 10^10 vg/eye |
| Control for RHO Arm | No intervention | –Will not receive any active study intervention |
| Control for Gene Agnostic Arm | No intervention | –Will not receive any active study intervention |
Who may qualify
- Ages
- 3 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Males or females ≥ 3 years of age
- Confirmed genetic diagnosis of autosomal dominant RHO mutation with clinical diagnosis of RP
- Clinical Diagnosis of Syndromic or Non-Syndromic RP with/without confirmed genetic diagnosis of any other RP associated mutation (except AD-NR2E3)
- BCVA ≤ 80 letters and ≥25 letters as measured by an ETDRS chart
- Visual field of >5° in any meridian as measured by a III4e isopter or equivalent
Key exclusion criteria (6 listed)
- Subject lacks evidence of outer nuclear layer
- Previous treatment with a gene-therapy or cell therapy product or treatment with any investigational drug or ocular device within one year.
- History of any corticosteroid contraindication, corticosteroid related IOP spikes or uncontrolled glaucoma.
- Cataract surgery within 3 months. YAG capsulotomy within 1 month. Any other intraocular surgery within 6 months.
- Active ocular/intraocular infection, any history of rhegmatogenous retinal detachment or Current retinal detachment or retinal implant.
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 |
|---|---|---|
| Sub-Retinal Administration of OCU400-301Sub-Retinal Administration of OCU400-301 | Genetic | Gene Agnostic Arm, RHO Arm |
Primary outcome
Change in functional vision from baseline to week 52 in pooled analysis when study eyes in treatment group were compared to study eyes in untreated control
Time frame: 52 weeks
Change in functional vision from baseline to week 52 in pooled analysis from retinitis pigmentosa subjects when study eyes in treatment group were compared to study eyes in untreated control, as measured by the ability of a study participant to navigate through a maze in Luminance Dependent Navigation Assessment (LDNA)
4 secondary outcomes
Change in functional vision from baseline to week 52 in all the treated eyes from RP subjects (study eyes + fellow treated eyes) when compared to all the eyes (study eyes and fellow eyes) in untreated control group
Time frame: 52 weeks
Change from Baseline in visual function in patients with retinitis pigmentosa when treatment group were compared to untreated controls
Time frame: 52 weeks
Change from baseline in LDNA Lux-level results in treated subjects when compared to the untreated controls
Time frame: 52 weeks
Number of participants with change in visual function in Gene Agnostic Arm as assessed by LLVA letter scores
Time frame: 52 weeks
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 31 Jul 2026.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
Jennifer Henrick, RN, MS study director
Ocugen
Mohamed Genead, MD study chair
Ocugen
17 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Sub-Retinal Administration of OCU400-301 in people with Retinitis Pigmentosa, run by Ocugen at 17 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 6 exclusion criteria. 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: under way, not looking for participants, as of the sponsor's last update on 31 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 sites in United States, Canada. 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 phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in functional vision from baseline to week 52 in pooled analysis when study eyes in treatment group were compared to study eyes in untreated control (time frame: 52 weeks). 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 31 Jul 2026.