Retinitis PigmentosaNCT06574997
Expanded Access Program for RP in Adults
A study testing OCU400 in people with Retinitis Pigmentosa, run by Ocugen at 5 sites in United States.
Trial summary
This intermediate-size patient population expanded access program is to provide access to investigational product OCU400 for up to 75 patients with Retinitis Pigmentosa (RP) outside of the ongoing clinical trial Phase 3 program for patients who do not have access to alternative Food and Drug Administration… Read more about this study
- Who is it for?
- Ages 18 to 99 years
- Conditions the study is for
- Retinitis Pigmentosa
- What is being tested?
- OCU400
- Type of treatment
- Injection or infusion
- How many people
- Not given
- Where
- 5 sitesUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
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 to 99 years
- All sexes
- Adult, Older adult
Who may be able to join
- Males or females ≥18 years of age at the time of informed consent.
- Confirmed clinical and CLIA certified genetic diagnosis of RP (except subjects with AD-NR2E3 mutation)
- Presence of photoreceptors as determined by spectral-domain optical coherence tomography (SD-OCT).
Who cannot join
- Subject lacks evidence of outer nuclear layer, i.e., containing the nuclei of the retinal photoreceptors as determined by SD- OCT.
- Considered unsuitable for any reason that may either place the subject at increased risk during participation or interfere with the interpretation of the study outcomes by the Investigator, or the Sponsor after reviewing the subject medical history and condition.
- Previous treatment with a gene-therapy or cell therapy product, excluding OCU400.
- Previous treatment with any investigational drug or device within one year, excluding OCU400.
- Any contraindications for subretinal injection.
- Cataract surgery within 3 months. YAG capsulotomy within 1 month. Any other intraocular surgery within 6 months
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Advanced Research
Deerfield Beach, Florida, 33064
Erie Retina Research
Erie, Pennsylvania, 16507
Gundersen Health
La Crosse, Wisconsin, 54601
Valley Retina Institute
McAllen, Texas, 78503
Associated Retina Consultants
Phoenix, Arizona, 85020
About this study
Official title: An Expanded Access Program for Retinitis Pigmentosa in Adults Aged 18 Years and Older
This intermediate-size patient population expanded access program is to provide access to investigational product OCU400 for up to 75 patients with Retinitis Pigmentosa (RP) outside of the ongoing clinical trial Phase 3 program for patients who do not have access to alternative Food and Drug Administration (FDA)-approved products for treatment of the disease.
Read the detailed description
This expanded access protocol provides access to OCU400 for a subset of patients. Subjects will undergo specific ocular testing included in the study. Subjects will follow-up with physical examination, review of concomitant medication, blood work and ocular examinations for up to 52 weeks (about 12 months) post dosing.
- Conditions
- Retinitis Pigmentosa
- Being tested
- OCU400
- Keywords
- Retinitis Pigmentosa, Modifier Gene Therapy, Modified Gene Therapy, 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.
OCU400Drug
Participants will receive a subretinal injection of OCU400 modifier gene therapy product.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Aug 2024
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
- Expanded access
Sponsor and investigators
- Lead sponsor
- Ocugen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jennifer Henrick, RN, MS, Study director, Ocugen., Inc.Mohamed Genead, MD, Study chair, Ocugen., Inc
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06574997
- Sponsor's protocol ID
- OCU400-EAP
- Status last verified
- Sep 2026
- First submitted
- 23 Aug 2024
- Last updated
- 2 Oct 2026
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Expanded Access Program for Retinitis Pigmentosa in Adults Aged 18 Years and Older
- ClinicalTrials.gov ID
- NCT06574997
- Acronym / sponsor ID
- OCU400-EAP
- Conditions
- Retinitis Pigmentosa
- Keywords
- Retinitis Pigmentosa, Modifier Gene Therapy, Modified Gene Therapy, RHO, RP, NR2E3, Subretinal Injection
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- OcugenIndustry (pharma, biotech, devices)
- First posted
- 28 Aug 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 intermediate-size patient population expanded access program is to provide access to investigational product OCU400 for up to 75 patients with Retinitis Pigmentosa (RP) outside of the ongoing clinical trial Phase 3 program for patients who do not have access to alternative Food and Drug Administration (FDA)-approved products for treatment of the disease.
Detailed description
This expanded access protocol provides access to OCU400 for a subset of patients. Subjects will undergo specific ocular testing included in the study. Subjects will follow-up with physical examination, review of concomitant medication, blood work and ocular examinations for up to 52 weeks (about 12 months) post dosing.
Study design
Who may qualify
- Ages
- 18 to 99 years
- Sex
- All
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Males or females ≥18 years of age at the time of informed consent.
- Confirmed clinical and CLIA certified genetic diagnosis of RP (except subjects with AD-NR2E3 mutation)
- Presence of photoreceptors as determined by spectral-domain optical coherence tomography (SD-OCT).
Key exclusion criteria (6 listed)
- Subject lacks evidence of outer nuclear layer, i.e., containing the nuclei of the retinal photoreceptors as determined by SD- OCT.
- Considered unsuitable for any reason that may either place the subject at increased risk during participation or interfere with the interpretation of the study outcomes by the Investigator, or the Sponsor after reviewing the subject medical history and condition.
- Previous treatment with a gene-therapy or cell therapy product, excluding OCU400.
- Previous treatment with any investigational drug or device within one year, excluding OCU400.
- Any contraindications for subretinal injection.
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 |
|---|---|---|
| OCU400Participants will receive a subretinal injection of OCU400 modifier gene therapy product. | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Oct 2026.
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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., Inc.
Mohamed Genead, MD study chair
Ocugen., Inc
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing OCU400 in people with Retinitis Pigmentosa, run by Ocugen at 5 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?
The sponsor lists 3 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: status not confirmed, as of the sponsor's last update on 2 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 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.
Are results available for this study?
Results are not available in the source record.
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.
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 2 Oct 2026.