Retinitis Pigmentosa (RP)Phase 1/2NYRVANANCT07807111
Open-Label Study to Evaluate the Safety, Tolerability, and Efficacy of Gene Therapy (SPVN20) in Subjects With Rod-Cone Dystrophy
A Phase 1/2 study testing SPVN20 (high dose), SPVN20 (medium dose) and SPVN20 (low dose) in people with Retinitis Pigmentosa (RP) and Rod Cone Dystrophy, run by SparingVision at 3 sites in 3 countries.
Trial summary
This open-label, multi-center study is to evaluate the safety, tolerability, and efficacy of escalating doses of a gene therapy called SPVN20 administered via a single intravitreal injection in participants with advanced Rod-Cone Dystrophy. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Retinitis Pigmentosa (RP), Rod Cone Dystrophy
- What is being tested?
- SPVN20 (high dose), SPVN20 (medium dose) and SPVN20 (low dose)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 27 participants
- Where
- 3 sitesin 3 countries
- Study dates
- 8 Oct 2025to Mar 2032
- 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
Who may be able to join
- Age ≥ 18 years old at enrollment.
- Participant with a genetically confirmed clinical diagnosis of advanced RCD in both eyes due to non-syndromic RCD.
- Participants enrolled in the Dose-Escalation cohorts must have a BCVA meeting the study eye criteria.
- Documented preservation of foveal cone cell bodies in the study eye (as shown on SD-OCT imaging).
- Participant with a history of formed vision.
- Participant willing and able to provide informed consent.
Who cannot join
- Participant participating in another clinical trial and receiving an investigational medicinal product (IMP) within either 5 half-lives of that IMP, or 90 days prior to the injection of SPVN20.
- Participant with cortical visual impairment.
- Participant with systemic disease or other pathology not related to their diagnosis of RCD, and whose symptoms or associated treatments may affect vision.
- Participant with known allergies to corticosteroids, or who will be unable to tolerate the corticosteroid regimen.
- Participant who has received immunosuppressive therapies or any other therapy known to impact the immune system during the last month prior to SPVN20 administration.
- Active ocular inflammation or recurrent history of idiopathic or autoimmune-associated uveitis.
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- Participant known to be allergic to any of the delivery vehicle constituents or to any other drugs planned to be used during the clinical study.
- Active alcohol or substance abuse.
- Participant positive for human immunodeficiency virus (HIV) or any other systemic immunocompromising disease.
- Participant with active Hepatitis B or Hepatitis C.
- Female participant currently pregnant or breastfeeding or intending to become pregnant.
- Participant with clinically active ocular infection of herpetic diseases.
- Participant with active coronavirus disease (COVID-19) infection.
- Participant who received any vaccination/immunization within 28 days prior to screening and/or during screening.
- Participant who previously received any gene therapy product, stem cell therapy, cell-based therapy for ocular or non-ocular disease.
- Participant with a retinal implant at enrollment.
- Participant with pre-existing eye conditions that would interfere with the interpretation of study endpoints, or increase the risk of surgical complications.
- Participant with significant media opacity impacting evaluation of the retina or vitreous.
- Participant who had intraocular surgery within 90 days of study treatment administration.
- Participant with history of corticosteroid-induced raised IOP of >25 mmHg following corticosteroid exposure, despite topical IOP-lowering pharmacologic therapy.
- Participant with history of unresolved rhegmatogenous retinal detachment in the fovea.
- Participant with history of unresolved ocular hypotony.
- Participant unwilling or unable (based on the Investigator's judgment) to comply with the study protocol.
- Participant with any condition that would not allow them to complete follow-up examinations during the study and, in the opinion of the Investigator, would make them unsuitable for the study.
- Participant currently taking restricted classes of medications listed in protocol
- Participant with ongoing cardiac or neurological disorders not managed by a restricted medication can be excluded based on the Investigator's judgement.
- Women of childbearing potential (WOCBP) and men and/or their partner(s) of childbearing potential who do not agree to use a highly effective contraceptive method.
Trial locations and contacts
3 sites in 3 countries. Contact the study team or a site near you to ask about taking part.
3 sites in 3 countries
Belgium1 site, 1 recruiting
- Recruiting
Ghent University Hospital
Ghent
Ophthalmic Clinical Trials Unit, 00 32 9 332 23, OCTU@uzgent.be
Prof. Dr. Bart Leroy
France1 site, 1 recruiting
- Recruiting
CHNO XV-XX Paris - CIC 1423
Paris, 75012
Dorothée Dagostinoz, 00 33 1 40 02 15 20, ddagostinoz@15-20.fr
Dr Amine Benadji
Ireland1 site, 1 recruiting
- Recruiting
Royal Victoria Eye And Ear Hospital
Dublin
Dr Emma Duignan, 00 353 1 664 4600, Emma.duignan@rveeh.ie
Dr Emma Duignan
About this study
Official title: A Phase I/IIa Clinical Trial to Assess the Safety, Tolerability, and Efficacy of a Single Intravitreal Injection of SPVN20 Gene Therapy in Participants With Advanced Rod-Cone Dystrophy
This open-label, multi-center study is to evaluate the safety, tolerability, and efficacy of escalating doses of a gene therapy called SPVN20 administered via a single intravitreal injection in participants with advanced Rod-Cone Dystrophy.
Read the detailed description
This is an open-label, non-randomized, multi-center, dose-escalation and dose-expansion, first-in-human, Phase I/IIa study. Participants will be followed for a total of five years after receiving a single unilateral intravitreal injection of SPVN20 in their study eye, and will be monitored by an independent Data Safety Monitoring Committee. Eligible patients will be assigned by sequential enrollment to one of the following three cohorts:
Cohort 1: low dose of SPVN20 will be evaluated in a total of 9 participants. Cohort 2: medium dose of SPVN20 will be evaluated in a total of 9 participants. Cohort 3: high dose of SPVN20 will be evaluated in a total of 9 participants.
- Conditions
- Retinitis Pigmentosa (RP), Rod Cone Dystrophy
- Being tested
- SPVN20 (high dose), SPVN20 (medium dose), SPVN20 (low dose)
- Keywords
- Retinal Disease, Eye Disease, Retinal Degeneration, Retinal Dystrophies, Rod-Cone Dystrophy, Gene Therapy, Retinitis Pigmentosa
- Medical subject headings
- Retinitis Pigmentosa, Cone-Rod Dystrophies, Retinal Diseases, Eye Diseases, Retinal Degeneration, Retinal Dystrophies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SPVN20 (high dose)Biological
SPVN20
SPVN20 (medium dose)Biological
SPVN20
SPVN20 (low dose)Biological
SPVN20
| Study group | Role | What they receive |
|---|---|---|
| SPVN20 (low dose). Single intravitreal injection of a low dose of SPVN20 on Day 0 | Experimental | SPVN20 Receives: Biological: SPVN20 (low dose) |
| SPVN20 (medium dose). Single intravitreal injection of a medium dose of SPVN20 on Day 0 | Experimental | SPVN20 Receives: Biological: SPVN20 (medium dose) |
| SPVN20 (high dose). Single intravitreal injection of a high dose of SPVN20 on Day 0 | Experimental | SPVN20 Receives: Biological: SPVN20 (high dose) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and Tolerability
Incidence and severity of ocular and non-ocular treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
Measured: 6 months
Ocular safety
General ocular assessments including complete ophthalmic examination of extraocular and intraocular structures
Measured: 6 months
Secondary outcomes (2)
Best Corrected Visual Acuity (BCVA)
Change in BCVA from Baseline to Month 6 after dosing with SPVN20.
Measured: 6 months
Full-field stimulus threshold (FST) test
Change in FST from Baseline to Month 6 after dosing with SPVN20
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Sep 2026
- Study start8 Oct 2025
- Primary completionMar 2032 (estimated)
- Study completionMar 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Retinitis Pigmentosa (RP)Studies 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 I/IIa open-label dose-escalation study
- Masking
- None (open label)
- Enrolment
- 27 participants (estimated)
Sponsor and investigators
- Lead sponsor
- SparingVision
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07807111
- Sponsor's protocol ID
- SPVN20-CLIN-01
- Other IDs
- 2025-520665-47-00, 2025
- Acronym
- NYRVANA
- Status last verified
- Sep 2026
- First submitted
- 10 Aug 2026
- Last updated
- 8 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 8 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/IIa Clinical Trial to Assess the Safety, Tolerability, and Efficacy of a Single Intravitreal Injection of SPVN20 Gene Therapy in Participants With Advanced Rod-Cone Dystrophy
- ClinicalTrials.gov ID
- NCT07807111
- Acronym / sponsor ID
- NYRVANA / SPVN20-CLIN-01
- Conditions
- Retinitis Pigmentosa (RP), Rod Cone Dystrophy
- Keywords
- Retinal Disease, Eye Disease, Retinal Degeneration, Retinal Dystrophies, Rod-Cone Dystrophy, Gene Therapy, Retinitis Pigmentosa
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 27 participants (estimated)
- Lead sponsor
- SparingVisionIndustry (pharma, biotech, devices)
- Study start
- 8 Oct 2025 (actual)
- Primary completion
- Mar 2032 (estimated)
- Study completion
- Mar 2032 (estimated)
- First posted
- 8 Sep 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 open-label, multi-center study is to evaluate the safety, tolerability, and efficacy of escalating doses of a gene therapy called SPVN20 administered via a single intravitreal injection in participants with advanced Rod-Cone Dystrophy.
Detailed description
This is an open-label, non-randomized, multi-center, dose-escalation and dose-expansion, first-in-human, Phase I/IIa study. Participants will be followed for a total of five years after receiving a single unilateral intravitreal injection of SPVN20 in their study eye, and will be monitored by an independent Data Safety Monitoring Committee. Eligible patients will be assigned by sequential enrollment to one of the following three cohorts:
Cohort 1: low dose of SPVN20 will be evaluated in a total of 9 participants. Cohort 2: medium dose of SPVN20 will be evaluated in a total of 9 participants. Cohort 3: high dose of SPVN20 will be evaluated in a total of 9 participants.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
- Model description
- Phase I/IIa open-label dose-escalation study
| Arm | Type | Receives |
|---|---|---|
| SPVN20 (low dose). Single intravitreal injection of a low dose of SPVN20 on Day 0 | Experimental | Biological: SPVN20 (low dose)SPVN20 |
| SPVN20 (medium dose). Single intravitreal injection of a medium dose of SPVN20 on Day 0 | Experimental | Biological: SPVN20 (medium dose)SPVN20 |
| SPVN20 (high dose). Single intravitreal injection of a high dose of SPVN20 on Day 0 | Experimental | Biological: SPVN20 (high dose)SPVN20 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Age ≥ 18 years old at enrollment.
- Participant with a genetically confirmed clinical diagnosis of advanced RCD in both eyes due to non-syndromic RCD.
- Participants enrolled in the Dose-Escalation cohorts must have a BCVA meeting the study eye criteria.
- Documented preservation of foveal cone cell bodies in the study eye (as shown on SD-OCT imaging).
- Participant with a history of formed vision.
Key exclusion criteria (27 listed)
- Participant participating in another clinical trial and receiving an investigational medicinal product (IMP) within either 5 half-lives of that IMP, or 90 days prior to the injection of SPVN20.
- Participant with cortical visual impairment.
- Participant with systemic disease or other pathology not related to their diagnosis of RCD, and whose symptoms or associated treatments may affect vision.
- Participant with known allergies to corticosteroids, or who will be unable to tolerate the corticosteroid regimen.
- Participant who has received immunosuppressive therapies or any other therapy known to impact the immune system during the last month prior to SPVN20 administration.
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 |
|---|---|---|
| SPVN20 (high dose)SPVN20 | Biological | SPVN20 (high dose). Single intravitreal injection of a high dose of SPVN20 on Day 0 |
| SPVN20 (medium dose)SPVN20 | Biological | SPVN20 (medium dose). Single intravitreal injection of a medium dose of SPVN20 on Day 0 |
| SPVN20 (low dose)SPVN20 | Biological | SPVN20 (low dose). Single intravitreal injection of a low dose of SPVN20 on Day 0 |
Primary outcome
Safety and Tolerability
Time frame: 6 months
Incidence and severity of ocular and non-ocular treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
Ocular safety
Time frame: 6 months
General ocular assessments including complete ophthalmic examination of extraocular and intraocular structures
2 secondary outcomes
Best Corrected Visual Acuity (BCVA)
Time frame: 6 months
Full-field stimulus threshold (FST) test
Time frame: 6 months
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 8 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.
3 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing SPVN20 (high dose), SPVN20 (medium dose) and SPVN20 (low dose) in people with Retinitis Pigmentosa (RP) and Rod Cone Dystrophy, run by SparingVision at 3 sites in 3 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 6 inclusion and 27 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: looking for participants now, as of the sponsor's last update on 8 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 3 sites in Belgium, France, Ireland. 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 Medical Director (+33143462060, info@sparingvision.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and Tolerability (time frame: 6 months). The study lists 2 primary outcomes in all. 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 8 Sep 2026.