A Phase 1/2 interventional study of AAV2/5-hPDE6B in Retinitis Pigmentosa, sponsored by eyeDNA Therapeutics. Terminated at 1 site in France. Open to participants aged 13 Years and older. Per ClinicalTrials.gov, last updated 2025-12-18.
Sponsored by eyeDNA Therapeutics · Phase 1/2, Interventional, and Treatment
The study is a Phase I/II, monocentric, open-label, dose-ranging safety and efficacy gene therapy intervention by subretinal administration of AAV2/5-hPDE6B.
At least twelve patients 18 years of age or older, within four consecutive cohorts of patients, will be recruited.
Then at least four patients 13 years of age or older, within a fifth cohort, will be recruited.
Retinitis pigmentosa (RP) is a disease where part of the eye (the retina) is degenerating over time. Patients initially present with night blindness, and later in life experience loss of central vision which leads to blindness. RP is a highly variable disorder with some patients developing symptomatic visual loss in childhood whereas others remain asymptomatic until mid-adulthood. There are no treatments available.
This study focuses on the form of RP caused by mutations (modifications) in the genetic information necessary to make the protein called rod cGMP phosphodiesterase 6 β subunit (or PDE6β). Clinical diagnosis is made by function tests of the eye and confirmed using a specific method called molecular testing to verify that the PDE6B gene is not correct.
This study uses a gene therapy vector inspired from an adeno-associated virus (AAV) called AAV2/5-hPDE6B. This vector intends to supply to the target cells the PDE6B therapeutic gene that is not functioning properly in the cell. The AAV parts of the gene therapy vector work as a vehicle to deliver the normal human PDE6B gene into the cells of the retina.
278 studies on the registry are indexed under Retinitis Pigmentosa; 72 are open to participants now.
This study's enrollment of 19 is below the median of 27 across 179 interventional studies indexed under Retinitis Pigmentosa.
Browse Retinitis Pigmentosa studies →This is the only study on the registry with eyeDNA Therapeutics as lead sponsor.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
Key Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biological: AAV2/5-hPDE6B Unilateral (one eye), subretinal, administration of the lowest dose. Dose-escalation will be performed after DSMC assessment.
Biological: AAV2/5-hPDE6B
Biological: AAV2/5-hPDE6B Unilateral (one eye), subretinal, administration of the medium dose. Confirmatory dose will be determined after DSMC assessment.
Biological: AAV2/5-hPDE6B
Biological: AAV2/5-hPDE6B Unilateral (one eye), subretinal, administration of the highest dose. Confirmatory dose will be determined after DSMC assessment.
Biological: AAV2/5-hPDE6B
Biological: AAV2/5-hPDE6B Unilateral (one eye), subretinal, administration of the confirmatory dose.
Biological: AAV2/5-hPDE6B
Biological: AAV2/5-hPDE6B Unilateral (one eye), subretinal, administration of the confirmatory dose.
Biological: AAV2/5-hPDE6B
Subretinal administration in one eye
Incidence of ocular and non-ocular adverse events
Time frame: 1 year + 4 years follow-up
Improvement in visual function
Improvement in visual function as assessed by mobility test
Time frame: 1 year + 4 years follow-up
Improvement in visual fields
Improvement in visual fields as assessed by visual fields measurements
Time frame: 1 year + 4 years follow-up
Improvement in visual function
Improvement in visual function as assessed by reading speed
Time frame: 1 year + 4 years follow-up
Improvement in Quality of Life
Quality of life will be measured by Quality of Life questionnaire National Eye Institute Visual Function Questionnaire (NEI VFQ-25)
Time frame: 1 year + 4 years follow-up
Plan to share: No
Ran from Nov 2017 to primary completion in Jun 2025, 91 months.
How these figures are worked out, and which studies are left out of them.
This study is terminated, as verified in Dec 2025. You cannot join it, but the record below documents what was studied.
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