Actively Recruiting
Promising ROd-cone DYstrophy Gene therapY
Led by SparingVision · Updated on 2025-09-16
33
Participants Needed
6
Research Sites
385 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This is a two-step, multicenter, Phase I/II study including an open-label dose-escalation phase (Step 1) and a three-arm, controlled, double-masked, randomized extension phase (Step 2), in subjects with advanced RCD due to a mutation in the RHO, PDE6A, or PDE6B gene.
CONDITIONS
Official Title
Promising ROd-cone DYstrophy Gene therapY
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Able to give signed informed consent and comply with study requirements
- Age 18 years or older at the time of consent
- Previously diagnosed with advanced rod-cone dystrophy due to biallelic mutations in PDE6B or PDE6A genes, or monoallelic dominant mutation in RHO gene, with documented genotyping
- Advanced stage defined by affected distance visual acuity and visual field in both eyes, with specific criteria for severe and intermediate stages
- For severe advanced RCD subjects in Step 1, difference in visual acuity between eyes must be ≤0.3 LogMAR (≤3 ETDRS lines)
- Clinical diagnosis based on history, visual field dysfunction, and fundoscopic appearance
- Diagnosis confirmed by prior full-field ERG
- Preservation of cone inner and outer segments deemed sufficient by investigator
- Negative pregnancy test for women of childbearing potential
- Agreement to use highly effective contraception from consent until 12 months after injection (except Cohort 4 after randomization)
- Affiliated to a health security system if included in a site based in France
- Clinical laboratory tests within normal or non-clinically relevant limits
- 12-lead ECG within normal limits or non-clinically relevant deviations (for treatment cohorts only)
- Physical exam without clinically relevant findings that could affect study participation
You will not qualify if you...
- Prior gene therapy or stem cell therapy for ocular or non-ocular disease
- Participation in another clinical trial with investigational drug within 5 half-lives or 90 days prior
- RCD due to mutations in genes other than RHO, PDE6A, or PDE6B
- Systemic disease or conditions affecting vision unrelated to RCD
- Narrow irido-corneal angles or contraindications to pupillary dilation
- Allergy to delivery vehicle constituents, drugs used in study, or corticosteroids
- Medical or psychiatric conditions precluding safe participation
- Use of immunosuppressive therapies other than protocol regimen
- Unwillingness to use effective contraception starting after consent for 12 months
- Pregnancy or breastfeeding
- Inability or unwillingness to comply with protocol
- Conditions preventing completion of follow-up
- Positive for HIV or other systemic immunocompromising diseases
- Significant ocular surgery within 6 months prior
- Eye disorders interfering with vision assessments
- Systemic or ocular diseases causing vision loss other than non-syndromic RP
- Prior vitrectomy or vitreomacular surgery
- Vitreomacular adhesion or traction affecting central vision
- Retinal detachment affecting central vision
- Active ocular inflammation or history of autoimmune uveitis
- Active ocular or periocular infections
- History of glaucoma or uncontrolled intraocular pressure
- Active cancer or cancer treatment
- History of ocular malignancy
- Significant cardiac disease or uncontrolled hypertension
- Pulmonary dysfunction or severe obstructive pulmonary disease
- Active tuberculosis
- Liver or renal insufficiency
- Unstable endocrine disease including diabetes or thyroid disease
- Active hepatitis B or C
- Active herpetic infections
- Immunocompromised status including transplants
- Live vaccine less than 4 weeks before injection
- COVID-19 infection less than 2 weeks before injection or recent/planned vaccination
- Incapacitated subjects as defined by national laws
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 6 locations
1
Bascom Palmer Eye Institute/University of Miami
Miami, Florida, United States, 33136
Actively Recruiting
2
Mass Eye and Ear
Boston, Massachusetts, United States, 02114
Actively Recruiting
3
Casey Eye Institute
Portland, Oregon, United States, 97239
Actively Recruiting
4
UPMC Eye Center
Pittsburgh, Pennsylvania, United States, 15213
Actively Recruiting
5
Retina Foundation of the Southwest
Dallas, Texas, United States, 75231
Actively Recruiting
6
CHNO XV-XX Paris - CIC 1423
Paris, France, 75012
Actively Recruiting
Research Team
S
SparingVision
CONTACT
M
Medical Director
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
6
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