Actively Recruiting
Safety and Tolerability of Intravitreal Administration of VG901 in Patients With Retinitis Pigmentosa Due to Mutations in the CNGA1 Gene
Led by VeonGen Therapeutics GmbH · Updated on 2025-03-28
6
Participants Needed
1
Research Sites
134 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The goal of this phase 1 clinical trial is to learn about the safety and efficacy of a gene therapy, VG901, in patients with a rare disorder of the eye called Retinitis Pigmentosa. The main questions the study aims to answer are: * What is the best tolerated dose and are there any side effects, in particular any inflammatory reactions post drug administration? * Are there any early signs of efficacy on visual function? Participants will be administered a single intravitreal dose of VG901 into the most affected eye through a syringe and followed up for a year to monitor safety and efficacy. There will be two cohorts of participants in this study. Study Cohort 1 will receive the low dose and Study Cohort 2 will receive the high dose as specified in the Protocol.
CONDITIONS
Official Title
Safety and Tolerability of Intravitreal Administration of VG901 in Patients With Retinitis Pigmentosa Due to Mutations in the CNGA1 Gene
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Able to understand and willing to provide written informed consent
- Male or female 18 years of age or older
- Clinical diagnosis of Retinitis Pigmentosa
- Confirmed pathogenic, biallelic variants in the CNGA1 gene
- Ellipsoid zone length of the fovea of at least 3000 micrometers in the study eye
You will not qualify if you...
- Presence of other eye conditions that could affect study results, such as cataracts, uveitis, or amblyopia
- History or presence of glaucoma
- Eye surgery or implantation of a medical device in the eye within 6 months before screening
- Mutations causing inherited retinal diseases other than CNGA1 gene variants
- History of eye infections with herpes simplex virus
- History of eye cancers
- History of internal retina disorders like retinal detachment
- Uncontrolled diabetes with HbA1c greater than 7%
- Other retinopathies including those from hypertension, vascular occlusion, trauma, or inflammation
- Contraindications to pupil dilation medication
- No visual function in the opposite eye
- Damage to the optic nerve
- Use of other therapies for Retinitis Pigmentosa within 3 months before the study
- Systemic conditions like autoimmune disorders affecting participation or outcomes
- History of immunodeficiency or conditions increasing risk with VG901
- Systemic illnesses such as hepatitis or HIV or abnormal lab tests including kidney and liver function
- Recent participation in other investigational studies or use of immunosuppressive drugs within 3 months
- Allergy or sensitivity to compounds used in the study
- Contraindications to systemic immunosuppression
- Increased bleeding risk or use of blood thinners within 7 days before VG901 administration
- Unwillingness to use contraception from screening through 30 days after treatment
- Positive pregnancy test at screening or baseline
- Currently breastfeeding
- Previous treatment with any AAV gene therapy
- Any condition that may prevent compliance or increase risk from study participation
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Center for Ophthalmology, University of Tuebingen
Tübingen, Germany, 72076
Actively Recruiting
Research Team
A
Andrea Rindtorff
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
1
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