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Tests treatment safety and results for Retinitis Pigmentosa
Official title Safety and Tolerability of Intravitreal Administration of VG901 in Patients With Retinitis Pigmentosa Due to Mutations in the CNGA1 Gene
ClinicalTrials.gov ID: NCT06291935
What this study is testing
What is VG901?
VG901 is an investigational medicine, being studied as a potential treatment for retinitis pigmentosa.
Also referred to as AAV2.NN-CNGA1.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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?
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- To be eligible for study entry, people must satisfy all the following criteria:
- Able to understand and willing to consent to study participation by a written informed consent
- Male or female ≥ 18 years of age
- Clinical diagnosis of RP
- Confirmed pathogenic, biallelic variants in the CNGA1 gene
You likely can't join if
- people will be excluded from the study if one or more of the following statements are applicable to either eye:
- Additional interfering ocular conditions which would impact study results (e.g., ocular opacity and advanced cataract, uveitis, amblyopia)
- History or presence of glaucoma
- Ocular surgery, intravitreal or subretinal implantation of a medical device (within 6 months of screening)
- Mutations known to cause inherited retinal disease other than biallelic variants in the CNGA1 gene
- History of ocular infection with herpes simplex virus
See the full eligibility criteria
- To be eligible for study entry, people must satisfy all the following criteria:
- Able to understand and willing to consent to study participation by a written informed consent
- Male or female ≥ 18 years of age
- Clinical diagnosis of RP
- Confirmed pathogenic, biallelic variants in the CNGA1 gene
- Ellipsoid zone (EZ) length of the fovea of ≥ 3000 μm in the study eye
- people will be excluded from the study if one or more of the following statements are applicable to either eye:
- Additional interfering ocular conditions which would impact study results (e.g., ocular opacity and advanced cataract, uveitis, amblyopia)
- History or presence of glaucoma
- Ocular surgery, intravitreal or subretinal implantation of a medical device (within 6 months of screening)
- Mutations known to cause inherited retinal disease other than biallelic variants in the CNGA1 gene
- History of ocular infection with herpes simplex virus
- History of ocular malignancies
- History of disorders of the internal retina (e.g., retinal detachment)
- Patients with uncontrolled diabetes (HbA1c \> 7%)
- Any other retinopathy due to other diseases - including, but not limited to arterial hypertension, previous vascular retinal occlusion, trauma or acquired inflammatory diseases, contraindication to pharmacological...
- Absence of visual function on the contralateral eye
- Any damage to the optic nerve
- Individuals performing any other therapy for RP within 3 months before the study, such as - but not limited to - transcorneal electrostimulation
- Systemic conditions (e.g., autoimmune disorders) which may affect study participation or outcome measures
- History of immunodeficiency or other medical conditions which may increase the risk of VG901 administration
- Systemic illness (e.g., hepatitis or human immunodeficiency virus [HIV] infection) or medically relevant abnormal laboratory values (3 x upper limit of normal [ULN]) in blood analysis including renal and hepatic function
- Current, or recent, participation in other study/ or administration of investigational biologic agent within 3 months of Screening; Use of any investigational agent, or systemic corticosteroids, or other...
- History of allergy or sensitivity to any compound used in the study
- Contraindications to systemic immunosuppression
- people with increased risk of bleeding (i.e., use of anticoagulants or anti-platelet agents within 7 days before VG901 administration and people with international normalized ratio \> 2 or Quick \ 50 seconds...
- Subject/partner of childbearing potential unwilling to use adequate contraception for the period between Screening and 30 days after treatment, defined as the period from Screening until 30 days after treatment (defined...
- For females of childbearing potential, a positive pregnancy test at Screening or Baseline
- Females who are breastfeeding
- Previous receipt of any AAV gene therapy product
- Any condition which leads the investigator to believe that subject cannot comply with the protocol requirements or that may place the subject at an unacceptable risk from participating
The study team makes the final eligibility decision.
Where it's taking place
- Tübingen, Germany
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Tübingen, Germany. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.