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Tests treatment safety and results for Retinal Dystrophy Due to Biallelic ABCA4 Mutations
Official title Safety and Preliminary Efficacy of VG801 in Patients With ABCA4 Mutation-associated Retinal Dystrophy (Stargardt Disease)
ClinicalTrials.gov ID: NCT07002398
What this study is testing
What is VG801?
VG801 is an investigational medicine, being studied as a potential treatment for retinal dystrophy due to biallelic abca4 mutations.
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
- This is a single-arm, open-label, non-randomized, single dose-escalation, first-in-human (FIH) clinical trial to evaluate the safety and preliminary efficacy of VG801 for treatment of patients with retinal dystrophy (Stargardt disease) due to biallelic ABCA4 mutations.
- Phase 2: a mid-size study of how well it works
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 6 and older
You may be able to join if
- To be eligible for study entry, people must satisfy all the following criteria:
- Written informed consent.
- people aged ≥ 6 years.
- Clinical diagnosis of a macular lesion phenotypically consistent with a recessive hereditary macular dystrophy (Stargardt disease).
- Confirmed molecular diagnosis of ABCA4 mutations (homozygotes or compound heterozygotes).
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:
- Pre-existing eye conditions such as uveitis, glaucoma, or diabetic retinopathy or implantation of a medical device in the vitreous cavity or...
- Systemic diseases that would preclude the planned surgery or interfere with the interpretation of study results.
- History of intraocular surgery within the previous 6 months.
- Previous participation in a gene therapy trial.
- Participation in a clinical trial (investigational drug or medical device) within the previous 6 months.
See the full eligibility criteria
- To be eligible for study entry, people must satisfy all the following criteria:
- Written informed consent.
- people aged ≥ 6 years.
- Clinical diagnosis of a macular lesion phenotypically consistent with a recessive hereditary macular dystrophy (Stargardt disease).
- Confirmed molecular diagnosis of ABCA4 mutations (homozygotes or compound heterozygotes).
- Poor vision in the study eye.
- people will be excluded from the study if one or more of the following statements are applicable to either eye:
- Pre-existing eye conditions such as uveitis, glaucoma, or diabetic retinopathy or implantation of a medical device in the vitreous cavity or subretinal space.
- Systemic diseases that would preclude the planned surgery or interfere with the interpretation of study results.
- History of intraocular surgery within the previous 6 months.
- Previous participation in a gene therapy trial.
- Participation in a clinical trial (investigational drug or medical device) within the previous 6 months.
- Any other eye disease that may affect the outcome of the study (e.g., ocular opacities, advanced cataracts, amblyopia, etc.).
The study team makes the final eligibility decision.
Where it's taking place
- Shanghai, Shanghai Municipality, China
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, 6 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Shanghai, Shanghai Municipality, China. 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.