BridgeMD
A trial in patients with ABCA4 mutation-associated retinal dystrophy.
EU CTIS ID: 2024-520425-37-00
What this study is testing
Safety Objectives: To evaluate the types, frequency, severity of ocular and systemic adverse events, and laboratory abnormalities To evaluate the tolerability of VG801 subretinal injection To determine the dose(s) below DLT that is potentially effective to conduct further efficacy clinical studies Preliminary Efficacy Objectives: To evaluate the changes of best corrected visual acuity (BCVA), measured with the Early Treatment of Diabetic Retinopathy (ETDRS) chart To evaluate the changes of ocular imaging studies To evaluate the mobility function via a novel virtual reality test
- Phase I and Phase II (Integrated)- First administration to humans
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Written informed consent.
- Subjects aged ≥ 6 years.
- Molecular diagnosis was confirmed due to ABCA4 mutations (homozygotes or compound heterozygotes) by a CLIA (Clinical Laboratory Improvement Amendments)-certified laboratory.
- Clinical diagnosis of a macular lesion phenotypically consistent with retinal disease.
- Visual acuity not better than 20/60 in the study eye.
- Detectable outer nuclear layer thickness in the macular region using OCT.
You likely can't join if
- Pre-existing eye conditions such as uveitis, glaucoma, or diabetic retinopathy.
- Positive for hepatitis C or human immunodeficiency virus (HIV) infection.
- Abnormal laboratory values (3 upper limit of normal [ULN]) in blood analysis including renal and hepatic function.
- Subjects with increased risk of bleeding (i.e., use of anticoagulants or anti-platelet agents within 7 days before VG801 administration and subjects with international normalized ratio > 2 or Quick < 50% or partial thromboplastin time > 50 seconds, thrombocytopenia, as well as any other known coagulopathy).
- Breast-feeding woman.
- Any other condition that would not allow the potential subject to complete follow-up examinations during the study and, in the opinion of the Investigator, makes the potential subject unsuitable for the study.
See the full eligibility criteria
- Written informed consent.
- Subjects aged ≥ 6 years.
- Molecular diagnosis was confirmed due to ABCA4 mutations (homozygotes or compound heterozygotes) by a CLIA (Clinical Laboratory Improvement Amendments)-certified laboratory.
- Clinical diagnosis of a macular lesion phenotypically consistent with retinal disease.
- Visual acuity not better than 20/60 in the study eye.
- Detectable outer nuclear layer thickness in the macular region using OCT.
- Study eye must have clear ocular media and adequate pupillary dilation, including no allergy to dilating eye drops and with sufficient fixation to permit good quality retinal imaging.
- Negative pregnancy test.
- Willing to avoid excessive exposure to sun light (e.g., by using a hat, ultraviolet absorbing sunglasses, and sunscreen with a minimum SPF of 30).
- Pre-existing eye conditions such as uveitis, glaucoma, or diabetic retinopathy.
- Positive for hepatitis C or human immunodeficiency virus (HIV) infection.
- Abnormal laboratory values (3 upper limit of normal [ULN]) in blood analysis including renal and hepatic function.
- Subjects with increased risk of bleeding (i.e., use of anticoagulants or anti-platelet agents within 7 days before VG801 administration and subjects with international normalized ratio > 2 or Quick < 50% or partial thromboplastin time > 50 seconds, thrombocytopenia, as well as any other known coagulopathy).
- Breast-feeding woman.
- Any other condition that would not allow the potential subject to complete follow-up examinations during the study and, in the opinion of the Investigator, makes the potential subject unsuitable for the study.
- 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.
- Previous therapy with an approved gene therapy product.
- Participation in a study (investigational drug or medical device) in previous 6 months.
- Incapable of performing visual function testing for reasons other than poor vision.
- Any absolute contraindication to immunosuppressant regimen applied during and after administration of the IMP.
- Uncontrolled diabetics (HbA1c > 7%).
The study team makes the final eligibility decision.
Where it's taking place
- United States
- 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, 0-17 years, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; 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 EU CTIS; the study team decides eligibility.