#1 Clinical Trials Search in Europe

VG801-2

Clinical trials are investigating VG801-2 in people with biallelic ABCA4 mutation-associated retinal dystrophy. These studies are looking at safety, tolerability, and early signs of benefit in vision and retinal function. The trial is in a small group of patients in a Phase 1/2 study.

Table of contents

Trial overview

This clinical trial is studying VG801 given by subretinal use in patients with biallelic ABCA4 mutation-associated retinal dystrophy.[1] The study is interventional, which means researchers are giving the study treatment and then measuring what happens.[1]

The trial is designed as a Phase 1/2 study and has an enrollment of 18 people.[1] The status is listed as authorised.[1]

Who is being studied

The target group is people with biallelic ABCA4 mutation-associated retinal dystrophy.[1] “Biallelic” means both copies of the gene are changed, and this eye disease affects the retina, which is the light-sensing layer at the back of the eye.[1]

The source data does not list every eligibility rule, so the full study criteria would be needed to know exactly who can join.[1]

What the trials are measuring

The main focus is safety, including the number of ocular and systemic adverse events.[1] Ocular adverse events are problems in the eye, while systemic adverse events are problems elsewhere in the body.[1]

Researchers are also checking the body’s humoral immune response against the AAV capsid, which means they are looking for antibody reactions to the outer shell of the viral vector used in the study.[1] They are measuring vector shedding in blood, tears, and saliva until two negative samples are obtained.[1]

Other safety checks include hematology, chemistry, and urinalysis, which are standard lab tests for blood and urine.[1] The study also includes comprehensive ophthalmic assessments such as slit lamp examination, optical coherence tomography (OCT), fundus photography, intraocular pressure monitoring, and fundus autofluorescence imaging.[1]

Trial phase and status

This is an early Phase 1/2 trial.[1] In simple terms, this means the study is trying to learn whether the treatment is safe and tolerable, and whether there are early signs that it may help.[1]

The study is listed as authorised.[1] The brief summary says the safety work also aims to find dose(s) below the dose-limiting toxicity level that may still be useful for later studies.[1]

Patient tests and follow-up

The trial includes several tests of vision and retinal health.[1] These include best corrected visual acuity (BCVA), Pelli Robson contrast testing, Goldmann kinetic visual fields, scotopic and photopic microperimetry, full-field stimulus threshold testing, and imaging tests such as OCT and fundus autofluorescence imaging.[1]

The study also measures mobility function using a novel virtual reality mobility test.[1] This kind of test looks at how well a person can move around in a simulated environment, which can help show how vision changes affect daily life.[1]

For preliminary efficacy, the study is looking for changes in visual function, retinal structure, retinal function, and mobility.[1] “Preliminary efficacy” means early signs that the treatment may help, but not final proof.[1]

Trial IDPhaseCondition studiedStatusEnrollment
2024-520425-37-00Phase 1/2Biallelic ABCA4 mutation-associated retinal dystrophyAuthorised18

FAQ

  • What is VG801-2 being studied for? VG801-2 is being studied in people with biallelic ABCA4 mutation-associated retinal dystrophy. The trial is checking safety first and also looking for early signs that vision and retinal function may improve.
  • Who can join this trial? The trial is for patients with biallelic ABCA4 mutation-associated retinal dystrophy. The source data does not list all other eligibility rules, so participation depends on the full study criteria.
  • What phase is the VG801-2 trial? This is a Phase 1/2 trial. That means it is an early study that looks at both safety and early effectiveness signals.
  • What safety checks are being done? The study measures ocular and systemic adverse events, which means side effects in the eye and in the rest of the body. It also checks blood, tears, saliva, lab tests, immune response, and detailed eye exams.
  • What outcomes are being measured? The trial measures vision, retinal structure, retinal function, and mobility. Examples include best corrected visual acuity, imaging tests such as OCT, visual field tests, and a virtual reality mobility test.

Ongoing Clinical Trials on VG801-2

  • Safety and early effectiveness study of VG801 subretinal injection in patients with ABCA4 mutation-associated retinal dystrophy

    Not yet recruiting

    1 1
    Investigated drugs:
    Germany

Glossary

  • Biallelic: A person has two changed copies of the same gene, one from each parent.
  • ABCA4 mutation-associated retinal dystrophy: An inherited eye disease caused by changes in the ABCA4 gene that can damage the retina and affect vision.
  • Retina: The light-sensing layer at the back of the eye that helps you see.
  • Phase 1/2: An early clinical trial stage that looks at safety first and also checks for early signs that the treatment may help.
  • Interventional study: A study where participants receive a treatment or procedure that researchers are testing.
  • Adverse event: A medical problem that happens during a study. It may or may not be caused by the treatment.
  • Humoral immune response: The body’s antibody response. In this study, researchers are checking whether the immune system reacts to the AAV capsid.
  • AAV capsid: The outer shell of the viral vector used in the study. Researchers are checking whether the body makes an immune response against it.
  • Vector shedding: Checking whether the study vector can be found in body fluids such as blood, tears, or saliva.
  • Optical coherence tomography (OCT): A scan that gives detailed pictures of the layers of the retina.
  • Best corrected visual acuity (BCVA): A measure of how clearly a person can see with the best possible glasses or contact lens correction.
  • Microperimetry: A test that checks how sensitive different parts of the retina are to light.

References