A trial in patients with ABCA4 mutation-associated retinal dystrophy.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

VeonGen Therapeutics GmbH

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Eye Diseases [C11]

Decision date (initial)

2025-09-17

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Funding sources

VeonGen Therapeutics GmbH (formerly ViGeneron GmbH)

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy

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

Conditions and MedDRA coding

Biallelic ABCA4 Mutation-Associated Retinal Dystrophy.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 9

1. Written informed consent.
2. Subjects aged ≥ 6 years.
3. Molecular diagnosis was confirmed due to ABCA4 mutations (homozygotes or compound heterozygotes) by a CLIA (Clinical Laboratory Improvement Amendments)-certified laboratory.
4. Clinical diagnosis of a macular lesion phenotypically consistent with retinal disease.
5. Visual acuity not better than 20/60 in the study eye.
6. Detectable outer nuclear layer thickness in the macular region using OCT.
7. 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.
8. Negative pregnancy test.
9. 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).

Exclusion criteria 14

1. Pre-existing eye conditions such as uveitis, glaucoma, or diabetic retinopathy.
2. Systemic diseases that would preclude the planned surgery or interfere with the interpretation of study results.
3. History of intraocular surgery within the previous 6 months.
4. Previous participation in a gene therapy trial.
5. Previous therapy with an approved gene therapy product.
6. Participation in a study (investigational drug or medical device) in previous 6 months.
7. Incapable of performing visual function testing for reasons other than poor vision.
8. Any absolute contraindication to immunosuppressant regimen applied during and after administration of the IMP.
9. Uncontrolled diabetics (HbA1c > 7%).
10. Positive for hepatitis C or human immunodeficiency virus (HIV) infection.
11. Abnormal laboratory values (3  upper limit of normal [ULN]) in blood analysis including renal and hepatic function.
12. 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).
13. Breast-feeding woman.
14. 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.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 10

1. Safety Endpoint: Number of Adverse events (ocular and systemic)
2. Safety Endpoint: Evaluation of the humoral immune responses against the AAV capsid
3. Safety Endpoint: Measurement of vector in blood, tears, and saliva (vector shedding) until two negative samples have been obtained
4. Safety Endpoint: Hematology, chemistry, and urinalysis
5. Safety Endpoint: Comprehensive ophthalmic assessments including slit lamp examination coupled with imaging including optical coherence tomography (OCT), fundus photography, intraocular pressure monitoring, and fundus autofluorescence imaging.
6. Preliminary Efficacy Endpoint: Visual Function (BCVA, Pelli Robson contrast test, OCT, fundus photography, IOP, fundus autofluorescence imaging)
7. Preliminary Efficacy Endpoint: Morphological retinal measures (Fundus autofluorescence imaging , Optical coherence tomography)
8. Preliminary Efficacy EndPoint: Local retinal function measures for rods and cones ( Full-field stimulus threshold testing)
9. Preliminary Efficacy Endpoint: Retinal function measures (Goldmann kinetic visual fields, Scotopic and photopic microperimetry)
10. Preliminary Efficacy Endpoint: Mobility function (novel virtual reality mobility test)

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

VeonGen Therapeutics GmbH

Sponsor organisation

VeonGen Therapeutics GmbH

Address

Am Klopferspitz 19, Martinsried Martinsried

City

Planegg

Postcode

82152

Country

Germany

Scientific contact point

Organisation

VeonGen Therapeutics GmbH

Contact name

Clinical Trial Information

Public contact point

Organisation

VeonGen Therapeutics GmbH

Contact name

Clinical Trial Information

Locations

1 EU/EEA country · 2 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 21 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

4 submissions — initial application plus substantial / non-substantial modifications