An Open-label, Multicenter Trial to Assess Safety and Efficacy of a Subretinal Administration of AAVB-039

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Aavantgarde Bio S.r.l.

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Eye Diseases [C11], Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Decision date (initial)

2026-04-10

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

External identifiers

EU CT number

2025-522207-15-01

ClinicalTrials.gov

NCT07161544

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Dose response, Pharmacogenomic, Prophylaxis, Therapy, Diagnosis, Efficacy, Safety

To determine the safety, tolerability and efficacy of a single administration of AAVB-039 via subretinal injection

Conditions and MedDRA coding

Stargardt Disease (STGD1)

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. Molecular diagnosis of Stargardt disease due to ABCA4 mutation
2. Aged ≥8 years to ≤55 years inclusive
3. Willingness to adhere to protocol per informed consent
4. No history of vitreoretinal or retinal macular surgery, No current history of cancer for 5 years, No history of myocardial infarct, stroke, or other heart conditions < 6 months

Exclusion criteria 5

1. Unwillingness to meet the requirements of the study
2. Participation in a clinical study with another Investigation Medicinal Product
3. Previous participation in another gene or cell therapy trial
4. Any condition that would preclude subretinal surgery
5. Complicating ocular and/or systemic diseases

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. To measure the number and severity of treatment related adverse events following treatment with AAVB-039

Secondary endpoints 4

1. Change in the area of atrophy (assessed via short wavelength fundus autofluorescence (SW-FAF), following treatment with AAVB-039.
2. Change in ellipsoid zone (EZ) loss following treatment with AAVB-039.
3. Change in macular sensitivity assessed via microperimetry following treatment with AAVB-039
4. Change in visual acuity following treatment with AAVB-039

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Aavantgarde Bio S.r.l.

Sponsor organisation

Aavantgarde Bio S.r.l.

Address

Via Uberto Visconti Di Modrone 18

City

Milan

Postcode

20122

Country

Italy

Scientific contact point

Organisation

Aavantgarde Bio S.r.l.

Contact name

Nicola Tuckwell

Public contact point

Organisation

Aavantgarde Bio S.r.l.

Contact name

Tessa Brandon

Third parties 1

Locations

4 EU/EEA countries · 4 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 45 files

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

Application history

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