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NCT Number: NCT07161544

A Study of AAVB-039 in Participants With Stargardt Disease (STGD1)

The purpose of the 039-101 study is to evaluate the safety and tolerability of a single subretinal injection of AAVB-039 in participants with Stargardt disease secondary to a biallelic mutation of the ABCA4 gene. The study will also assess initial efficacy following AAVB-039 administration.

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Key information

Conditions

Stargardt Disease Congenital, Hereditary, and Neonatal Diseases and Abnormalities Eye Diseases Eye Diseases, Hereditary Genetic Diseases, Inborn Macular Degeneration Retinal Degeneration Retinal Diseases

Age range

8 year–55 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Moorfields Eye Hospital NHS Foundation Trust, London, England, United Kingdom

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Molecular diagnosis of Stargardt disease due to ABCA4 mutation
  • Willingness to adhere to protocol per informed consent

Exclusion criteria

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

Treatment and study plan

AAVB-039

Biological

Single subretinal administration

Primary outcomes

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

    Time frame: 0-60 months

Secondary outcomes

  1. Change in the area of atrophy (assessed via short wavelength fundus autofluorescence (SW-FAF), following treatment with AAVB-039

    Time frame: 0-60 months

  2. Change in ellipsoid zone (EZ) loss following treatment with AAVB-039

    Time frame: 0-60 months

  3. Change in macular sensitivity assessed via microperimetry following treatment with AAVB-039

    Time frame: 0-60 months

  4. Change in visual acuity following treatment with AAVB-039

    Time frame: 0-60 months

Study contacts

Contact information is provided by the study sponsor or research team.

AAVantgarde Clinical Operations Lead

CONTACT

[email protected]

+448000465680

Sponsors and collaborators

Lead sponsor

AAVantgarde Bio Srl

Industry

Collaborators

  • AAVantgarde Bio UK Ltd

Registry information

Official study title

An Open-label, Multicenter, Two Part, Ascending Dose Followed by a Controlled Trial to Assess the Safety and Efficacy of a Subretinal Administration of AAVB-039 in Participants With Stargardt Disease (STGD1) (CELESTE)

Acronym: CELESTE

Important dates

Study start
2025
Primary completion
2032
Study completion
2032
First posted
Sep 8, 2025
Registry last updated
Jul 30, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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