A Study of AAVB-039 in Participants With Stargardt Disease (STGD1)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: AAVB-039.
- Who it may be relevant to
- Registry conditions: Stargardt Disease. Basic parameters: 8 years — 55 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, United Kingdom
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official 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)
Overview
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.
Interventions
- Biological AAVB-039
Single subretinal administration
Primary outcome measures
- To measure the number and severity of treatment related adverse events following treatment with AAVB-039 [Time frame: 0-60 months]
Secondary outcome measures (4)
- Change in the area of atrophy (assessed via short wavelength fundus autofluorescence (SW-FAF), following treatment with AAVB-039 [Time frame: 0-60 months]
- Change in ellipsoid zone (EZ) loss following treatment with AAVB-039 [Time frame: 0-60 months]
- Change in macular sensitivity assessed via microperimetry following treatment with AAVB-039 [Time frame: 0-60 months]
- Change in visual acuity following treatment with AAVB-039 [Time frame: 0-60 months]
Eligibility criteria
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Non-randomized
- Model
- Sequential
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 7 centers
- Associated Retina Consultants — Phoenix
- Retina Vitreous Associates Medical Group — Los Angeles
- University of Florida Health Science Center — Jacksonville
- Mayo Clinic — Rochester
- Retina Consultants of Texas — Bellaire
- Retina Foundation of the Southwest — Dallas
- University of Utah — Salt Lake City
United Kingdom · 2 centers
- Moorfields Eye Hospital NHS Foundation Trust — London
- The Retina Clinic — London
Identifiers
NCT: NCT07161544 · 039-101