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

NCT ID: NCT07161544

Last Updated: 2026-01-13

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

75 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-09-29

Study Completion Date

2032-07-31

Brief Summary

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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.

Detailed Description

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Conditions

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Stargardt Disease

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cohort 1

AAVB-039 dose level 1 (low dose)

Group Type EXPERIMENTAL

AAVB-039

Intervention Type BIOLOGICAL

Single subretinal administration

Cohort 2

AAVB-039 dose level 2 (mid dose)

Group Type EXPERIMENTAL

AAVB-039

Intervention Type BIOLOGICAL

Single subretinal administration

Cohort 3

AAVB-039 dose level 3 (high dose)

Group Type EXPERIMENTAL

AAVB-039

Intervention Type BIOLOGICAL

Single subretinal administration

Cohorts 4-5

AAVB-039 dose level 1, 2 or 3

Group Type EXPERIMENTAL

AAVB-039

Intervention Type BIOLOGICAL

Single subretinal administration

Interventions

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AAVB-039

Single subretinal administration

Intervention Type BIOLOGICAL

Eligibility Criteria

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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
Minimum Eligible Age

8 Years

Maximum Eligible Age

55 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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AAVantgarde Bio Srl

INDUSTRY

Sponsor Role lead

AAVantgarde Bio UK Ltd

UNKNOWN

Sponsor Role collaborator

Responsible Party

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Responsibility Role SPONSOR

Locations

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Retina Consultants of Texas

Bellaire, Texas, United States

Site Status RECRUITING

Retina Foundation of the Southwest

Dallas, Texas, United States

Site Status RECRUITING

Moorfields Eye Hospital NHS Foundation Trust

London, England, United Kingdom

Site Status RECRUITING

The Retina Clinic

London, England, United Kingdom

Site Status RECRUITING

Countries

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United States United Kingdom

Central Contacts

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AAVantgarde Clinical Operations Lead

Role: CONTACT

+448000465680

Facility Contacts

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Senior Clinical Research Coordinator

Role: primary

Other Identifiers

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039-101

Identifier Type: -

Identifier Source: org_study_id

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