Health TrialsbyHealthPanel

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

ConditionStargardt Disease
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age8-55
SponsorAAVantgarde Bio Srl

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Molecular diagnosis of Stargardt disease due to ABCA4 mutation

Willingness to adhere to protocol per informed consent

Disqualifiers

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

Trial design

Design model

Sequential

Treatments tested in this trial

  • AAVB-039

    Biological/Vaccine

    Single subretinal administration

Treatment groups

75 Participants
are divided into 4 treatment groups
Group A: Cohort 1Experimental treatment 1 intervention
Group B: Cohort 2Experimental treatment 1 intervention
Group C: Cohort 3Experimental treatment 1 intervention
Group D: Cohorts 4-5Experimental treatment 1 intervention

Trial outcomes

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

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

AAVantgarde Bio Srl

Lead sponsor

AAVantgarde Bio UK Ltd

Collaborator

Source ClinicalTrials.gov
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