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

Part of paid clinical trials in Phoenix, Arizona.

Sponsor
AAVantgarde Bio Srl
Study ID
NCT07161544
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

  • Stargardt Disease

Eligibility Criteria

Sex
ALL
Age
8 Years - 55 Years
Healthy Volunteers
Not accepted

Interventions

  • AAVB-039 — BIOLOGICAL
    Single subretinal administration

Study Details

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.

Key Dates

First listed
Sep 8, 2025
Start date
Sep 29, 2025
Status verified
Jun 2026
Primary completion
Jul 31, 2032
Completion
Jul 31, 2032

Study Design

Enrollment
75 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Cohort 1
    AAVB-039 dose level 1 (low dose)
  • Experimental: Cohort 2
    AAVB-039 dose level 2 (mid dose)
  • Experimental: Cohort 3
    AAVB-039 dose level 3 (high dose)
  • Experimental: Cohorts 4-5
    AAVB-039 dose level 1, 2 or 3

Primary Outcome Measure

To measure the number and severity of treatment related adverse events following treatment with AAVB-039 [ Time Frame: 0-60 months ]

Central Contacts

Locations (6)

FacilityCityStateZIPSite coordinators
Associated Retina ConsultantsPhoenixArizona85020
Benjamin Bakall (PRINCIPAL_INVESTIGATOR)
Retina Vitreous Associates Medical GroupLos AngelesCalifornia90211
Kristina Passoian, Trials Director
[email protected]
Study Referrals
[email protected]
David Liao (PRINCIPAL_INVESTIGATOR)
Mayo ClinicRochesterMinnesota55905
Suzy Wernimont
[email protected]
507-538-8119
Brittni Scruggs (PRINCIPAL_INVESTIGATOR)
Retina Consultants of TexasBellaireTexas77401
Kenneth Fan (PRINCIPAL_INVESTIGATOR)
Retina Foundation of the SouthwestDallasTexas75231
Senior Clinical Research Coordinator
[email protected]
Karl Csaky (PRINCIPAL_INVESTIGATOR)
University of UtahSalt Lake CityUtah84112
Kimberley Wegner Lead Coordinator
[email protected]
Paul Bernstein (PRINCIPAL_INVESTIGATOR)

Find similar trials in Phoenix, AZ

By research site
Associated Retina Consultants· Phoenix, AZRetina Vitreous Associates Medical Group· Los Angeles, CAMayo Clinic· Rochester, MNRetina Consultants of Texas· Bellaire, TXRetina Foundation of the Southwest· Dallas, TXUniversity of Utah· Salt Lake City, UT

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