2025-522207-15-01RecruitingPhase 2
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)
Aavantgarde Bio S.r.l.5 sites in 4 countries21 target enrollmentStarted: December 29, 2025Last updated:
Conditions
Interventions
Drugs
Trial Snapshot
- Phase
- Phase 2
- Status
- Recruiting
- Sponsor
- Enrollment
- 21
- Locations
- 5
- Primary Endpoint
- To measure the number and severity of treatment related adverse events following treatment with AAVB-039
Study Overview
Brief Summary
To determine the safety, tolerability and efficacy of a single administration of AAVB-039 via subretinal injection
Study Design
- Allocation
- Na
- Primary Purpose
- Follow-up Period
- Masking
- None
Eligibility Criteria
- Ages
- 0 years to 64 years (18-64 Years, 0-17 Years)
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Molecular diagnosis of Stargardt disease due to ABCA4 mutation
- •Aged ≥8 years to ≤55 years inclusive
- •Willingness to adhere to protocol per informed consent
- •No history of vitreoretinal or retinal macular surgery, No current history of cancer for 5 years, No history of myocardial infarct, stroke, or other heart conditions < 6 months
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
Arms & Interventions
AAVB-039
Test
Intervention: AAVB-039 (Drug)
Outcomes
Primary Outcomes
To measure the number and severity of treatment related adverse events following treatment with AAVB-039
To measure the number and severity of treatment related adverse events following treatment with AAVB-039
Secondary Outcomes
- Change in the area of atrophy (assessed via short wavelength fundus autofluorescence (SW-FAF), following treatment with AAVB-039.
- Change in ellipsoid zone (EZ) loss following treatment with AAVB-039.
- Change in macular sensitivity assessed via microperimetry following treatment with AAVB-039
- Change in visual acuity following treatment with AAVB-039
Investigators
Nicola Tuckwell
Scientific
Aavantgarde Bio S.r.l.
Study Sites (5)
Loading locations...
Similar Trials
Recruiting
Phase 2
A Study to Assess Adverse Events and Change in Disease Activity of Oral ABBV-453 Alone or in Combination With Subcutaneous and/or Oral Antimyeloma Agents in Adult Participants With Multiple Myeloma (MM)Multiple Myeloma (MM).2024-517140-65-00AbbVie Deutschland GmbH & Co. KG76
Recruiting
Phase 2
Study of Intravenous ABBV-400 in Combination With Intravenous Budigalimab to Assess Adverse Events and Change in Disease Activity in Adult Participants With Advanced or Metastatic Non-Squamous NSCLCAdvanced or Metastatic Non-Squamous NSCLC2024-514465-18-00AbbVie Deutschland GmbH & Co. KG105
Recruiting
Phase 2
Study to test the safety and how well patients with severe hemophilia A respond to treatment with BAY 2599023 (DTX 201), a drug therapy that delivers a healthy version of the defective Factor VIII gene into the nucleus of liver cells using an altered, non-infectious virus (AAV) as a "shuttle".Hemophilia A2023-505827-29-00Bayer AG, Bayer AG6
Recruiting
Phase 1
A Phase 1 Study of ABF-101 in Single- and Multiple-Ascending DosesAge Related Macular Degeneration (ARMD)AMD - Age-Related Macular DegenerationNCT07330674Aptabio Therapeutics, Inc.68
Recruiting
Phase 2
Study of ASP2957 in Male Patients with X-linked Myotubular Myopathy Who Need VentilatorsX-linked myotubular myopathy2025-523213-29-00Astellas Gene Therapies Inc.1