AAVB-039 for Stargardt Disease

(CELESTE Trial)

Not yet recruiting at 1 trial location

Overseen By AAVantgarde Clinical Operations Lead

Age: < 65

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: AAVantgarde Bio Srl

Disqualifiers: Other trials, Surgery issues, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: Single administration

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called AAVB-039 for individuals with Stargardt disease, a genetic eye condition that can cause vision loss. The study aims to determine if AAVB-039 is safe and well-tolerated when injected into the eye. Researchers are also assessing the treatment's potential effectiveness. The trial involves different groups receiving various doses to identify the most effective one. Individuals with Stargardt disease due to a specific genetic mutation (ABCA4) who are willing to follow the study procedures are suitable candidates for this trial. As a Phase 1, Phase 2 trial, this research focuses on understanding how the treatment works in people and measuring its effectiveness in an initial, smaller group.

Do I need to stop my current medications for this trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that AAVB-039 is likely to be safe for humans?

A previous study tested AAVB-039 to assess its safety and tolerability. This treatment targets Stargardt disease, a genetic eye disorder. Although limited human data exists, early lab research has shown promise. Scientists believe it might be safe, but further testing is necessary to confirm this.

As AAVB-039 remains in early testing stages, researchers continue to gather information about its safety for humans. The FDA's approval to proceed with these tests indicates some confidence in the treatment's potential safety. However, as the study progresses, it is important to monitor updates as more information becomes available. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about AAVB-039 for Stargardt Disease because it introduces a novel approach to treating this condition. Unlike current treatments, which mainly focus on managing symptoms, AAVB-039 uses gene therapy to address the root cause by delivering a healthy copy of the ABCA4 gene directly into retinal cells. This innovative mechanism has the potential to not only slow down the progression of the disease but possibly restore vision by repairing the underlying genetic defect. By targeting the disease at its genetic source, AAVB-039 could offer a more effective and lasting solution compared to existing care options.

What evidence suggests that AAVB-039 might be an effective treatment for Stargardt disease?

Research has shown that AAVB-039 could be a promising treatment for Stargardt disease, a genetic eye disorder. In early animal tests, AAVB-039 successfully delivered genes into cells and produced the necessary proteins, indicating it might address the disease at its genetic root. The FDA has also recognized AAVB-039 as an orphan drug, a status given to promising treatments for rare diseases. Although data collection from human trials is ongoing, these early results suggest that AAVB-039 has potential in treating Stargardt disease. Participants in this trial will receive different dose levels of AAVB-039 to evaluate its safety and effectiveness. ^{1 6 7 8 9}

Are You a Good Fit for This Trial?

This trial is for individuals with Stargardt disease caused by ABCA4 gene mutations. Participants must be willing to follow the study's procedures and consent to them. Those who've had other gene or cell therapies, are in another drug trial, can't have subretinal surgery, or have complicating diseases cannot join.

Inclusion Criteria

Willingness to adhere to protocol per informed consent

I have Stargardt disease caused by an ABCA4 gene mutation.

Exclusion Criteria

I am willing to follow the study's requirements.

I do not have any health issues that would prevent eye surgery.

I have eye or other health conditions that could complicate treatment.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single subretinal injection of AAVB-039 at varying dose levels

Single administration

Follow-up

Participants are monitored for safety and initial efficacy following AAVB-039 administration

12 weeks

What Are the Treatments Tested in This Trial?

Interventions

• AAVB-039

Trial Overview The study tests AAVB-039 through a one-time injection into the eye's subretinal space. It aims to check how safe and tolerable this treatment is for people with Stargardt disease and also looks at its initial effectiveness.

How Is the Trial Designed?

Group I: Cohorts 4-6 Experimental Treatment 1 Intervention

AAVB-039 dose level 1, 2 or 3

Group II: Cohort 3 Experimental Treatment 1 Intervention

AAVB-039 dose level 3 (high dose)

Group III: Cohort 2 Experimental Treatment 1 Intervention

AAVB-039 dose level 2 (mid dose)

Group IV: Cohort 1 Experimental Treatment 1 Intervention

AAVB-039 dose level 1 (low dose)

Who Is Running the Clinical Trial?

AAVantgarde Bio Srl

Lead Sponsor

Trials

2

Recruited

100+

AAVantgarde Bio UK Ltd

Collaborator

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT07161544

A Study of AAVB-039 in Participants With Stargardt ... 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 ...

2. sofinnovapartners.com sofinnovapartners.com/news/aavantgarde-bio-announces-fda-fast-track-designation-for-aavb-039-for-the-treatment-of-stargardt-disease

AAVantgarde Bio Announces FDA Fast Track Designation ... AAVB-039 is currently being evaluated in the Phase 1/2 CELESTE clinical trial, which is assessing safety, tolerability, and preliminary efficacy ...

3. clinicaltrialvanguard.com clinicaltrialvanguard.com/news/aavb-039-fda-clearance-for-stargardt-disease-celeste-trial/

AAVB-039 FDA Clearance for Stargardt Disease Celeste ... This platform has demonstrated promising preclinical results, including high transduction, protein expression, and safety in animal models. The ...

4. aavantgarde.com aavantgarde.com/en/news/aavantgarde-announces-fda-orphan-drug-designation-and-uk-cta-approval-for-aavb-039-for-the-treatment-of-stargardt-disease/

AAVantgarde Announces FDA Orphan Drug Designation ... AAVB-039 is currently being evaluated in the Phase 1/2 CELESTE clinical trial, which assesses safety, tolerability, and preliminary efficacy of ...

5. biopharminternational.com biopharminternational.com/view/fda-designation-and-uk-approval-for-aavb-039-highlight-progress-in-complex-gene-therapy-development

FDA Designation and UK Approval for AAVB-039 Highlight ... AAVB-039 received orphan drug designation from the FDA and CTA approval from the UK's MHRA, advancing its development for Stargardt disease.

6. ophthalmologytimes.com ophthalmologytimes.com/view/aavantgarde-s-aavb-039-gene-therapy-to-treat-stargardt-disease-receives-orphan-drug-status-from-fda

AAVantgarde's AAVB-039 gene therapy to treat Stargardt ... AAVB-039 is currently being evaluated in the phase 1/2 CELESTE clinical trial, which is assessing safety, tolerability, and preliminary efficacy ...

7. aavantgarde.com aavantgarde.com/en/news/aavantgarde-receives-fda-ind-clearance-to-progress-stargardt-disease-program-aavb-039/

AAVantgarde Receives FDA IND Clearance to Progress ... AAVantgarde Receives FDA Clearance to Progress Stargardt Disease Asset, AAVB-039, into CELESTE, a Phase 1/2 Clinical Trial.

8. cgtlive.com cgtlive.com/view/aavantgarde-stargardt-gene-therapy-aavb-039-garners-fda-fast-track-designation

AAVantgarde's Stargardt Gene Therapy AAVB-039 ... AAVB-039 is intended to deliver a functional copy of the disease-targeted ABCA4 gene. The gene therapy product utilizes a dual AAV vector, as ...

9. sofinnovapartners.com sofinnovapartners.com/news/AAVantgarde%20presents%20positive%20clinical%20data%20from%20its%20AAVB-081%20program%20for%20Usher%201B%20and%20preclinical%20data%20from%20its%20AAVB-039%20program%20for%20Stargardt%20at%20the%20ARVO%202025%20annual%20meeting

AAVantgarde presents positive clinical data from its AAVB- ... AAVantgarde presents positive clinical data from its AAVB-081 program for Usher 1B and preclinical data from its AAVB-039 program for Stargardt ...