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Gene Therapy

ACDN-01 for Stargardt Disease (STELLAR Trial)

Phase 1 & 2
Recruiting
Research Sponsored by Ascidian Therapeutics, Inc
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Presence of mutations in the ABCA4 gene
ABCA4 retinopathy phenotype (Stargardt disease type 1 or cone-rod dystrophy)
Must not have
The presence of pathogenic or likely pathogenic mutations in other genes known to cause cone-rod dystrophy or Stargardt maculopathy
Retinal disease other than ABCA4-related retinopathy
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 24 months
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing a new treatment called ACDN-01 in people with ABCA4-related retinopathies. It will be the first time ACDN-01 is given to humans

Who is the study for?
The STELLAR study is for individuals with ABCA4-related retinopathies, which include conditions like Stargardt Disease and various forms of macular degeneration. Participants should have a diagnosis related to these eye diseases.
What is being tested?
ACDN-01 is being tested in this trial. It's an experimental treatment given as a single subretinal injection to see if it's safe, tolerable, and potentially effective at treating the symptoms of ABCA4-related retinopathy.
What are the potential side effects?
Since ACDN-01 is new and this is its first use in humans, potential side effects are not fully known but may include typical risks associated with subretinal injections such as eye irritation, discomfort or infection.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have a mutation in the ABCA4 gene.
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I have been diagnosed with Stargardt disease type 1 or cone-rod dystrophy.
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I have thinning in the central part of my eye's retina.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have genetic mutations linked to specific eye diseases.
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I do not have ABCA4-related eye disease.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~24 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 24 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Safety and tolerability of ACDN-01 as measured by the number and severity of adverse events and serious adverse events.
Secondary study objectives
Maximum tolerated dose for subsequent clinical evaluation will be determined by review of all available safety and tolerability data.
To evaluate for evidence of preliminary efficacy based on fundus autofluorescence (FAF).
To evaluate for evidence of preliminary efficacy based on optical coherence tomography (OCT).
Other study objectives
To assess for evidence of preliminary efficacy of ACDN-01 based on best corrected visual acuity.
To assess for evidence of preliminary efficacy of ACDN-01 based on contrast sensitivity.
To assess for evidence of preliminary efficacy of ACDN-01 based on microperimetry.
+1 more

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

3Treatment groups
Experimental Treatment
Group I: Mid-dose of ACDN-01Experimental Treatment1 Intervention
One time mid-dose of ACDN-01.
Group II: Low dose of ACDN-01Experimental Treatment1 Intervention
One time low dose of ACDN-01.
Group III: High dose of ACDN-01Experimental Treatment1 Intervention
One time high dose of ACDN-01.

Find a Location

Who is running the clinical trial?

Ascidian Therapeutics, IncLead Sponsor
1 Previous Clinical Trials
50 Total Patients Enrolled
1 Trials studying Stargardt Disease
50 Patients Enrolled for Stargardt Disease
Alia RashidStudy DirectorAscidian Therapeutics
1 Previous Clinical Trials
50 Total Patients Enrolled
1 Trials studying Stargardt Disease
50 Patients Enrolled for Stargardt Disease
~9 spots leftby Aug 2030
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