ACDN-01 for ABCA4-related Stargardt Retinopathy

Study to Evaluate ACDN-01 in ABCA4-related Stargardt Retinopathy (STELLAR)

Recruiting Phase 1 Phase 2 Interventional Study
Stargardt Disease Cone Rod Dystrophy Juvenile Macular Degeneration Stargardt Disease 1
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
Ready to participate?

Review the details below, then apply to join this clinical trial.

At a Glance
Age
12 and older
Sex
Any
Trial phase
Phase 1/2
Study type
Interventional
Purpose
Treatment
Participants needed
15 (estimated)
Sponsor
Ascidian Therapeutics, Inc · Industry
Who this trial is looking for

This trial is looking for people with specific genetic eye diseases related to the ABCA4 gene. Participants will receive a single injection of the study drug ACDN-01 to see how safe it is and if it helps.

Are You a Good Fit for This Trial?

You may be able to join if

  • I have mutations in the ABCA4 gene
  • I have Stargardt disease type 1 or cone-rod dystrophy
  • I have damage in the macula of my study eye
  • I have a vision score of 20/50 or worse

You may not be able to join if

  • I have harmful mutations in other genes causing similar eye diseases
  • I have retinal disease not related to ABCA4
  • I have a serious health issue that might affect my study participation
  • I have a psychiatric condition including substance abuse disorder

Summarized in plain language from this trial's official eligibility criteria. The full criteria are further down this page — only the research team can confirm whether you qualify.

Think this trial could be right for you?

Answer a few quick questions to see if you may meet the eligibility requirements.

Check Your Eligibility
About This Trial

This study is an open-label, single ascending dose clinical trial in participants who have ABCA4-related retinopathies. This is the first-in-human clinical trial in which ACDN-01 will be evaluated for safety, tolerability, and preliminary efficacy following a single subretinal injection of ACDN-01.

Trial Locations
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Eligibility Criteria
Key Inclusion Criteria: * Presence of mutations in the ABCA4 gene * ABCA4 retinopathy phenotype (Stargardt disease type 1 or cone-rod dystrophy) * Area of atrophy located in the macula of the study eye * BCVA of 20/50 (0.4 logMAR) or worse Key Exclusion Criteria: * The presence of pathogenic or l…
Contacts

Associate Director, Clinical Operations

207-573-0412

researchtrials@ascidian-tx.com

CONTACT