ACDN-01 for Stargardt Disease
(STELLAR Trial)
Recruiting in Palo Alto (17 mi)
+9 other locations
Age: 18+
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 1 & 2
Recruiting
Sponsor: Ascidian Therapeutics, Inc
Disqualifiers: Other retinal diseases, psychiatric conditions, others
No Placebo Group
Approved in 1 Jurisdiction
Trial Summary
What is the purpose of 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.
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.
Eligibility Criteria
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.Inclusion Criteria
I have a mutation in the ABCA4 gene.
I have been diagnosed with Stargardt disease type 1 or cone-rod dystrophy.
I have thinning in the central part of my eye's retina.
+1 more
Exclusion Criteria
I have genetic mutations linked to specific eye diseases.
I do not have ABCA4-related eye disease.
Presence of a medical condition (systemic or ophthalmic), psychiatric condition, including substance abuse disorder, or physical examination or laboratory finding that may in the opinion of the principal investigator and sponsor preclude adherence to the scheduled study visits, safe participation in the study, or affect the results of the study.
Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
2-4 weeks
Treatment
Participants receive a single subretinal injection of ACDN-01 at low, medium, or high dose levels
1 day
1 visit (in-person)
Primary Follow-up
Participants are monitored for safety and preliminary efficacy endpoints
24 months
Regular visits (in-person and virtual)
Long-term Follow-up
Participants continue to be monitored for long-term safety and efficacy
36 months
Periodic visits (in-person and virtual)
Participant Groups
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.
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.
ACDN-01 is already approved in United States for the following indications:
🇺🇸 Approved in United States as ACDN-01 for:
- Stargardt disease
- ABCA4-related retinopathies
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
Vitreo Retinal AssociatesGainesville, FL
Cincinnati Eye InstituteCincinnati, OH
Retina Foundation of the SouthwestDallas, TX
Retina Consultants of TexasHouston, TX
More Trial Locations
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Who Is Running the Clinical Trial?
Ascidian Therapeutics, IncLead Sponsor
References
[Clinical Tests Testing New Therapies for Stargardt Disease]. [2019]To provide information on currently ongoing clinical trials for Stargardt disease.
Assessment of AAV Dual Vector Safety in theAbca4-/- Mouse Model of Stargardt Disease. [2021]Label="Purpose">Adeno-associated viral (AAV) gene therapy treatment for Stargardt disease currently requires a dual vector approach owing to the size of the ATP-binding cassette transporter family member gene (ABCA4). The nature of the dual vector system creates the potential for adverse events. Here we have investigated an overlapping adeno-associated viral ABCA4 dual vector system for signs of toxicity in Abca4-/- mice as a prelude to dual vector first in human clinical trials.
Longitudinal Microperimetric Changes of Macular Sensitivity in Stargardt Disease After 12 Months: ProgStar Report No. 13. [2021]Functional end points for clinical trials investigating the efficacy of emerging treatments for Stargardt disease type 1 (STGD1) are needed.
Stargardt macular dystrophy and therapeutic approaches. [2023]Stargardt macular dystrophy (Stargardt disease; STGD1; OMIM 248200) is the most prevalent inherited macular dystrophy. STGD1 is an autosomal recessive disorder caused by multiple pathogenic sequence variants in the large ABCA4 gene (OMIM 601691). Major advances in understanding both the clinical and molecular features, as well as the underlying pathophysiology, have culminated in many completed, ongoing and planned human clinical trials of novel therapies.The aims of this concise review are to describe (1) the detailed phenotypic and genotypic characteristics of the disease, multimodal imaging findings, natural history of the disease, and pathogenesis, (2) the multiple avenues of research and therapeutic intervention, including pharmacological, cellular therapies and diverse types of genetic therapies that have either been investigated or are under investigation and (3) the exciting novel therapeutic approaches on the translational horizon that aim to treat STGD1 by replacing the entire 6.8 kb ABCA4 open reading frame.
Deep Scleral Exposure: A Degenerative Outcome of End-Stage Stargardt Disease. [2021]To describe a distinct phenotypic outcome of outer retinal degeneration in a cohort of genetically confirmed patients with recessive Stargardt disease (STGD1).