ACDN-01 for Stargardt Disease

(STELLAR Trial)

Recruiting at 9 trial locations

Overseen By Associate Director, Clinical Operations

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Ascidian Therapeutics, Inc

Disqualifiers: Other retinal diseases, psychiatric conditions, others

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

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 1

Duration: 1 day

13 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called ACDN-01 for individuals with Stargardt Disease, a genetic eye condition affecting vision. The goal is to determine if ACDN-01 is safe and effective when administered as a one-time injection into the eye. Participants will receive a low, mid, or high dose of ACDN-01 to identify the optimal dose. Suitable candidates for this trial include those diagnosed with Stargardt Disease or cone-rod dystrophy, caused by changes in the ABCA4 gene, and who experience noticeable vision loss. As a Phase 1 trial, this research aims to understand how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.

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.

Is there any evidence suggesting that ACDN-01 is likely to be safe for humans?

Research shows that ACDN-01 is undergoing its first human trials to assess safety and tolerability. As this is an early-stage trial, detailed safety information from other studies is not yet available. However, the FDA has approved the trial plan and granted it fast-track status, indicating promise in lab studies. This trial will test different doses—low, medium, and high—administered as a one-time injection. As this is the first study in humans, researchers will closely monitor safety and side effects. Participants will contribute to understanding how well people tolerate ACDN-01 and what side effects might occur. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Stargardt Disease?

Researchers are excited about ACDN-01 for Stargardt Disease because it offers a novel approach compared to current treatments like nutritional supplements, gene therapy, or visual aids. Unlike these options, which primarily manage symptoms, ACDN-01 is designed to directly address the underlying causes of the disease. This treatment is unique because it is delivered in a single dose, potentially simplifying the treatment process. Moreover, ACDN-01 is available in low, mid, and high doses, allowing researchers to determine the most effective dose for patients, which could lead to more personalized treatment strategies.

What evidence suggests that ACDN-01 might be an effective treatment for Stargardt Disease?

Research has shown that ACDN-01 is a promising new treatment for Stargardt Disease, a genetic eye disorder. This treatment uses RNA exon editing to target the genetic cause of the disease. In early studies with eye tissue and animals, ACDN-01 demonstrated positive effects. Although human studies provide limited information, ACDN-01's innovative approach offers hope for addressing the genetic issues of Stargardt Disease. Initial results are encouraging, but further research is needed to fully understand its impact on improving vision. ^{1 3 5 6 7}

Who Is on the Research Team?

Alia Rashid

Principal Investigator

Ascidian Therapeutics

Are You a Good Fit for This Trial?

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

BCVA of 20/80 (0.6 logMAR) or worse

I have a mutation in the ABCA4 gene.

I have been diagnosed with Stargardt disease type 1 or cone-rod dystrophy.

Exclusion Criteria

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.

I have genetic mutations linked to specific eye diseases.

I do not have ABCA4-related eye disease.

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 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)

What Are the Treatments Tested in This Trial?

Interventions

• ACDN-01

Trial Overview 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.

How Is the Trial Designed?

Group I: Mid-dose of ACDN-01 Experimental Treatment 1 Intervention

One time mid-dose of ACDN-01.

Group II: Low dose of ACDN-01 Experimental Treatment 1 Intervention

One time low dose of ACDN-01.

Group III: High dose of ACDN-01 Experimental Treatment 1 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

Who Is Running the Clinical Trial?

Ascidian Therapeutics, Inc

Lead Sponsor

Trials

2

Recruited

60+

Published Research Related to This Trial

In a study of 200 patients with Stargardt disease type 1 (STGD1), microperimetry revealed a significant yearly decline in macular sensitivity, with an average decrease of -0.68 dB, indicating a measurable progression of the disease over approximately one year.

The increase in deep scotoma points by an average of 1.56 points per year suggests worsening visual function, highlighting the potential of microperimetry as a valuable tool for assessing treatment efficacy in clinical trials for STGD1.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Longitudinal Microperimetric Changes of Macular Sensitivity in Stargardt Disease After 12 Months: ProgStar Report No. 13. Schönbach, EM., Strauss, RW., Muñoz, B., et al.[2021]

Stargardt macular dystrophy (STGD1) is the most common inherited macular dystrophy caused by mutations in the ABCA4 gene, leading to significant advances in understanding its clinical and molecular features.

Current research is exploring various therapeutic interventions, including pharmacological treatments, cellular therapies, and innovative genetic therapies aimed at replacing the entire ABCA4 gene, which could potentially offer new hope for patients with STGD1.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Stargardt macular dystrophy and therapeutic approaches. Fujinami, K., Waheed, N., Laich, Y., et al.[2023]

There are currently eight ongoing clinical trials for Stargardt disease, focusing on various treatment strategies including gene therapy, neuroprotection, and stem cell therapy.

These trials aim to address the disease through different mechanisms, such as inhibiting toxic vitamin A dimers and restoring the function of the ABCA4 gene, which raises hope for effective treatments in the future.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Clinical Tests Testing New Therapies for Stargardt Disease]. Kousal, B., Ďuďáková, Ľ., Hlavatá, L., et al.[2019]

In a study of 12 patients with recessive Stargardt disease (STGD1), a distinct phenotype was observed characterized by advanced outer retinal degeneration, leading to a pale fundus appearance due to scleral exposure after approximately 50 years of disease progression.

The patients exhibited severe visual impairment, with best-corrected visual acuity ranging from 20/400 to hand motion, and significant loss of outer retinal structures was confirmed through imaging techniques, indicating a severe impact on retinal function.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Deep Scleral Exposure: A Degenerative Outcome of End-Stage Stargardt Disease. Lee, W., Zernant, J., Nagasaki, T., et al.[2021]

The overlapping adeno-associated viral (AAV) dual vector system for delivering the ABCA4 gene showed no signs of toxicity in Abca4-/- mice, indicating it is safe for potential use in humans.

Both structural and functional assessments of the retina, including optical coherence tomography and electroretinography, confirmed that the dual vector system did not cause any adverse effects compared to sham injections, supporting its advancement to clinical trials.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Assessment of AAV Dual Vector Safety in theAbca4-/- Mouse Model of Stargardt Disease. McClements, ME., Barnard, AR., Charbel Issa, P., et al.[2021]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06467344

Study to Evaluate ACDN-01 in ABCA4-related Stargardt ... To assess for evidence of preliminary efficacy of ACDN-01 based on patient reported outcome measures.

2. ophthalmologytimes.com ophthalmologytimes.com/view/fda-approves-trial-plan-and-fast-tracks-ascidian-therapeutics-acdn-01-in-stargardt-disease

FDA approves trial plan and fast tracks Ascidian ... ACDN-01 is the first-ever RNA exon editor to enter clinical development and the only clinical-stage therapeutic targeting the genetic cause of Stargardt ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12142558/

Innovative therapies for inherited retinal dystrophies Preclinical studies using retinal explants and non-human primates have shown positive effects. The ongoing STELLAR trial in Phase 1/2 (NCT06467344), will begin ...

4. ctv.veeva.com ctv.veeva.com/study/study-to-evaluate-acdn-01-in-abca4-related-retinopathy-stellar

Study to Evaluate ACDN-01 in ABCA4-related Stargardt ... The study is designed to evaluate the safety, tolerability, and evidence of biological effect of SAD levels (low, medium, and high) of ACDN-01 ...

5. retinalphysician.com retinalphysician.com/issues/2025/october/novel-therapies-for-stargardt-disease/

Novel Therapies for Stargardt Disease No clinically significant changes in visual function were observed. However, 27% of treated eyes showed worsening hypoautofluorescent changes, ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT06445322

Prescreening Study to Identify Potential Stargardt ... Members are chosen based on the scientific skills and knowledge needed to monitor the particular trial. Also called a data safety and monitoring board, or DSMB.

7. withpower.com withpower.com/trial/phase-2-macular-degeneration-5-2024-69fe6

ACDN-01 for Stargardt Disease (STELLAR Trial) In a study of 200 patients with Stargardt disease type 1 (STGD1), microperimetry revealed a significant yearly decline in macular sensitivity, with an average ...