The STELLAR
Clinical Trial

The STELLAR clinical trial is a research study of a potential new treatment for people with an eye condition called ABCA4 retinopathy – the most common form is called Stargardt disease. Retinopathy means “damage to the retina.” The retina is the part of the back of eye that detects light.

Stargardt disease is a genetic condition caused by mutations in a gene called ABCA4. Without normal ABCA4 protein, the cells in the retina cannot clear out toxic waste products. This leads to loss of vision.

Study Purpose

The main purpose of the STELLAR research study is to determine if the study drug, called ACDN-01, is safe when given to people with Stargardt disease.

The STELLAR research study may also help scientists understand the potential of ACDN-01 to slow down or stop the damage to the retina experienced by people with Stargardt disease and other ABCA4 retinopathies.

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Study Drug: ACDN-01

ACDN-01 is an investigational treatment. This means ACDN-01 has not been approved by the U.S. Food and Drug Administration (FDA) or any other regulatory agency for the treatment of Stargardt disease or any other medical condition.

ACDN-01 is a new type of drug, called an RNA exon editor. It acts on RNA in the retina and is designed to correct the mutations that lead to Stargardt disease. ACDN-01 contains a healthy copy of sections of ABCA4 RNA, called exons. These healthy sections replace the sections of the ABCA4 RNA that contain the mutations and create healthy ABCA4 RNA in the retina. This is intended to produce normal ABCA4 protein that can then help clear the eye of toxic waste products.

Before being treated in the STELLAR research study, each participant will be tested to confirm that ACDN-01 would edit the mutations present in their ABCA4 RNA.

ACDN-01 is administered by sub-retinal injection to a central part of the retina called the macula. We believe one treatment may last for many years, perhaps even a person’s lifetime.

Participation Criteria

You may be eligible to participate in the STELLAR research study if you are:

Aged 18 or older.

Diagnosed with Stargardt disease type 1 or cone-rod dystrophy.

Confirmed to have the mutations that could be treated by ACDN-01. This will be done through a simple blood test before being enrolled in the study.

*The above inclusion criteria are representative and not a full list. If you are considering participating in the STELLAR research study, discuss these criteria with your ophthalmologist.

Study Locations

STELLAR is a U.S.-based study that is currently enrolling, or in the future will be enrolling, participants in the locations indicated on the map below:

TRAVEL FOR STUDY-RELATED ASSESSMENTS WILL BE PROVIDED FOR ELIGIBLE STUDY PARTICIPANTS AND THEIR CARE PARTNERS

At this time, you must live in the U.S. to participate in the STELLAR research study.
For information about specific study sites, please visit clinicaltrials.gov

How To Participate

To see if participation in STELLAR is right for you, speak to your ophthalmologist or one of the study doctors at the study locations.

Participants will be given a single dose of ACDN-01 in one eye, through a one-time subretinal injection. Participants will then visit the study site regularly over several years to assess the impact of ACDN-01 on their disease.

This is an open-label study. All participants will be treated with study drug in one eye.

STELLAR Frequently Asked Questions (FAQs)

To determine if the study drug ACDN-01 is safe when given to people with Stargardt disease or cone-rod dystrophy, and to learn about the effects of ACDN-01 at different doses, including whether it can potentially slow down or stop the progression of the disease.

ACDN-01 is a new investigational treatment called an RNA exon editor. This means that ACDN-01 has not yet been approved by the FDA or any other regulatory agency for the treatment of Stargardt disease or any other medical condition. ACDN-01 contains a healthy copy of sections of ABCA4 RNA, called exons. These healthy sections replace the sections of the ABCA4 RNA that contain the mutations to create healthy ABCA4 RNA in the retina. This is intended to produce normal ABCA4 protein, which then helps clear the eye of toxic waste products.

If you participate in the study, you will have a subretinal procedure to inject the study drug in one eye. After the procedure, you will go to a study site at regular intervals to assess how you are doing and to assess the effects of ACDN-01.

Participants will be in the study for approximately five years, which includes the one-time subretinal injection of ACDN-01 and then regular monitoring for two years after treatment. For the final three years, there will be annual visits.