If you’re living with Stargardt disease — or caring for a child who is — Ascidian is conducting two clinical studies that may be of interest to you. Each study has a different purpose, and each supports future progress in potentially treating Stargardt disease.
If you or your child (ages 12+) has Stargardt disease, you may be eligible to take part in the STELLAR study.
This clinical trial is testing ACDN-01, a potential new treatment. It is given to participants as a one-time injection to the back of the eye.
The study’s main goal is to understand whether ACDN-01 is safe when given to people with Stargardt disease.
It may also help researchers understand if ACDN-01 slows or stops the damage to the eye experience by people with Stargardt disease.
If you or your child (ages 5+) has Stargardt disease, you may be eligible to take part in the STARPATH study.
This is what we call a pre-screening study, which means you do not get a study drug or injection. Instead, you’ll receive genetic testing and high-quality retinal imaging. These tests can help provide you more information about your or your child’s disease and also let you know if you or your child qualify for any future clinical trials with Ascidian.
Participants may also be prioritized for future ACDN-01 studies.
Stargardt disease is an inherited eye condition passed down through families.
There are currently no approved treatments.
It is caused by changes known as mutations in a gene named ABCA4. The ABCA4 gene provides instructions for making protein that helps keep the retina — the light-sensitive tissue at the back of the eye — healthy.
When ABCA4 protein does not work properly, waste materials build up in the retina and can damage the light-sensing cells responsible for central vision, leading to gradual vision loss.
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 study, each participant will be tested to confirm they have mutations within the ABCA4 gene.
ACDN-01 is administered by injection to the back of the eye. We believe one treatment may last for many years, perhaps even a person’s lifetime.
At this time, ACDN-01 may only be administered to people participating in the STELLAR study, who must be ages 12 and older.
You may be eligible to participate in the STELLAR study if you are:
Aged 12 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.
YOU MAY BE ELIGIBLE TO PARTICIPATE IN THE STARPATH STUDY IF YOU ARE:
Aged 5 or older.
Diagnosed with ABCA4-related Stargardt disease or cone-rod dystrophy.
*The above inclusion criteria are representative and not a full list. If you are considering participating in the STELLAR study or the STARPATH study, discuss these criteria with your doctor, and answer a few questions to learn more.
TRAVEL FOR STUDY-RELATED ASSESSMENTS WILL BE PROVIDED FOR ELIGIBLE STUDY PARTICIPANTS AND THEIR CARE PARTNERS
The STELLAR study and the STARPATH study are both U.S.-based, and open to participants who live in the United States, as well as participants who live Canada or Mexico who are able to travel to a study location in the United States. Each study is currently enrolling, or in the future will be enrolling participants in the locations indicated on the map below:
At this time, you must live in the U.S., Canada or Mexico to participate in the STELLAR study or the STARPATH study.
Study Sites:
California:
University of San Francisco
San Francisco, CA
Florida:
Vitreo Retinal Associates
Gainesville, FL
Bascom Palmer Eye Institute
Miami, FL
Maryland:
Wilmer Eye Institute at John Hopkins
Baltimore, MD
Massachusetts:
Massachusetts Eye and Ear
Boston, MA
Michigan:
University of Michigan Kellogg Eye Center
Ann Arbor, MI
Oregon:
Casey Eye Institute OHSU
Portland, OR
Ohio:
Cincinnati Eye Institute
Cincinnati, OH
Texas:
Retina Foundation of the Southwest
Dallas, TX
Retina Consultants of Texas
Houston, TX
To see if participation in STELLAR or STARPATH is right for you, answer a few questions and speak to your doctor or one of the study doctors at the study locations.
Participants will be given a single dose of ACDN-01 through a one-time injection to the back of one eye. 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, meaning everyone receives the study drug and both participants and the study team know the treatment being given. The study is for children ages 12 and older and adults with Stargardt disease type 1 or cone rod dystrophy. All participants will be treated in one eye.
For more details, please contact 
.
Participants will complete a visit at the study doctor’s office, which will take about 4 hours. During the visit, the study doctor will conduct genetic and vision tests.
This is an observational study for children ages 5 and older and adults with Stargardt disease type 1 or cone rod dystrophy to evaluate potential eligibility to participate in a future clinical trial of ACDN-01. People taking part will not be given a study drug, but they will be prioritized for consideration to participate in future clinical studies of ACDN-01.
For more details, please contact .
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 surgical procedure to inject the study drug to the back of 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 injection of ACDN-01 to the back of one eye and then regular monitoring for two years after treatment. For the final three years, there will be annual visits.
No. If travel or overnight stays are required for study visits, the study sponsor will cover those costs for eligible participants and their care partners. At this time, you must live in the U.S., Canada or Mexico to participate in the STELLAR study or the STARPATH study.
Prescreening studies help researchers identify people who may be a good match for future clinical trials. Prescreening studies do not test new treatments or therapies, instead they help prioritize potential participants for future treatment studies.
To provide the opportunity for genetic testing and high-quality imaging that will be used to identify potential pediatric and adult participants for future ACDN-01 clinical trials.
Participation in the STARPATH study does not obligate you or your child to participate in an ACDN-01 clinical trial in the future.
Your participation will mean that you or your child may be prioritized for consideration in future clinical studies. Further screening procedures will confirm eligibility for any future studies once enrolling.
No. If travel or overnight stays are required for study visits, the study sponsor will cover those costs for eligible participants and their care partners. At this time, you must live in the U.S., Canada or Mexico to participate in the STELLAR study or the STARPATH study.