Prescreening for Stargardt Disease

Recruiting at 7 trial locations

Overseen By Associate Director, Clinical Operations

Age: Any Age

Sex: Any

Trial Phase: Academic

Sponsor: Ascidian Therapeutics, Inc

Disqualifiers: Other gene mutations, Retinal disease, others

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

Compensation: Varies

Number of Visits: 1

50 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial seeks individuals who may be suitable for future research on a treatment for Stargardt disease, a genetic eye disorder affecting vision. Participants will undergo genetic and eye tests during at least one visit to determine eligibility for the upcoming ACDN-01 clinical trials. Those with Stargardt disease or cone-rod dystrophy due to changes in the ABCA4 gene (a specific genetic mutation) may qualify for this trial.

As an unphased trial, this study offers participants the chance to contribute to groundbreaking research that could lead to future treatments.

Do I need to stop my current medications for the trial?

The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Why are researchers excited about this trial?

Researchers are excited about this trial because it aims to identify individuals with Stargardt disease who may benefit from future ACDN-01 clinical trials. Stargardt disease, a form of inherited retinal degeneration, currently has limited treatment options focused on managing symptoms and slowing progression. This prescreening study is unique because it involves genetic and visual assessments to pinpoint eligible participants, potentially leading to more targeted and effective treatments in the future. By focusing on genetic markers, this approach could pave the way for personalized therapies that directly address the underlying causes of the disease, offering new hope for those affected.

Who Is on the Research Team?

Alia Rashid

Principal Investigator

Ascidian Therapeutics

Are You a Good Fit for This Trial?

Inclusion Criteria

I have a mutation in the ABCA4 gene.

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

Timeline for a Trial Participant

Prescreening

The prescreening study consists of genetic and visual assessments to determine research eligibility for ACDN-01 clinical trials

12 months

At least 1 onsite visit

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 years

What Are the Treatments Tested in This Trial?

Interventions

• Prescreening Assessments

How Is the Trial Designed?

Group I: Prescreening Group Experimental Treatment 1 Intervention

The prescreening study consists of genetic and visual assessments and will require at least 1 onsite visit. All clinical assessments performed are for the purpose of determining research eligibility for ACDN-01 clinical trials.

Who Is Running the Clinical Trial?

Ascidian Therapeutics, Inc

Lead Sponsor

Trials

2

Recruited

60+