75 Participants Needed

Observational Study for Stargardt Disease

(POLARIS Trial)

Recruiting at 19 trial locations
SS
S
Overseen BySpliceBio
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to observe the progression of Stargardt Disease Type 1 (STGD1), a genetic eye condition caused by specific mutations, over time. Researchers are not testing a new treatment; instead, they focus on understanding the disease's natural course. Suitable candidates for this study include individuals diagnosed with STGD1 who have experienced worsening symptoms in the past two years. Participants should also be able to undergo detailed eye imaging without complications. As an unphased trial, this study allows participants to contribute to a deeper understanding of STGD1, potentially aiding future research and treatment strategies.

Why are researchers excited about this trial?

Researchers are excited about this trial because it aims to gather crucial information on the progression of Stargardt Disease Type 1 (STGD1) caused by mutations in the ABCA4 gene. Unlike existing treatments that primarily focus on managing symptoms, this observational study seeks to better understand the disease's natural history and progression. By closely monitoring patients over time, the study could pave the way for future targeted therapies that address the root causes of the disease, potentially leading to more effective treatments.

Are You a Good Fit for This Trial?

Inclusion Criteria

I have signed a consent form.
I am between 12 and 65 years old.
My STGD1 is confirmed to be caused by specific genetic changes.
See 7 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Observational

Participants are observed to follow the progression of Stargardt Disease Type 1 (STGD1) and to enhance understanding of disease progression and structural and functional markers.

12 months

Follow-up

Participants are monitored for safety and effectiveness after the observational period

6 months

What Are the Treatments Tested in This Trial?

Find a Clinic Near You

Who Is Running the Clinical Trial?

Splice Bio

Lead Sponsor

Trials
1
Recruited
80+
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