Scientists track rare eye disease to prepare for future treatments

NCT ID NCT01736293

First seen Jun 25, 2026 · Last updated Jul 01, 2026 · Updated 3 times

Summary

This study follows 68 people aged 12 and older who have ABCA4 gene mutations, which can cause vision loss from diseases like Stargardt disease. Over 10 years, researchers will collect blood and skin samples and perform detailed eye exams to understand how the disease progresses. No treatment is given; the goal is to gather information for future clinical trials.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

What this could lead to

If successful, this study could help design future clinical trials for treatments targeting ABCA4-related eye diseases.

What could go wrong

This is an observational study with no treatment given, so it will not directly improve vision. It only tracks disease progression over time.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

ABCA4-related retinopathy cone-rod dystrophy retinal degeneration severe early-childhood-onset retinal dystrophy Stargardt disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • National Institutes of Health Clinical Center

    Bethesda, Maryland, 20892, United States