Study of BEST1 Vitelliform Macular Dystrophy
Recruiting
Observational Study
Best Vitelliform Macular Dystrophy
Retinitis Pigmentosa
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Sex
- Any
- Study type
- Observational
- Participants needed
- 52 (estimated)
- Sponsor
- Columbia University · Other
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About This Trial
The purpose of this study is to establish the natural history of of participants with BESTROPHIN 1 Vitelliform Macular Dystrophy.
The blinding disorder Best Vitelliform Macular Dystrophy (VMD) is caused by any one of more than 250 different mutations in the BEST1 gene.
As new treatments are developed, a clear understanding of the natural history of disease progression of BEST1 VMD is necessary. …
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* Ability to provide informed consent
* Diagnosis of BEST1-associated VMD by study physician, who are trained retinal specialists in the university clinic Must be able to commit to 4 follow-up study visits (3 years)
Exclusion Criteria:
* Systemic condition that prevents the pa…
Contacts