Last reviewed · How we verify
NCT05809635
Study of BEST1 Vitelliform Macular Dystrophy
trial testing Natural History Study in Best Vitelliform Macular Dystrophy in 52 participants. Currently enrolling.
31 May 2026
Quick facts
| Lead sponsor | Columbia University |
|---|---|
| Status | Recruiting now |
| Study type | OBSERVATIONAL |
| Enrollment | 52 |
| Start date | 30 March 2021 |
| Primary completion | 31 May 2026 |
| Estimated completion | 31 May 2026 |
| Sites | 3 locations across France, United States, Germany |
Drugs / interventions tested
- Natural History Study
Conditions studied
- Best Vitelliform Macular Dystrophy — all drugs for Best Vitelliform Macular Dystrophy →
- Retinitis Pigmentosa — all drugs for Retinitis Pigmentosa →
Sponsor
Columbia University
Who can join
Eligibility, any sex, with Best Vitelliform Macular Dystrophy or Retinitis Pigmentosa. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
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. The goals of this natural history study are to: 1. Report the natural history of retinal degeneration in participants with a clinical diagnosis of VMD with molecular confirmation of a pathogenic BEST1 mutation(s). 2. Identify sensitive structural and functional outcome measures to use for future multicenter clinical trials for the treatment of BESTROPHIN 1 VMD. 3. Compare progression of the identified structural and functional measures between the two eyes to judge the suitability of the second untreated eye as a control for a future clinical trial involving unilateral treatment 4. Identify well-defined patient populations for future clinical trials of investigative treatments for BEST1 VMD.
Publications & conference data
1 peer-reviewed publication reference this trial (live from Europe PMC):
-
Review of the clinical electrooculogram - Part 2: the bestrophinopathies and modified protocols.
Padhy SK, Habjan MŠ, Constable PA. · · 2026 · PMID 41795754 · DOI 10.1007/s10633-026-10093-y
Verify or expand the search:
- PubMed search for NCT05809635
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
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Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT05809635 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Columbia University
- Last refreshed: 30 July 2025
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT05809635.
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