A Phase 1 interventional study of QR-504a in Fuchs Endothelial Corneal Dystrophy, FECD3 and Corneal Diseases, sponsored by ProQR Therapeutics. Withdrawn at 1 site in United Kingdom. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2022-05-11.
Sponsored by ProQR Therapeutics · Phase 1, Interventional, and Treatment
PQ-504a-001 (Fuchs Focus) is an open-label, single-dose, exploratory study to evaluate safety, tolerability, and corneal endothelium molecular biomarker(s) in subjects with Fuchs Endothelial Corneal Dystrophy with Trinucleotide Repeat Expansion in the TCF4 gene (FECD3).
PQ-504a-001 (Fuchs Focus) is an open-label, intra-subject-controlled study in subjects with FECD3 scheduled for Descemet's Membrane Endothelial Keratoplasty (DMEK) in both eyes or with concurrent, preemptive lens replacement surgery (LRS/DMEK), if indicated as part of routine clinical practice. Each subject will go through the following two parts in this study:
92 studies on the registry are indexed under Fuchs' Endothelial Dystrophy; 28 are open to participants now.
Browse Fuchs' Endothelial Dystrophy studies →ProQR Therapeutics is the lead sponsor of 7 studies on the registry; none are open to participants now.
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Exclusion Criteria:
Open label single dose cohort: Dose level 1
Drug: QR-504a
Open label single dose cohort: Dose level 2
Drug: QR-504a
Intravitreal (IVT) injection
Incidence and severity of ocular and non-ocular adverse events (AEs)
Time frame: 20 months
Change from baseline in the isoform levels of MBNL1 and MBNL2 in corneal endothelium
Time frame: 20 months
Plan to share: No
Ran from Aug 2021 to primary completion in Apr 2022, 8 months.
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This study is withdrawn, as verified in May 2022. You cannot join it, but the record below documents what was studied.
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Fuchs' Endothelial Dystrophy →
ProQR Therapeutics