An observational study in Leber Congenital Amaurosis, sponsored by MeiraGTx UK II Ltd. Completed at 2 sites in 2 countries. Open to participants aged 3 Years and older. Per ClinicalTrials.gov, last updated 2026-09-21.
Sponsored by MeiraGTx UK II Ltd · Observational
MGT005 is a natural history study to collect longitudinal prospective data from patients with Leber Congenital Amaurosis associated with defects in RPE65.
Leber Congenital Amaurosis (LCA) is a diagnosis for a group of severe, autosomal recessively inherited rod - cone dystrophies that typically result in complete visual loss in the third or fourth decade of life. One form, LCA2, is caused by a mutation in the gene encoding RPE56, an RPE-specific 65-kDa isomerase. Non-functional RPE65 results in photoreceptor cells that are unable to respond to light resulting in these patients being visually impaired.
In preparation for human clinical trials, a detailed prospective phenotypic study will be undertaken to investigate the natural history of RPE65-LCA. Such a study will help identify suitable patients for therapeutic intervention. Furthermore through greater phenotyping an optimal window for intervention and specific parameters to help quantify effect and identify clinical end points may have been ascertained .
32 studies on the registry are indexed under Leber Congenital Amaurosis; 4 are open to participants now.
Browse Leber Congenital Amaurosis studies →MeiraGTx UK II Ltd is the lead sponsor of 10 studies on the registry; none are open to participants now.
Of its 5 completed or terminated interventional studies of FDA-regulated products, 4 (80%) have results posted.
Counted across the registry records on this site, refreshed daily.
Patients with RPE65-LCA condition
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Analysis of retinal structure and function
Retinal structure will be analysed using Adaptive optics and SD-OCT and Fundal autofluorescence. This will be correlated with assessment of visual acuity, psychophysical visual assessment, visual mobility, retinal sensitivity and visual fields
Time frame: 6 years
Quality of Life Questionnaires
Assessment of Visual impairment using appropriate, validated questionnaires
Time frame: 6 years
Retinal Sensitivity
To be assessed in Microperimetry
Time frame: 6 years
Retinal Structural analysis
Retinal Structure analysis with Adaptive Optics
Time frame: 6 years
Fundal Autofluorescence
Presence or Absence
Time frame: 6 years
Assessment of Visual Fields
Assessment of Visual Fields with analysis of hill of vision
Time frame: 6 years
Plan to share: No
This study is completed, as verified in Sep 2026. You cannot join it, but the record below documents what was studied.
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MeiraGTx UK II Ltd