Table 1 Main inclusion criteria for VN phase1/2 and 3 clinical trials [7, 15].

From: Voretigene neparvovec for inherited retinal dystrophy due to RPE65 mutations: a scoping review of eligibility and treatment challenges from clinical trials to real practice

Clinical feature

Inclusion criteria

Age

≥8 years in phase 1/2, ≥3 years in phase 3

VA

Patient should show one of the following:

• ≤20/160 in the eye candidate to treatment

• worse than 20/60 for both eyes

Viable cellsa

Defined as either:

• an area of retina within the posterior pole of >100 μm thickness measured by OCT scan

• ≥3-disc areas within the posterior pole of retina without atrophy or pigmentary degeneration

• a remaining VF within 30° of fixation as measured by a III4e isopter or equivalent meridian as measured by a III4e isopter or equivalent (both eyes)

  1. VA visual acuity, VF visual field.
  2. aEvaluation of sufficient viable cells was performed by non-invasive tests, such as optical coherence tomography (OCT) and/or ophthalmoscopy and/ or visual field.
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