Table 2 Summary of ocular AEs

From: Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10: a phase 1b/2 trial

 

Sepofarsen 160 μg/80 μg (n = 6)a

Sepofarsen 320 μg/160 μg (n = 5)b

Sepofarsen combined (n = 11)c

Relationship to study drug

Severity

Number of patients (%)

Ocular AE

5 (83.3)

5 (100)

10 (90.9)

  

Treated eye

     

 Conjunctival hemorrhage

4 (66.7)

4 (80.0)

8 (72.7)

Not related

Mild

 Cataractd

3 (50.0)

5 (100)

8 (72.7)

Probable to

Mild to

 SAEs

2 (33.0)

4 (80.0)

6 (54.5)

definite

severe

 Conjunctival hyperemia

1 (16.7)

2 (40.0)

3 (27.3)

Not related

Mild

 Dry eye

1 (16.7)

0

1 (9.1)

Not related

Mild

 Retinal degeneratione

0

2 (40.0)

2 (18.2)

Probable

Mild to moderate

 Anterior chamber cells

0

1 (20.0)

1 (9.1)

Not related

Mild

 Conjunctival edema

0

1 (20.0)

1 (9.1)

Not related

Mild

 Corneal lesionf

0

1 (20.0)

1 (9.1)

Not related

Mild

 Cystoid macular edema

0

1 (20.0)

1 (9.1)

Probable

Mild

 Diplopia

0

1 (20.0)

1 (9.1)

Definite

Moderate

 Eyelid cyst

0

1 (20.0)

1 (9.1)

Not related

Mild

 Eyelid edema

0

1 (20.0)

1 (9.1)

Not related

Mild

 Hypotony of eye

0

1 (20.0)

1 (9.1)

Not related

Mild

 Injection site hemorrhage

0

1 (20.0)

1 (9.1)

Not related

Mild

 Lens disorderg

0

1 (20.0)

1 (9.1)

Possible

Mild

 Metamorphopsia

0

1 (20.0)

1 (9.1)

Possible

Mild

 Post-procedural complicationh

0

1 (20.0)

1 (9.1)

Not related

Mild

 Posterior capsule opacification

0

1 (20.0)

1 (9.1)

Not related

Mild

 Retinal cysti

0

1 (20.0)

1 (9.1)

Possible

Mild

 Vitreal cells

0

1 (20.0)

1 (9.1)

Not related

Mild

 Vitreous opacities

0

1 (20.0)

1 (9.1)

Possible

Mild

Untreated eye

 Eye pruritus

0

1 (20.0)

1 (9.1)

Not related

Mild

  1. aAdults n = 3, children n = 3
  2. bAdults n = 3, children n = 2
  3. cAdults n = 6, children n = 5
  4. dCataract events comprised events coded as the following (number in 160-µg/80-µg dose group, number in 320-µg/160-µg dose group): cataracts (3, 4), cataract subcapsular (1, 0), cataract cortical (1, 0) and lenticular opacities (may represent an early stage of cataract development; 1, 3).
  5. eRetinal degeneration comprised retinal thinning and retinal atrophy on optical coherence tomography imaging events.
  6. fCorneal lesion corresponds to punctiform lesions in the cornea after lens replacement.
  7. gFewer than ten small round vacuoles in lens nucleus
  8. hAir bubble in superior periphery of vitreous, after injection
  9. iRetinal cyst event refers to the event of parafoveal intraretinal fluid cysts in the text (mild cystoid macular edema).
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