title: A RANDOMIZED, PLACEBO-CONTROLLED STUDY OF INTRAVITREAL OCRIPLASMIN IN PEDIATRIC PATIENTS SCHEDULED FOR VITRECTOMY.
abstract: OBJECTIVE
  To evaluate safety and preliminary efficacy of 175 μg of intravitreal ocriplasmin in pediatric patients scheduled for vitrectomy.
  
  
  METHODS
  Based on a single-center, prospective, randomized, placebo-controlled, and double-masked Phase 2 study, 22 pediatric patients scheduled for vitrectomy were randomized 2:1 to 175-μg ocriplasmin injection or placebo. Treatment was administered midvitreous 30 minutes to 60 minutes before vitrectomy. Safety was assessed by reported adverse events (AEs), ophthalmologic examinations, fundus photography, and fluorescein angiography. The primary efficacy endpoint was the proportion of eyes with total macular posterior vitreous detachment before vitrectomy or after application of suction. Secondary endpoints included vitreous liquefaction assessment before vitrectomy and immediate postoperative retinal reattachment.
  
  
  RESULTS
  All patients experienced at least 1 AE. Most AEs were ocular and occurred in the study eye. Serious AEs were reported for 2 of 16 eyes (3.4%) in the ocriplasmin group and 2 of 8 eyes (11.1%) in the placebo group. One case of lens subluxation due to zonular disruption was observed in the ocriplasmin group. A clear efficacy signal was not observed.
  
  
  CONCLUSIONS
  Intravitreal injection of 175 μg of ocriplasmin was tolerated in pediatric patients before vitrectomy; however, the small sample size in this study precluded adequate efficacy comparisons. AEs reported were consistent with those anticipated in pediatric patients.
authors.affiliation: *Associated Retinal Consultants, Royal Oak, Michigan; †NightstaRx Ltd, London, United Kingdom (formerly of ThromboGenics NV, Leuven, Belgium).
authors.forename: Kimberly
authors.initials: K
authors.lastname: Drenser
authors.forename: Aniz
authors.initials: A
authors.lastname: Girach
authors.forename: Antonio
authors.initials: A
authors.lastname: Capone
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D004311:Double-Blind Method; D005260:Female; D005341:Fibrinolysin / Q000008:administration & dosage* / Q000009:adverse effects; D005343:Fibrinolytic Agents / Q000008:administration & dosage* / Q000009:adverse effects; D005451:Fluorescein Angiography; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D058449:Intravitreal Injections; D008297:Male; D010446:Peptide Fragments / Q000008:administration & dosage* / Q000009:adverse effects; D011446:Prospective Studies; D012164:Retinal Diseases / Q000503:physiopathology / Q000601:surgery*; D014792:Visual Acuity / Q000502:physiology; D014821:Vitrectomy; D014822:Vitreous Body / Q000187:drug effects; D020255:Vitreous Detachment / Q000209:etiology / Q000503:physiopathology
chemical_list: D005343:Fibrinolytic Agents; D010446:Peptide Fragments; C054561:microplasmin; D005341:Fibrinolysin