Study to Assess the Efficacy, Safety, and Tolerability of Intravitreal Aflibercept Compared to Laser Photocoagulation in Patients With Retinopathy of Prematurity (BUTTERFLEYE)

Regeneron Pharmaceuticals

Status and phase: Completed; Phase 3

Conditions: Retinopathy of Prematurity

Treatments: Procedure: laser photocoagulation; Drug: aflibercept

Study type: Interventional

Funder types: Industry

Identifiers

NCT04101721

2019-001764-29 (EudraCT Number)

VGFTe-ROP-1920

Details and patient eligibility

About

The primary objective of the study is to assess the efficacy of aflibercept compared to laser in patients diagnosed with retinopathy of prematurity (ROP). The secondary objectives of the study are to assess the need for a second treatment modality, to assess the recurrence of ROP in the study and to assess the safety and tolerability of aflibercept.

Enrollment: 127 patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Gestational age at birth ≤ 32 weeks or birth weight ≤1500 g

• Patients with treatment-naïve retinopathy of prematurity (ROP) classified according to the International Classification for ROP in at least one eye as:

  • Zone I Stage 1 plus, or 2 plus, or 3 non-plus or 3 plus, or
  • Zone II Stage 2 plus or 3 plus, or
  • Aggressive posterior retinopathy of prematurity (AP-ROP)

Key Exclusion Criteria:

• Known or suspected chromosomal abnormality, genetic disorder, or syndrome
• Previous exposure to any Intravitreal (IVT) or systemic anti-vascular endothelial growth factor (VEGF) agent, including maternal exposure during pregnancy and/or during breastfeeding
• Clinically significant neurological disease (eg, intraventricular hemorrhage grade 3 or higher, periventricular leukomalacia, congenital brain lesions significantly impairing optic nerve function, severe hydrocephalus with significantly increased intracranial pressure)
• Pediatric conditions rendering the infant ineligible for study intervention at baseline or for repeated blood draws as evaluated by a neonatal intensive care unit specialist and a study ophthalmologist
• Presence of active ocular infection within 5 days of the first treatment
• Advanced stages of ROP with partial or complete retinal detachment (ROP stage 4 and stage 5)
• ROP involving only Zone III

NOTE: Other protocol defined inclusion/exclusion criteria apply.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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127 participants in 2 patient groups

Aflibercept Group

Experimental group

Description:

Patients will receive a single intravitreal (IVT) injection per eligible eye at baseline.

Treatment:

Drug: aflibercept

Laser Group

Experimental group

Description:

Patients will undergo laser treatment in each eligible eye at baseline.

Treatment:

Procedure: laser photocoagulation