Design of a Phase 2 Randomized Controlled Trial and Long-Term Extension Study of Recombinant Human IGF-1/IGFBP-3 for Prevention of Comorbidities of Extreme Prematurity

Abstract

Introduction: Insulin-like growth factor 1 (IGF-1) is involved in fetal development of multiple organ systems, including the eye, lung, and brain. Levels of serum IGF-1 fall after preterm birth and are notably lower in preterm infants than in fetuses of corresponding gestational age (GA). Supplementation to fetal IGF-1 levels with recombinant human (rh) IGF-1/rhIGFBP-3 is being investigated for prevention of retinopathy of prematurity (ROP) and other comorbidities of prematurity. A phase 2 study was initiated to assess dosing, efficacy and safety of rhIGF-1/rhIGFBP-3 administered as a continuous intravenous (IV) infusion in extremely preterm infants. The early sections (A–C) of this study demonstrated safety of rhIGF-1/rhIGFBP-3 administration in infants <28 weeks GA. We report the design of the final section (D) of this study and the associated long-term extension study (PEDAL).