Dataset Information

RhIGF-1/rhIGFBP-3 in Preterm Infants: A Phase 2 Randomized Controlled Trial.

ABSTRACT:

Objective

To investigate recombinant human insulin-like growth factor 1 complexed with its binding protein (rhIGF-1/rhIGFBP-3) for the prevention of retinopathy of prematurity (ROP) and other complications of prematurity among extremely preterm infants.

Study design

This phase 2 trial was conducted from September 2014 to March 2016. Infants born at a gestational age of 23^0/7 weeks to 27^6/7 weeks were randomly allocated to rhIGF-1/rhIGFBP-3 (250 µg/kg/ 24 hours, continuous intravenous infusion from <24 hours of birth to postmenstrual age 29^6/7 weeks) or standard neonatal care, with follow-up to a postmenstrual age of 40^4/7 weeks. Target exposure was ≥70% IGF-1 measurements within 28-109 µg/L and ≥70% intended therapy duration. The primary endpoint was maximum severity of ROP. Secondary endpoints included time to discharge from neonatal care, bronchopulmonary dysplasia, intraventricular hemorrhage, and growth measures.

Results

Overall, 61 infants were allocated to rhIGF-1/rhIGFBP-3, 60 to standard care (full analysis set); 24 of 61 treated infants achieved target exposure (evaluable set). rhIGF-1/rhIGFBP-3 did not decrease ROP severity or ROP occurrence. There was, however, a 53% decrease in severe bronchopulmonary dysplasia in the full analysis set (21.3% treated vs 44.9% standard care), and an 89% decrease in the evaluable set (4.8% vs 44.9%; P = .04 and P = .02, respectively) for severity distribution between groups. There was also a nonsignificant trend toward decrease in grades 3-4 intraventricular hemorrhage in the full analysis set (13.1% vs 23.3%) and in the evaluable set (8.3% vs 23.3%). Fatal serious adverse events were reported in 19.7% of treated infants (12/61) and 11.7% of control infants (7/60). No effect was observed on time to discharge from neonatal care/growth measures.

Conclusions

rhIGF-1/rhIGFBP-3 did not affect development of ROP, but decreased the occurrence of severe bronchopulmonary dysplasia, with a nonsignificant decrease in grades 3-4 intraventricular hemorrhage.

Trial registration

ClinicalTrials.gov: NCT01096784.

SUBMITTER: Ley D

PROVIDER: S-EPMC6389415 | biostudies-literature | 2019 Mar

REPOSITORIES: biostudies-literature

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rhIGF-1/rhIGFBP-3 in Preterm Infants: A Phase 2 Randomized Controlled Trial.

Ley David D Hallberg Boubou B Hansen-Pupp Ingrid I Dani Carlo C Ramenghi Luca A LA Marlow Neil N Beardsall Kathryn K Bhatti Faizah F Dunger David D Higginson Jason D JD Mahaveer Ajit A Mezu-Ndubuisi Olachi J OJ Reynolds Peter P Giannantonio Carmen C van Weissenbruch Mirjam M Barton Norman N Tocoian Adina A Hamdani Mohamed M Jochim Emily E Mangili Alexandra A Chung Jou-Ku JK Turner Mark A MA Smith Lois E H LEH Hellström Ann A

The Journal of pediatrics 20181122

<h4>Objective</h4>To investigate recombinant human insulin-like growth factor 1 complexed with its binding protein (rhIGF-1/rhIGFBP-3) for the prevention of retinopathy of prematurity (ROP) and other complications of prematurity among extremely preterm infants.<h4>Study design</h4>This phase 2 trial was conducted from September 2014 to March 2016. Infants born at a gestational age of 23<sup>0/7</sup> weeks to 27<sup>6/7</sup> weeks were randomly allocated to rhIGF-1/rhIGFBP-3 (250 µg/kg/ 24 hour ...[more]

PMID: 30471715

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