A first-in-human clinical study of a new SP-B and SP-C enriched synthetic surfactant (CHF5633) in preterm babies with respiratory distress syndrome: two-year outcomes

Abstract

Objective

To assess at 24 months corrected age (CA) the neurological, respiratory, and general health status of children born prematurely from 27⁺⁰ to 33⁺⁶ weeks’ gestation who were treated in a first-in-human study with a new fully synthetic surfactant (CHF5633) enriched with SP-B and SP-C proteins.

Outcome measures

Children were assessed using Bayley Scales of Infant Development (BSID), with a score below normal defined as BSID-II Mental Development Index score <70, or BSID-III cognitive composite score <85. In addition, a health status questionnaire was used to check for functional disability including respiratory problems and related treatments, sensory and neurodevelopment assessments, communication skills as well as the number of hospitalizations.

Results

35 of 39 survivors had a neurodevelopmental assessment, 24 infants being evaluated by Bayley’s Scales and 11 by health status questionnaires only. 23 children had scores within normal limits and one had BSID-III <85. The remaining 11 were judged clinically to have normal development. Health status questionnaires detected only issues that would normally be expected in preterm-born children.

Conclusions

This assessment offers reassurance that treatment with CHF5633 surfactant was not associated with adverse neurodevelopmental, respiratory, or health outcomes by two years corrected age.

Keywords:

• Synthetic surfactant
• respiratory distress syndrome
• first-in-human study
• 24-month developmental assessment

Acknowledgments

The authors would like to thank the following Investigators for their help in collecting 24-month data or previously recruited study patients: Samir Gupta (University of Durham & North Tees University Hospital, Stockton-on-Tees, UK), Suzanne Schmidtke (Asklepios Klinik Barmbek, Abteilung Neonatologie, Hamburg, Germany), Monika Wolf (Sektion Neonatologie und Pädiatrische Intensivmedizin, Universitätsklinikum Eppendorf, Hamburg), Alison Walker (Neonatal Unit, Royal Maternity Hospital, Belfast). The authors would also like to thank the patients and their families for their participation in the study as well as Chiesi Farmaceutici S.p.A. (Parma, Italy) for the support in conducting this study, and Pharm-Olam International (The Brackens, Ascot, UK) for the monitoring activities, data collection and management, and statistical analysis.

Disclosure statement

Laura Fabbri, Debora Santoro, Annalisa Piccinno, Dorothea Del Buono, and Guido Varoli are full employees of Chiesi Farmaceutici S.p.A., sponsor of the study. D. Sweet has previously acted in an advisory capacity for Chiesi Pharmaceuticals UK. C. P. Speer is a consultant for Chiesi Farmaceutici S.p.A. (Italy). M. Turner serves as a consultant to Chiesi Farmaceutici S.p.A. (Italy) with respect to the development of CHF5633 on behalf of the University of Liverpool without deriving any personal benefit from this consultancy. The remaining authors have no conflict of interest to declare.

Additional information

Funding

The present work was financially supported by Chiesi Farmaceutici S.p.A. (Italy).