IMPE2023 Poster Presentations Growth and Syndromes (15 abstracts)
A Randomized Controlled Trial of Vosoritide in in Infants and Toddlers with Achondroplasia
Lynda E. Polgreen 1 , Ravi Savarirayan 2 , William W. Wilcox 3 , Paul Harmatz 4 , John Phillips , III 5 , Louise Tofts 6 , Keiichi Ozono 7 , Paul Arundel 8 , Melita Irving 9 , Carlos A. Bacino 10 , Donald Basel 11 , Michael B. Bober 12 , Joel Charrow 13 , Hiroshi Mochizuki 14 , Yumiko Kotani 15 , Howard M. Saal 16 , George Jeha 17 , Lynn Han 17 , Elena Fisheleva 18 , Alice Huntsman-Labed 18 & Jonathan Day 18
1Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center, Torrance, USA. 2Murdoch Children’s Research Institute, Royal Children’s Hospital, and University of Melbourne, Parkville, Australia. 3Emory University, Atlanta, USA. 4UCSF Benioff Children’s Hospital Oakland, Oakland, USA. 5Vanderbilt University Medical Center, Nashville, USA. 6Kids Rehab, The Children's Hospital at Westmead, Westmead, Australia. 7Osaka University Hospital, Osaka, Japan. 8Sheffield Children's NHS Foundation Trust, Sheffield Children's Hospital, Sheffield, United Kingdom. 9Guy’s and St. Thomas’ NHS Foundation Trust, Evelina Children's Hospital, London, United Kingdom. 10Baylor College of Medicine, Houston, USA. 11Medical College of Wisconsin, Milwaukee, USA. 12Nemours /Alfred I. du Pont Hospital for Children, Wilmington, USA. 13Ann and Robert H. Lurie Children’s Hospital of Chicago, Chicago, USA. 14Saitama Children’s Hospital, Saitama, Japan. 15Tokushima University Hospital, Tokushima, Japan. 16Cincinnati Children’s Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, USA. 17BioMarin Pharmaceutical Inc, Novato, USA. 18BioMarin (U.K.) Limited, London, United Kingdom
Background: Vosoritide increases annualized growth velocity (AGV) in children with achondroplasia aged 5 to 18 years. This global, phase 2, randomized, double-blind, placebo-controlled study evaluated the safety and efficacy of vosoritide on growth in children with achondroplasia aged 3 months to <5 years.
Methods: This study compared once-daily subcutaneous administration of vosoritide, at doses of 15 or 30 mg/kg of body weight, with placebo. Eligible patients had participated, for up to 6 months, in an observational growth study to calculate their baseline AGV. The primary objective was to evaluate the safety and tolerability of vosoritide in children with achondroplasia. The primary efficacy evaluation was the change from baseline in height Z-score vs placebo at week 52 using an ANCOVA model. Secondary efficacy analyses included change from baseline in AGV and upper-to-lower body segment ratio vs placebo at Week 52 using an ANCOVA model.
Results: A total of 75 patients were enrolled in three sequential cohorts based on age at screening, with 11 sentinel subjects who received vosoritide to establish PK and safety. A further 32 were randomized to receive vosoritide and 32 to receive placebo. A total of 73 patients completed the 52-week trial. All patients reported at least one adverse event. Four serious adverse events occurred with vosoritide and 8 with placebo, none were treatment-related. Two participants discontinued, one on vosoritide with pre-existing respiratory morbidity who had a fatal respiratory arrest and one on placebo who withdrew consent. In the full analysis population, vosoritide (n=43) compared to placebo (n=32) increased height Z-score by 0.30 SD (95% CI 0.07, 0.54), increased AGV by 0.92cm/year (95% CI 0.24, 1.59), and did not worsen upper-to-lower body segment ratio which changed by -0.06 (95% CI -0.15, 0.03). The increases in height Z-score and AGV were consistent across all age cohorts.
Conclusions: Daily, subcutaneous administration of vosoritide to young children with achondroplasia was safe and resulted in increases in height Z-score and AGV.
(Funded by BioMarin; ClinicalTrials.gov NCT03583697)
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