Asfotase Alfa for Infants and Young Children with Hypophosphatasia: 7 Year Outcomes of a Single-Arm, Open-Label, Phase 2 Extension Trial
Articles Asfotase alfa for infants and young children with hypophosphatasia: 7 year outcomes of a single-arm, open-label, phase 2 extension trial Michael P Whyte, Jill H Simmons, Scott Moseley, Kenji P Fujita, Nicholas Bishop, Nada J Salman, John Taylor, Dawn Phillips, Mairead McGinn, William H McAlister Summary Background Our previous phase 2, open-label study of 11 infants and young children with life-threatening perinatal or Lancet Diabetes Endocrinol infantile hypophosphatasia showed 1 year safety and efficacy of asfotase alfa, an enzyme replacement therapy. We 2019; 7: 93–105 aimed to report the long-term outcomes over approximately 7 years of treatment. Published Online December 14, 2018 http://dx.doi.org/10.1016/ Methods We did a prespecified, end of study, 7 year follow-up of our single-arm, open-label, phase 2 trial in which S2213-8587(18)30307-3 children aged 3 years or younger with life-threatening perinatal or infantile hypophosphatasia were recruited from This online publication has been ten hospitals (six in the USA, two in the UK, one in Canada, and one in the United Arab Emirates). Patients received corrected. The corrected version asfotase alfa (1 mg/kg three times per week subcutaneously, adjusted to 3 mg/kg three times per week if required) for first appeared at thelancet. up to 7 years (primary treatment period plus extension phase) or until the product became commercially available; com/diabetes-endocrinology on January 22, 2019 dosage adjustments were made at each visit according to changes in the patient’s weight. The primary objectives of See Comment page 76 this extension study were to assess the long-term tolerability of asfotase alfa, defined as the number of patients with Center for Metabolic Bone one or more treatment-emergent adverse events, and skeletal manifestations associated with hypophosphatasia, Disease and Molecular evaluated using the Radiographic Global Impression of Change (RGI-C) scale (−3 indicating severe worsening, and Research, Shriners Hospital for +3 complete or near-complete healing).
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