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A Phase 1/2, Multi-Center, Open-Label, Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Exploratory Efficacy of Olipudase Alfa in Pediatric Patients Aged <18 Years With Acid Sphingomyelinase Deficiency

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Trial Profile

A Phase 1/2, Multi-Center, Open-Label, Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Exploratory Efficacy of Olipudase Alfa in Pediatric Patients Aged <18 Years With Acid Sphingomyelinase Deficiency

Status: Completed
Phase of Trial: Phase I/II

Latest Information Update: 02 Jan 2024

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At a glance

  • Drugs Olipudase alfa (Primary)
  • Indications Niemann-Pick disease type A
  • Focus Adverse reactions; Registrational
  • Acronyms ASCEND-Peds
  • Sponsors Sanofi; Sanofi Genzyme

Most Recent Events

  • 14 Nov 2023 Results assessing liver and lipid values at baseline and after 2years of olipudase alfa treatment in ongoing clinical trials and extensions (NCT02004691, NCT02292654 NCT02004704, NCT01722526) and Phase 1b trial presented at The Liver Meeting 2023: 74th Annual Meeting of the American Association for the Study of Liver Diseases
  • 15 Jun 2023 Results presented at the 28th Congress of the European Haematology Association
  • 31 Aug 2022 According to a Sanofi media release, the U.S. Food and Drug Administration (FDA) has approved XenpozymeTM for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients, based on data from ASCEND and ASCEND-Peds clinical trials.

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