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A Phase I / 2, Multicenter, Open-label, Single-dose, Dose-ranging Study to Assess the Safety and Tolerability of SB-913, a rAAV2/6-based Gene Transfer in Subjects With Mucopolysaccharidosis II (MPS II)

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Trial Profile

A Phase I / 2, Multicenter, Open-label, Single-dose, Dose-ranging Study to Assess the Safety and Tolerability of SB-913, a rAAV2/6-based Gene Transfer in Subjects With Mucopolysaccharidosis II (MPS II)

Status: Discontinued
Phase of Trial: Phase I/II

Latest Information Update: 13 Oct 2022

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At a glance

  • Drugs SB 913 (Primary)
  • Indications Mucopolysaccharidosis II
  • Focus Adverse reactions
  • Acronyms CHAMPIONS
  • Sponsors Sangamo Therapeutics

Most Recent Events

  • 03 Jun 2021 Status changed from active, no longer recruiting to discontinued as all nine subjects dosed in the study have rolled over to the Long-Term Follow-up Study IVPRP-LT01 [NCT04628871].
  • 01 Mar 2021 Planned End Date changed from 1 Feb 2022 to 1 Jun 2022.
  • 01 Mar 2021 Planned primary completion date changed from 1 Feb 2021 to 1 Jun 2021.

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