The phase I/II study to evaluate the effect of SB-913, an investigational in vivo genome editing therapy, in patients with mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome.

Trial Profile

The phase I/II study to evaluate the effect of SB-913, an investigational in vivo genome editing therapy, in patients with mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome.

Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 22 Nov 2017

At a glance

  • Drugs SB 913 (Primary)
  • Indications Mucopolysaccharidosis II
  • Focus Adverse reactions; Pharmacokinetics; Therapeutic Use
  • Acronyms CHAMPIONS study
  • Most Recent Events

    • 22 Nov 2017 New trial record
    • 15 Nov 2017 According to a Sangamo Therapeutics media release, Paul Harmatz, M.D., a pediatric gastroenterologist is a principal investigator of the study.
    • 15 Nov 2017 According to a Sangamo Therapeutics media release, first patients has been dosed in this trial.
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