A Phase I/II, 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)

Trial Profile

A Phase I/II, 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)

Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 09 Jun 2018

At a glance

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

    • 04 Jun 2018 According to a Sangamo Therapeutics media release, the Medicines and Healthcare Products Regulatory Agency (MHRA) of the United Kingdom granted the Clinical Trial Authorisation (CTA) for enrolment of subjects in the trial. The CTA allows for treatment of children as young as five years of age following a review of cumulative safety data from adult and adolescent cohorts. U.K. clinical trial sites are expected to initiate later this year.
    • 06 Feb 2018 According to a Sangamo Therapeutics media release, the company anticipate to report additional safety and initial efficacy data by mid-year.
    • 06 Feb 2018 According to a Sangamo Therapeutics media release, the second patient from this trial was also treated with a dose of 5.00E+12 vg/kg of SB-913 in mid Jan 2018.
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