A Phase I, Multicenter, Open-label, Single-dose, Dose Ranging Study to Assess the Safety and Tolerability of SB-318, a Recombinant Adeno-associated Virus (rAAV)2/6-based Gene Transfer in Subjects With Mucopolysaccharidosis I (MPS I)

Trial Profile

A Phase I, Multicenter, Open-label, Single-dose, Dose Ranging Study to Assess the Safety and Tolerability of SB-318, a Recombinant Adeno-associated Virus (rAAV)2/6-based Gene Transfer in Subjects With Mucopolysaccharidosis I (MPS I)

Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 23 Aug 2017

At a glance

  • Drugs SB 318 (Primary)
  • Indications Mucopolysaccharidosis I
  • Focus Adverse reactions
  • Sponsors Sangamo Therapeutics
  • Most Recent Events

    • 26 Apr 2017 Planned number of patients changed from 12 to 9.
    • 26 Apr 2017 Planned initiation date changed from 1 Jan 2017 to 1 May 2017.
    • 27 Feb 2017 Status changed from not yet recruiting to recruiting, as reported in a Sangamo Therapeutics media release.
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