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A phase I/II single center, open-label study to evaluate the safety, tolerability, pharmacodynamics and preliminary efficacy of intracisternal RGX-111 gene therapy in subjects with severe mucopolysaccharidosis type I

Trial Profile

A phase I/II single center, open-label study to evaluate the safety, tolerability, pharmacodynamics and preliminary efficacy of intracisternal RGX-111 gene therapy in subjects with severe mucopolysaccharidosis type I

Status: Not yet recruiting
Phase of Trial: Phase I/II

Latest Information Update: 15 Nov 2018

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At a glance

  • Drugs RGX 111 (Primary)
  • Indications Mucopolysaccharidosis I
  • Focus Adverse reactions
  • Sponsors REGENXBIO
  • Most Recent Events

    • 15 Nov 2018 New trial record
    • 07 Nov 2018 According to a REGENXBIO media release, the company has ubmitted an application to the Brazilian Health Surveillance Agency (ANVISA) to proceed with a Phase I clinical trial evaluating RGX-111 for treatment of MPS I in patients under the age of 3 which it expects will allow the company to activate an initial site in Brazil during the first half of 2019. Dosing of first subject is expected in the first half of 2019.
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