Phase I/II safety, tolerability and initial efficacy study of adeno-associated viral vector serotype 9 containing human sulfamidase gene after intracerebroventricular administration to patients with MPSIIIA. Estudio de fase I/II para evaluar la seguridad, tolerabilidad y eficacia inicial del vector viral adenoasociado de serotipo 9 conteniendo el gen de la sulfamidasa humana tras su administración por vía intracerebroventricular a pacientes con MPSIIIA.

Trial Profile

Phase I/II safety, tolerability and initial efficacy study of adeno-associated viral vector serotype 9 containing human sulfamidase gene after intracerebroventricular administration to patients with MPSIIIA. Estudio de fase I/II para evaluar la seguridad, tolerabilidad y eficacia inicial del vector viral adenoasociado de serotipo 9 conteniendo el gen de la sulfamidasa humana tras su administración por vía intracerebroventricular a pacientes con MPSIIIA.

Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 14 Jun 2016

At a glance

  • Drugs EGT 101 (Primary)
  • Indications Mucopolysaccharidosis III
  • Focus Adverse reactions; First in man; Pharmacodynamics; Therapeutic Use
  • Sponsors Esteve
  • Most Recent Events

    • 10 Jun 2016 Status changed from planning to recruiting.
    • 01 Mar 2016 New trial record
    • 25 Feb 2016 According to ESTEVE and UAB media release, company is planning to initiate this trial by the end of 2016. EGT-101 received orphan drug designation by the FDA and EMA in 2011.
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