An Open-label, Single Arm, Monocentric, Phase I/II Clinical Study of Intracerebral Administration of Adeno-associated Viral Vector Serotype 10 Carrying the Human SGSH and SUMF1 cDNAs for the Treatment of Sanfilippo Type A Syndrome.

Trial Profile

An Open-label, Single Arm, Monocentric, Phase I/II Clinical Study of Intracerebral Administration of Adeno-associated Viral Vector Serotype 10 Carrying the Human SGSH and SUMF1 cDNAs for the Treatment of Sanfilippo Type A Syndrome.

Completed
Phase of Trial: Phase I/II

Latest Information Update: 10 Feb 2014

At a glance

  • Drugs SAF 301 (Primary)
  • Indications Mucopolysaccharidosis III
  • Focus Adverse reactions
  • Sponsors LYSOGENE
  • Most Recent Events

    • 10 Feb 2014 New trial record
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