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Clinical trial

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.

NCT01474343 is registered in ClinicalTrials.gov with LYSOGENE as lead sponsor.

NCT NCT01474343
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Registry record

As filed in ClinicalTrials.gov.

Title
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.
Lead sponsor
LYSOGENE
Phase
Phase 1/2
Overall status
Completed
Start date
August 31, 2011
Completion date
May 31, 2013
Enrollment
4 (actual)
Sites
2

Conditions

As the registry lists them; a condition with its own page is linked.

mucopolysaccharidosis type iii a, sanfilippo disease type a