Sanfilippo Syndrome B Clinical Trial
Official title:
Protocol AMT110-CD-001: A Phase I/II, Open-label, Study of Intracerebral Administration of Adeno-associated Viral Vector Containing the Human Alpha-N-acetylglucosaminidase cDNA in Children With Sanfilippo Type B Syndrome
This is an open-label, phase I/II study of intra-cerebral administration of adenovirus-associated viral vector containing the human NAGLU cDNA to children suffering from Sanfilippo type B syndrome.
This is an open-label, phase I/II study of intra-cerebral administration of
adenovirus-associated viral vector containing the human NAGLU cDNA to children suffering from
Sanfilippo type B syndrome.
Four patients, 18 months up to the 5th birthday, have been included.
The inclusion period will be 8 to 12 months. The duration of follow-up for each patient is 1
year post-surgery. The duration of the first extension phase is 18 months. The duration of
the second extension phase is 36 months. Therefore, the maximum time of the follow-up will be
66 months
;