NCT03300453

Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome

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

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 4 (actual)
• Sponsor: UniQure Biopharma B.V. · Industry
• Sex: All
• Age: 18 Months – 60 Months
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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

Conditions

• Sanfilippo Syndrome B

Interventions

Type	Name	Description
DRUG	rAAV2/5-hNAGLU	one-time brain intraparenchymal gene therapy dose

Timeline

• Start date: 2013-09-17
• Primary completion: 2019-11-27
• Completion: 2019-11-27
• First posted: 2017-10-03
• Last updated: 2019-12-02

Locations

1 site across 1 country: France