Trial | NCT03300453  Report issue

Title

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

  • Phase

    Phase 1/Phase 2

  • Study Type

    Interventional

  • Status

    Completed No Results Posted

  • Study Participants

    4
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
Study Started
Sep 17
2013
Primary Completion
Nov 27
2019
Study Completion
Nov 27
2019
Last Update
Dec 02
2019

Drug rAAV2/5-hNAGLU

one-time brain intraparenchymal gene therapy dose

  • Other names: AAV5

rAAV2/5-hNAGLU Experimental

Each patient will receive 960 µL of vector suspension. The vector suspension will be deposited simultaneously at 16 sites, each deposit containing 2.4x 1011 vg (4x1012 vg in total).

Criteria 

Inclusion criteria:

Age: 18 months up to 60 months (5th birthday);
Onset of clinical manifestations related to mucopolysaccharidosis type IIIB (MPSIIIB);
NAGLU activity in peripheral blood cell and/or cultured fibroblast extracts of less than 10% of controls;
Patient affiliated to, or covered by a French social security regimen, or European patients with European Health Insurance Card;
Family understanding the procedure and the informed consent;
Signed informed consent by both parents or legal representative;
Vital laboratory parameters within normal range.

Exclusion Criteria:

Presence of brain atrophy on baseline MRI judged on a cortico-dural distance of more than 0.6 cm;
Any condition that would contraindicate general anesthesia;
Any other permanent medical condition not related to MPSIIIB that could contraindicate the study participation;
No independent walking (ability to walk without help);
Any medication aiming at modifying the natural course of MPSIIIB given during the 6 months before vector injection (sleep and mood regulators are accepted);
Any condition that would contraindicate treatment with Modigraf®, Cellcept® and prednisolone (Solupred® and Solumedrol®).
No Results Posted