Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome

• Status: Completed
• Trial end date: 2019-11-27
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: UniQure Biopharma B.V.
• Collaborators: Institut Pasteur; Venn Life Sciences

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®).