Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome (NCT03300453) | Clinical Trial Compass
CompletedPhase 1/2
Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome
France4 participantsStarted 2013-09-17
Plain-language 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.
Who can participate
Age range18 Months – 60 Months
SexALL
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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®).
What they're measuring
1
Number of Participants With Treatment-Related (Serious) Adverse Events as assessed by continuous evaluation of change from baseline