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

Institut Pasteur0 个研究点开始时间: 2014年5月15日最近更新: 2017年10月2日

适应症

试验速览

暂无简介。

•Age: 18 months up to the 5th birthday;
•Onset of clinical manifestations related to 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
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 4
•F.1.2 Adults (18-64 years) no
•F.1.2.1 Number of subjects for this age range
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•Presence of brain atrophy on pre-inclusion MRI judged on a cortico-dural distance of more than 0.6 cm
•Any condition that would contraindicate general anaesthesia;
•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 Tacrolimus, Cellcept® and Solupred®/Solumedrol®.

发起方