Phase I/II safety, tolerability and initial efficacy study of adeno-associated viral vector serotype 9 containing human sulfamidase gene after intracerebroventricular administration to patients with MPSIIIA.

aboratorios del Dr. Esteve, S.A.0 个研究点开始时间: 2016年2月3日最近更新: 2016年6月13日

适应症

试验速览

暂无简介。

•1.- Male and female patients aged 2 years or older.
•2.- Patients with confirmed MPSIIIA (by genotype), with underlying missense mutation at least in one of the alleles for the disease and documented deficiency in sulfamidase enzyme activity of less than or equal to 10%.
•3.- Onset of clinical manifestations related to MPSIIIA during the first 6 years of life.
•4.- Patients with an adaptive behaviour score between 40 and 90 as evaluated by the Vineland Adaptive Behaviour Scale (Vineland-II).
•5.- Patients not dependent on a wheelchair.
•6.- Patients without severe sensory deficit (blindness, deafness that requires headset).
•7.- Patients with stable symptomatic treatment (depending on weight) within the last 3 months, with no anticipated changes in medication regimen.
•8.- Patients with no contraindication for surgical procedure and/or anaesthesia. Patients taking non-steroidal anti-inflammatory drugs (NSAIDs) should discontinue their use.
•9.- Patients medically stable to accommodate the protocol requirements, including travelling and assessments.
•10.- Signed informed consent.
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 6
•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

•1.- Patient deterioration that may compromise the interpretation of the study results.
•2.- Patients with neutralising antibodies (NAb) against AAV9 in cerebrospinal fluid.
•3.- Epilepsy resistant to treatment.
•4.- Patients with significant co-morbid conditions.
•5.- Any contraindication for anaesthesia and product administration procedure, including major risk factors for haemorrhage.
•6.- Any vaccination 30 days before investigational product administration.
•7.- Patients who have received any medication with the objective of modifying the natural course of the disease, i.e. gene transfer agents or enzyme replacement therapy.

发起方

aboratorios del Dr. Esteve, S.A.