A trial of antigen-specific immune tolerance induction in mucopolysaccharidosis I (MPS I) patients initiating enzyme replacement therapy with Aldurazyme® (laronidase)

Genzyme, a Sanofi Company0 个研究点目标入组 12 人开始时间: 2015年5月11日最近更新: 2015年7月10日

研究概览

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•Patient’s parent(s) or legal guardian(s) willing and able to provide written informed consent, after the nature of the trial has been explained.
•Patient’s parent(s) or legal guardian(s) allow their child’s participation and are willing and able to comply with the trial procedures.
•Less than 5 years of age at the time of enrollment.
•Clinical diagnosis of severe MPS I (Hurler syndrome).
•Confirmed presence of 2 nonsense mutations in the IDUA gene (eg, compound heterozygosity or homozygosity).
•Confirmed a-L-iduronidase deficiency with a fibroblast, plasma, serum, or leukocyte a-L-iduronidase enzyme activity level
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 7
•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

•Clinically significant organic disease including: cardiovascular, hepatic, pulmonary, neurologic, or renal disease, other serious intercurrent illness, or extenuating circumstances that, in the opinion of the Investigator, would preclude participation in
•the trial or potentially decrease survival.
•Prior treatment with Aldurazyme.
•Known severe hypersensitivity to any excipients of the delivery solution for Aldurazyme.
•Prior HSCT (regardless of outcome) or currently under consideration for such a transplant. If a family later decides to obtain a haematopoeitic stem cell transplant (HSCT), the patient will be discontinued from the trial.
•Receipt of an investigational product within the 30 days prior to enrollment.
•Prior treatment in any experimental protocol (eg, fibroblast injections) that might potentially induce antibodies to laronidase or might affect the interpretation of an antibody response to laronidase.
•Receipt of vaccination(s) within 1 month prior to enrollment, or unwilling to postpone vaccinations during the period of participation in the trial.
•Homozygous thiopurine methyltransferase (TPMT) deficiency, as determined by genotype (the presence of two known null alleles for TPMT) or phenotype (near to complete absence of TPMT enzyme activity).

发起方

Genzyme, a Sanofi Company