A Single Centre Study Investigating the Safety and Efficacy of an Immune Modulation Regimen in Mitigating the Alloimmune Response to Intravenous Laronidase in Infants With Severe Mucopolysaccharidosis type I (Hurler syndrome) Prior to Haematopoietic Stem Cell Transplantation - Immune Tolerance Induction with Methotrexate in Hurler Syndrome

Central Manchester University Hospitals NHS Foundation Trust0 个研究点目标入组 4 人开始时间: 2015年10月27日最近更新: 2019年6月30日

试验速览

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•Written informed consent of a legally authorised guardian(s)
•A diagnosis of MPS I as defined by deficient a-L-iduronidase activity in leukocytes or cultured fibroblasts, in a laboratory accepted by the investigators
•Clinical status consistent with severe Hurler phenotype
•If available, mutations consistent with a classical Hurler genotype e.g. homozygosity or compound heterozygosity for nonsense mutations (Q70X, W402X) in IDUA gene
•Age =3 months and =2.5 years at diagnosis
•Decision to offer HSCT with an appropriate donor after a short period (minimum 4 weeks) of ERT
•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 0
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range 0

•The patient has previously received intravenous laronidase
•The patient has undergone haematopoietic stem cell transplantation
•The patient has a known primary immune defect
•The patient has a known sensitivity or allergy to methotrexate
•The patient has known contraindications to receiving methotrexate such as pre-existing hepatic or renal dysfunction, or the patient is receiving concomitant medications that interact with methotrexate
•The patient has received any immunomodulatory or immunosuppressive medication within 90 days prior to enrolment
•The patient has been exposed to any other investigational product within 90 days prior to enrolment in the trial
•The patient has any clinically significant organic disease (with the exception of symptoms relating to MPS I), 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

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