A trial of antigen-specific immune tolerance induction in mucopolysaccharidosis I (MPS I) patients initiating enzyme replacement therapy with Aldurazyme® (laronidase)
- Conditions
- Mucopolysaccharidosis IMedDRA version: 18.0Level: PTClassification code 10056886Term: Mucopolysaccharidosis ISystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2007-001163-30-Outside-EU/EEA
- Lead Sponsor
- Genzyme, a Sanofi Company
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- A
- Sex
- All
- Target Recruitment
- 12
• 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).
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method