A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients
- Conditions
- Mucopolysaccharidosis IHurler's SyndromeHurler-Scheie SyndromeScheie's SyndromeMedDRA 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
- EUCTR2015-000585-61-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
- 6
•Have a documented diagnosis of MPS I confirmed by measurable signs and symptoms and deficient a-L-iduronidase activity (<10% of the lower limit of normal).
•For a patients receiving Aldurazyme therapy prior to study entry: Have available as baseline data the results of urinary GAG levels and IgG antibody titers collected prior to the patient's first Aldurazyme infusion.
•Provide signed, written informed consent prior to any protocol-related procedures being performed. Consent of a legally authorized guardian(s) is (are) required for patients under 18 years.
Are the trial subjects under 18? yes
Number of subjects for this age range: 5
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 1
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
•Have previously received Aldurazyme without the collection of baseline samples as specified.
•Have a suspected hypersensitivity to Aldurazyme or a know hypersensitivity to components of infusion solution.
•Have received a Hematopoietic Stem Cell Transplantation, injection fibroblast therapy, or major organ transplant.
•Are receiving chronic immunosuppressant therapy.
•Have a medical condition, serious intercurrent illness, or other extenuating circumstances that may interfere with study compliance including all prescribed evaluations and follow-up activities.
•Are pregnant or lactating
•Have received investigational drug within 30 days prior to study enrollment
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: The primary objective of this study was to determine whether the development of antibodies to laronidase in patients with MPS I receiving Aldurazyme impairs the clearance of the glycosaminoglycan (GAG) substrate.<br>;Secondary Objective: Safety was assessed by evaluating the incidence of adverse events (AEs), standard clinical chemistry, hematology, and urinalysis variables; physical examination results, and immunogenicity testing.;Primary end point(s): •Urinary GAG (glycosaminoglycans) <br>•Immunogenicity Testing<br><br>;Timepoint(s) of evaluation of this end point: •Urinary GAG (glycosaminoglycans) : Up to 4 years<br>•Immunogenicity Testing : Up to 4 years<br><br>
- Secondary Outcome Measures
Name Time Method Secondary end point(s): Safety ;Timepoint(s) of evaluation of this end point: Up to 4 years