A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients

• Register: EUCTR
• Last refreshed on: 10 July 2015
• Main ID: EUCTR2015-000585-61-Outside-EU/EEA
• Date of registration: 18/05/2015
• Prospective Registration: Yes
• Primary sponsor: Genzyme, a Sanofi Company
• Public title: A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients
• Scientific title: A Multicenter, Multinational, Open-Label Study of Anti-Laronidase Antibody Formation and Urinary GAG Levels in Patients with Mucopolysaccaridosis I (MPS I) Being Treated with Aldurazyme® (laronidase)
• Target sample size: 6
• Recruitment status: NA
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2015-000585-61
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no Number of treatment arms in the trial: 1

Countries of recruitment

United States

Contacts

• Name: Trial Transparency Team
• Affiliation: Genzyme, a Sanofi Company

• Name: Trial Transparency Team
• Affiliation: Genzyme, a Sanofi Company

Key inclusion & exclusion criteria

Inclusion criteria:
•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

Exclusion criteria:
•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



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Mucopolysaccharidosis I Hurler's Syndrome Hurler-Scheie Syndrome Scheie's Syndrome
MedDRA version: 18.0 Level: PT Classification code 10056886 Term: Mucopolysaccharidosis I System Organ Class: 10010331 - Congenital, familial and genetic disorders

Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Intervention(s)

Product Name: Laronidase
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: LARONIDASE
CAS Number: 210589-09-6
Concentration unit: U/ml unit(s)/millilitre
Concentration type: equal
Concentration number: 100-

Primary Outcome(s)

Timepoint(s) of evaluation of this end point: •Urinary GAG (glycosaminoglycans) : Up to 4 years
•Immunogenicity Testing : Up to 4 years

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.

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)
•Immunogenicity Testing

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: Up to 4 years

Secondary end point(s): Safety

Secondary ID(s)

ALID02003

NCT00144768

Source(s) of Monetary Support

Genzyme, a Sanofi Company