EUCTR2015-000585-61-Outside-EU/EEAActive, not recruitingNot Applicable
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)
Genzyme, a Sanofi Company0 sites6 target enrollmentStarted: May 18, 2015Last updated:
Trial Snapshot
- Phase
- Not Applicable
- Status
- Active, not recruiting
- Sponsor
- Enrollment
- 6
Study Overview
Brief Summary
No summary available.
Study Design
- Study Type
- Interventional clinical trial of medicinal product
Eligibility Criteria
- Sex
- All
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
Investigators
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