Ohtahara Syndrome |
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Clinical Trial: A Phase 4 Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG Levels in Aldurazyme® (Laronidase) Treated Patients.
This study is currently recruiting patients.
Verified by Genzyme September 2005
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Purpose
| Condition | Intervention | Phase |
|---|---|---|
| Mucopolysaccharidosis I Hurler''''s Syndrome Hurler-Scheie Syndrome Scheie Syndrome | Drug: Recombinant Human Alpha-L-Iduronidase, Aldurazyme® (laronidase) | Phase IV |
MedlinePlus related topics: Metabolic Disorders
Study Type: Interventional
Study Design: Treatment, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
Official Title: A Multicenter, Multinational, Open-Label Study of Anti-Laronidase Antibody Formation and Urinary GAG Levels in Patients With Mucopolysaccharidosis I (MPS I) Being Treated With Aldurazyme® (Laronidase).
Study start: July 2004; Expected completion: July 2011
Last follow-up: December 2010; Data entry closure: March 2011
Eligibility
Inclusion Criteria:
- Have a document diagnosis of MPS I confirmed by measurable signs and symptoms and deficient α-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.
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 30days prior to study enrollment
Location and Contact Information
Medical Information 617-252-7832 medinfo@genzyme.com
California
Children''''s Hospital Los Angeles, Los Angeles, California, 90027, United States; Recruiting
More Information
Last Updated: September 2, 2005
Record first received: September 2, 2005
ClinicalTrials.gov Identifier: NCT00144768
Health Authority: United States: Food and Drug Administration
ClinicalTrials.gov processed this record on 2005-09-06

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