A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old
Information source: Sanofi
ClinicalTrials.gov processed this data on August 20, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Mucopolysaccharidosis I; Hurler Syndrome; Hurler-Scheie Syndrome; Scheie Syndrome
Intervention: Aldurazyme (Recombinant Human Alpha-L-Iduronidase) (Biological); Aldurazyme (Recombinant Human Alpha-L-Iduronidase) (Biological)
Phase: Phase 2
Status: Completed
Sponsored by: Genzyme, a Sanofi Company Official(s) and/or principal investigator(s): Medical Monitor, Study Director, Affiliation: Genzyme, a Sanofi Company
Summary
The main objectives of this study are to evaluate the safety and pharmacokinetics (PK) of
enzyme replacement therapy with recombinant human alpha-L-iduronidase [Aldurazyme®
(laronidase)] in mucopolysaccharidosis I (MPS I) patients less than 5 years old. Efficacy
measurements will also be evaluated in this study.
Clinical Details
Official title: A Phase II Open-Label Clinical Trial of Recombinant Human Alpha-L-iduronidase (Aldurazyme®) to Evaluate the Safety and Pharmacokinetics in Mucopolysaccharidosis I (MPS I) Patients Less Than 5 Years Old
Study design: Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary outcome: Safety EvaluationPharmacokinetics - Area Under the (Plasma Concentration-time) Curve (AUC∞) Pharmacokinetics - Elimination Half Life (t1/2) Pharmacokinetics - Total Plasma Clearance (CL) Pharmacokinetics - Volume of Distribution (Vz)
Eligibility
Minimum age: N/A.
Maximum age: 5 Years.
Gender(s): Both.
Criteria:
Inclusion Criteria:
- Written informed consent is required from the parent(s) or legal guardian(s) prior to
any protocol-related procedures being performed. (A separate informed consent will be
requested from the parent(s) for their genotyping, which is independent of the
inclusion.)
- Be less than 5 years of age at the time of enrollment.
- Have confirmed iduronidase deficiency with a fibroblast or leukocyte
alpha-L-iduronidase enzyme activity level of less than 10. 0 % of the lower limit of
the normal range, or below the detection range of the measuring laboratory.
- Have a clinical diagnosis of MPS I based on genotyping.
- Documentation in his/her medical record that the parent(s) or legal guardian(s) have
had counseling or a consultation regarding HSCT in order to assure that the parent(s)
or legal guardian(s) are fully informed regarding the risks and benefits of this
alternative treatment for patients eligible for the trial and with the severe
manifestations of MPS I with neurodegeneration.
Exclusion Criteria:
- The patient is under consideration for or has undergone hematopoietic stem cell
transplantation (HSCT).
- The patient has acute hydrocephalus at the time of enrollment.
- The patient has a clinically significant organic disease (with the exception of
symptoms relating to MPS I) 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.
- The patient has received any investigational product within 30 days prior to trial
enrollment.
- The patient has known severe hypersensitivity to Aldurazyme® (laronidase) or
components of the delivery solution.
Locations and Contacts
Hôpital E. Herriot, Lyon, France
Sophia Children's Hospital, Rotterdam, Netherlands
Willink Biochemical Genetics Unit Royal Hospital for Children, Manchester, United Kingdom
Johannes Gutenberg Universität, Kinderklinik, Mainz, Germany
Additional Information
Link to Results Synopsis for ALID-014-02
Starting date: October 2002
Last updated: March 17, 2015
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