ALID01402: A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old

This study has been completed.

Sponsored By:	Genzyme BioMarin/Genzyme LLC
Information Provided By:	Genzyme
ClinicalTrials.gov Identifier:	NCT00146757

Purpose

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.

Condition	Intervention	Phase
confirmed diagnosis of Mucopolysaccharidosis I less than 5 years of age	Drug: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)	Phase II

Study Type: Interventional

Study Design: Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study

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

Further Study Details:

Primary Outcomes: To evaluate the safety, PK and efficacy of Aldurazyme® (laronidase) in a patient population younger than 5 years old

Study Start: 2002-12; Study Completion: 2005-05

Eligibility

Ages Eligible for Study: up to 5 Years, Genders Eligible for Study: 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 enrolment.

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 diagnosis of MPS I based on genotyping.

Documentation in his/her medical record that the parent(s) or legal guardian(s) have had counselling 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 HSCT.

The patient has acute hydrocephalus at the time of enrolment.

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 enrolment.

The patient has known severe hypersensitivity to Aldurazyme® (Laronidase) or components of the delivery solution.

Location Information

United Kingdom
Willink Biochemical Genetics Unit Royal Hospital for Children, Manchester, M25 4HA, United Kingdom

More Information

US FDA Approved Full Prescribing Information for Aldurazyme®

Results synopsis for Clinical Trial Protocol ALID01402

Study ID Numbers: ALID-014-02
ClinicalTrials.gov Identifier: NCT00146757
Health Authority: European Union: European Medicines Agency