ALID02205: Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I)

This study has been completed.

Sponsored By:	Genzyme Genzyme Corporation (Genzyme Japan K.K.) Genzyme Corporation/BioMarin, LLC
Information Provided By:	Genzyme
ClinicalTrials.gov Identifier:	NCT00258011

Purpose

This is a multi-center, open label, study conducted to evaluate the safety of laronidase administered by intravenous drip infusion in patients with MPS I disease.

Following baseline evaluation, patients will receive weekly infusions of JC0498 at an intravenous dose of 100 units/kg. Patient safety will be monitored continuously throughout the trial. In addition, the effects of JC0498 treatment in this patient population will be assessed by periodically evaluating aspects of MPS I disease in patients at scheduled intervals over the duration of the trial.

Since patients may be eligible for the trial if they have received JC0498, a portion of the data may be captured retrospectively and recorded onto the case report forms (CRFs).

This study represents the first good clinical practice (GCP) effort to characterize MPS I in the Japanese population and evaluate the effects of JC0498 on disease manifestations.

Condition	Intervention	Phase
Mucopolysaccharidosis I	Drug: Recombinant Human Alpha-L-Iduronidase, Aldurazyme® (laronidase)	N/A

Study Type: Interventional

Study Design: Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group

Assignment, Safety/Efficacy Study

Official Title: A Safety Confirmatory Study of JC0498 (Laronidase) in Mucopolysaccharidosis I

(MPS I) Patients

Further Study Details

Primary Outcomes: Safety assessment of JC0498 (laronidase)

Secondary Outcomes: Pharmacodynamic assessment of JC0498 (laronidase) with urinary glycosaminoglycan(GAG) excretion.; Tertiary objective: Efficacy assessment of JC0498 (laronidase).

Total Enrollment: 5

Study Start: 2005-11; Study Completion: 2006-10

Safety:
Safety monitoring will include physical examinations, vital signs, clinical laboratory evaluations, electrocardiogram, echocardiogram, immunogenicity testing, and adverse event monitoring.

Efficacy:
Clinical evaluations will consist of measuring GAG clearance by measuring urinary GAG excretion; presence or absence of hepatomegaly; desaturation index; Investigator global assessment of overall changes in the patient’s condition; and at the discretion of the Investigator 6-minute walk test (6MWT) and Forced Vital Capacity (FVC).

Pharmacokinetic: At the discretion of the Investigator, PK profiles may also be evaluated

Eligibility

Genders Eligible for Study: Both

Criteria

Inclusion Criteria:

written informed consent/assent of the patient or written informed consent of the parent(s) or the legal guardian(s), depending on the age of the patient, is required prior to any protocol-related procedures being performed; this includes information regarding hematopoietic stem cell transplantation (HSCT) in order to assure that the guardian(s) is fully informed regarding the risks and benefits of this alternative treatment for patients eligible for the trial and who have severe manifestations of MPS I with neurodegeneration.

have a clinical diagnosis of MPS, confirmed by measurable clinical signs and symptoms of MPSI.

have confirmed iduronidase deficiency with a leukocyte alpha-L-iduronidase enzyme activity level of less than 10.0 % of the lower limit of the normal range of the measuring laboratory (SRL);

Exclusion Criteria:

The patient is under consideration for or has previously undergone Hematopoietic Stem Cell Transplantation.

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 (exception: JC0498).

The patient has known severe hypersensitivity to JC0498 or components of the delivery solution.

Location Information

Japan
National Center for Child Health and Development, Tokyo, 157-8535, Japan

Osaka City University Hospital, Osaka, 545-8586, Japan

More Information

US FDA Approved Full Prescribing Information for Aldurazyme®

Study ID Numbers: ALID02205; JC0498
ClinicalTrials.gov Identifier: NCT00258011
Health Authority: Japan: Ministry of Health, Labor and Welfare