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

Primary Purpose

Mucopolysaccharidosis I, Hurler Syndrome, Hurler-Scheie Syndrome

Status
Completed
Phase
Phase 3
Locations
Japan
Study Type
Interventional
Intervention
Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
Sponsored by
Genzyme, a Sanofi Company
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Mucopolysaccharidosis I focused on measuring MPS I Disease

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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 MPS I. 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.

Sites / Locations

  • Osaka City University Hospital
  • National Center for Child Health and Development

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Aldurazyme (laronidase) treatment

Arm Description

Patients received weekly infusions of JC0498 (laronidase) at an intravenous dose of 100 Units/kg (0.58 mg/kg) body weight for up to 73 weeks.

Outcomes

Primary Outcome Measures

Safety Evaluation
Overall Safety Summary of Adverse Events (AEs) during Treatment Safety assessment was based on the incidence of AE reports.

Secondary Outcome Measures

Urinary Glycosaminoglycan (GAG) Excretion
Percentage change in the concentration of GAG relative to creatinine in urine (ug GAG/mg creatinine) from baseline to last study visit. Greater decrease indicates greater response.

Full Information

First Posted
November 22, 2005
Last Updated
February 4, 2014
Sponsor
Genzyme, a Sanofi Company
Collaborators
BioMarin/Genzyme LLC
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1. Study Identification

Unique Protocol Identification Number
NCT00258011
Brief Title
Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease
Official Title
A Safety Confirmatory Study of JC0498 (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients
Study Type
Interventional

2. Study Status

Record Verification Date
February 2014
Overall Recruitment Status
Completed
Study Start Date
December 2005 (undefined)
Primary Completion Date
October 2006 (Actual)
Study Completion Date
October 2006 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
Genzyme, a Sanofi Company
Collaborators
BioMarin/Genzyme LLC

4. Oversight

5. Study Description

Brief Summary
This is a multi-center, open label, study conducted to evaluate the safety of laronidase administered by intravenous drip infusion in Japanese 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Mucopolysaccharidosis I, Hurler Syndrome, Hurler-Scheie Syndrome, Scheie Syndrome
Keywords
MPS I Disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
3 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Aldurazyme (laronidase) treatment
Arm Type
Experimental
Arm Description
Patients received weekly infusions of JC0498 (laronidase) at an intravenous dose of 100 Units/kg (0.58 mg/kg) body weight for up to 73 weeks.
Intervention Type
Biological
Intervention Name(s)
Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
Intervention Description
0.58 mg/kg every week
Primary Outcome Measure Information:
Title
Safety Evaluation
Description
Overall Safety Summary of Adverse Events (AEs) during Treatment Safety assessment was based on the incidence of AE reports.
Time Frame
Up to 73 Weeks
Secondary Outcome Measure Information:
Title
Urinary Glycosaminoglycan (GAG) Excretion
Description
Percentage change in the concentration of GAG relative to creatinine in urine (ug GAG/mg creatinine) from baseline to last study visit. Greater decrease indicates greater response.
Time Frame
Up to 73 Weeks

10. Eligibility

Sex
All
Accepts Healthy Volunteers
No
Eligibility 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 MPS I. 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Shigetoyo Oguri
Organizational Affiliation
Corp. GCP Compliance - Clinical Affairs, Genzyme Japan K.K.
Official's Role
Study Director
Facility Information:
Facility Name
Osaka City University Hospital
City
Osaka
ZIP/Postal Code
545-8586
Country
Japan
Facility Name
National Center for Child Health and Development
City
Tokyo
ZIP/Postal Code
157-8535
Country
Japan

12. IPD Sharing Statement

Learn more about this trial

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

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