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

This study has been completed.
Sponsor:
Collaborator:
BioMarin/Genzyme LLC
Information provided by:
Sanofi
ClinicalTrials.gov Identifier:
NCT00258011
First received: November 22, 2005
Last updated: February 4, 2014
Last verified: February 2014

November 22, 2005
February 4, 2014
December 2005
October 2006   (final data collection date for primary outcome measure)
Safety Evaluation [ Time Frame: Up to 73 Weeks ] [ Designated as safety issue: Yes ]
Overall Safety Summary of Adverse Events (AEs) during Treatment Safety assessment was based on the incidence of AE reports.
Safety assessment of JC0498 (laronidase)
Complete list of historical versions of study NCT00258011 on ClinicalTrials.gov Archive Site
Urinary Glycosaminoglycan (GAG) Excretion [ Time Frame: Up to 73 Weeks ] [ Designated as safety issue: No ]
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.
  • Pharmacodynamic assessment of JC0498 (laronidase) with urinary glycosaminoglycan(GAG) excretion.
  • Tertiary objective: Efficacy assessment of JC0498 (laronidase).
Not Provided
Not Provided
 
Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease
A Safety Confirmatory Study of JC0498 (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients

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.

Not Provided
Interventional
Phase 3
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
  • Mucopolysaccharidosis I
  • Hurler Syndrome
  • Hurler-Scheie Syndrome
  • Scheie Syndrome
Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
0.58 mg/kg every week
Experimental: Aldurazyme (laronidase) treatment
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: Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
3
October 2006
October 2006   (final data collection date for primary outcome measure)

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.
Both
Not Provided
No
Contact information is only displayed when the study is recruiting subjects
Japan
 
NCT00258011
ALID02205
Not Provided
Medical Monitor, Genzyme Corporation
Genzyme, a Sanofi Company
BioMarin/Genzyme LLC
Study Director: Shigetoyo Oguri Corp. GCP Compliance - Clinical Affairs, Genzyme Japan K.K.
Sanofi
February 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP