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

NCT00258011 | Completed Phase 3 Results

• 1 other identifier: ALID02205
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 2

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.

Conditions

Mucopolysaccharidosis I; Hurler Syndrome; Hurler-Scheie Syndrome; Scheie Syndrome

Keywords

MPS I Disease

Outcome Measures

Primary Outcomes (1)

• Safety Evaluation

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

  Up to 73 Weeks

Secondary Outcomes (1)

• Urinary Glycosaminoglycan (GAG) Excretion

  Up to 73 Weeks

Study Arms (1)

Aldurazyme (laronidase) treatment

EXPERIMENTAL

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.

Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)

Interventions

Aldurazyme (Recombinant Human Alpha-L-Iduronidase) BIOLOGICAL

0.58 mg/kg every week

Aldurazyme (laronidase) treatment

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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.

Sponsors & Collaborators

• Genzyme, a Sanofi Company: lead
• BioMarin/Genzyme LLC: collaborator

Study Sites (2)

Osaka City University Hospital

Osaka, 545-8586, Japan

Location

National Center for Child Health and Development

Tokyo, 157-8535, Japan

Location

MeSH Terms

Conditions

Mucopolysaccharidosis I

Interventions

Iduronidase

Condition Hierarchy (Ancestors)

Mucopolysaccharidoses; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lysosomal Storage Diseases; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Glycoside Hydrolases; Hydrolases; Enzymes; Enzymes and Coenzymes

Limitations and Caveats

Due to the rarity of the disease, this study is limited by its small sample size, lack of a control group and patients had variable treatment durations.

Results Point of Contact

• Title: Genzyme Medical Information
• Organization: Genzyme Corporation

800-745-4447

Study Officials

• Shigetoyo Oguri

  Corp. GCP Compliance - Clinical Affairs, Genzyme Japan K.K.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY

Study Record Dates

• First Submitted: November 22, 2005
• First Posted: November 24, 2005
• Study Start: December 1, 2005
• Primary Completion: October 1, 2006
• Study Completion: October 1, 2006
• Last Updated: March 7, 2014
• Results First Posted: February 12, 2009

Record last verified: 2014-02

Locations

JP (2)