NCT00144781

Brief Summary

The main purpose of this study is to evaluate differences in the pharmacodynamic response of 4 Aldurazyme® (laronidase) dose regimens in patients with Mucopolysaccharidosis I (MPS I).

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
34

participants targeted

Target at below P25 for phase_4

Timeline
Completed

Started Dec 2004

Shorter than P25 for phase_4

Geographic Reach
2 countries

5 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

December 1, 2004

Completed
9 months until next milestone

First Submitted

Initial submission to the registry

September 2, 2005

Completed
3 days until next milestone

First Posted

Study publicly available on registry

September 5, 2005

Completed
4 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2006

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2006

Completed
3.5 years until next milestone

Results Posted

Study results publicly available

June 16, 2009

Completed
Last Updated

April 3, 2015

Status Verified

March 1, 2015

Enrollment Period

1.1 years

First QC Date

September 2, 2005

Results QC Date

March 27, 2009

Last Update Submit

March 17, 2015

Conditions

Mucopolysaccharidosis IHurler's SyndromeHurler-Scheie SyndromeScheie Syndrome

Outcome Measures

Primary Outcomes (1)

  • Percent Change From Baseline to Week 26 in Urinary Glycosaminoglycan (GAG) Level

    Urinary GAG Level - Concentration of GAG relative to creatinine in urine. A greater decrease in GAG level indicates a greater response.

    Baseline to 26 Weeks

Secondary Outcomes (2)

  • Percent Change From Baseline to Week 26 in Liver Organ Volume

    Baseline to 26 Weeks

  • Change From Baseline to Week 26 in Six Minute Walk Test (6MWT)

    Baseline to 26 Weeks

Study Arms (4)

1

ACTIVE COMPARATOR

0.58 mg Aldurazyme/kg of body weight (100 U/kg) administered every week (labeled dose). Final Visit is Week 27 for patients randomized to every week regimen.

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

2

ACTIVE COMPARATOR

1.2 mg Aldurazyme/kg of body weight (200 U/kg) administered every week. Final Visit is Week 27 for patients randomized to every week regimen.

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

3

ACTIVE COMPARATOR

1.2 mg Aldurazyme/kg of body weight (200 U/kg) administered every 2 weeks. Final Visit is Week 26 for patients randomized to every 2 week regimen.

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

4

ACTIVE COMPARATOR

1.8 mg Aldurazyme/kg of body weight (300 U/kg) administered every 2 weeks. Final Visit is Week 26 for patients randomized to every 2 week regimen.

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

Interventions

Aldurazyme (Recombinant Human Alpha-L-Iduronidase)BIOLOGICAL

0.58 mg/kg every week

1

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Have a documented diagnosis of MPS I, confirmed by measurable clinical signs and symptoms of MPS I, and a documented fibroblast or leukocyte α-L-iduronidase enzyme activity level of less than 10% of the lower limit of the normal range of the measuring laboratory.
  • Weigh at least 12.5 kg.
  • Have serum creatinine and blood urea nitrogen (BUN) values within age appropriate normal ranges.
  • Provide signed, written informed consent prior to any protocol-related procedures being performed. Consent of a legally authorized guardian(s) is (are) required for patients under 18 years. If the patient is under 18 years old and can understand the consent, written informed consent will be required from both the patient and the authorized guardian(s).

You may not qualify if:

  • Have previously received Aldurazyme® (laronidase).
  • Have a suspected hypersensitivity to Aldurazyme® (laronidase) or known hypersensitivity to components of the infusion solution.
  • Have previously undergone hematopoietic stem cell transplantation (HSCT; i.e., from bone marrow \[BMT\], peripheral blood, or umbilical cord blood) or other major organ transplantation.
  • Have a medical condition, serious inter-current illness, or other extenuating circumstance that may interfere with study compliance including all prescribed evaluations and follow-up activities, except the 6MWT. (Note: All patients may not be capable of performing the 6MWT due to age and/or maturity level. Exemption from performing the 6MWT must be obtained in writing by the investigator from the sponsor's medical monitor prior to enrollment).
  • Have an acute illness that requires surgical intervention, and/or anticipates surgery during study participation, and/or has had surgery within 30 days prior to study enrollment.
  • Have received an investigational drug within 30 days prior to study enrollment.
  • Is pregnant or lactating. Female patients of childbearing potential must have a negative pregnancy test \[urine β-human chronic gonadotropin (hCG)\] at entry (prior to the first infusion). Note: All female patients of childbearing potential and sexually mature males must be advised to use a medically accepted method of contraception throughout the study.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (5)

Hospital Infantil Joana de Gusmao

Florianópolis, Santa Catarina, CEP 88025-601, Brazil

Location

Universidade Federal de Minas Gerais

Belo Horizonte, CEP 30130-100, Brazil

Location

Hospital de Clinical de Porto Alegre

Porto Alegre, CEP 90035-003, Brazil

Location

Universidade Federal de Sao Paulo

São Paulo, CEP 04023-062, Brazil

Location

Division of Clinical and Metabolic Genetics

Toronto, Ontario, M5G 1X8, Canada

Location

MeSH Terms

Conditions

Mucopolysaccharidosis I

Interventions

Iduronidase

Condition Hierarchy (Ancestors)

MucopolysaccharidosesCarbohydrate Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLysosomal Storage DiseasesMucinosesConnective Tissue DiseasesSkin and Connective Tissue DiseasesMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Glycoside HydrolasesHydrolasesEnzymesEnzymes and Coenzymes

Limitations and Caveats

This study is limited by its small sample size and ability to detect small differences in urinary GAG levels between groups.

Results Point of Contact

Title
Genzyme Medical Information
Organization
Genzyme Corporation
800-745-4447

Study Officials

  • Medical Information

    Genzyme, a Sanofi Company

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 4
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY

Study Record Dates

First Submitted

September 2, 2005

First Posted

September 5, 2005

Study Start

December 1, 2004

Primary Completion

January 1, 2006

Study Completion

January 1, 2006

Last Updated

April 3, 2015

Results First Posted

June 16, 2009

Record last verified: 2015-03

Locations

BR(4)CA(1)