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Completed

NCT Number: NCT00912925

Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I

This study is being conducted to demonstrate the safety and clinical efficacy of Aldurazyme treatment in MPS I patients

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Key information

Conditions

Mucopolysaccharidosis I Carbohydrate Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Genetic Diseases, Inborn Hurler-Scheie Syndrome Hurlers Syndrome Lysosomal Storage Diseases Metabolic Diseases Metabolism, Inborn Errors Mucinoses Mucopolysaccharidoses Nutritional and Metabolic Diseases Skin and Connective Tissue Diseases

Age range

5 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Vancouver, British Columbia, Canada

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • The patient had a documented diagnosis of MPS I confirmed by measurable clinical signs and symptoms of MPS I and a fibroblast or leukocyte alpha-L-iduronidase enzyme activity level of less than 10% of the lower limit of the normal range of the measuring laboratory.
  • Female patients of childbearing potential had a negative pregnancy test (urine-beta-human chorionic gonadotropin (hCG)) at baseline (all female patients of childbearing potential and sexually mature male patients were advised to use a medically accepted method of contraception throughout the study).
  • The patient was capable of standing independently for 6 minutes and walking a minimum of 5 meters within 6 minutes.
  • The patient was capable of performing a reproducible FVC maneuver.
  • The patient had a baseline FVC value that was less than or equal to 80% of the patient's predicted normal FVC value based on polgar predicted values for standing height for children 5 through 7 years of age and the Hankinson predicted values for ages 8 and above.

Exclusion criteria

  • The patient had undergone a tracheostomy.
  • The patient had previously undergone a bone marrow transplantation.
  • The patient was pregnant or lactating.
  • The patient has received an investigational drug within 30 days prior to study enrollment.
  • The patient had a medical condition, serious intercurrent illness, or other extenuating circumstance that could have significantly interfered with study compliance including all prescribing evaluations and follow-up activities.
  • The patient had a known hypersensitivity to rhIDU or to components of the active or placebo test solutions.

Treatment and study plan

rhIDU (recombinant human-Alpha-L-Iduronidase)

Biological

Patients in the active treatment group received Aldurazyme intravenously at a dose of 100 units/kg (approximately 0.58mg/kg) administered intravenously over approximately 4 hours once weekly for 26 weeks.

Placebo

Biological

Patients in the Placebo-control group were administered a solution of 100mM sodium phosphate , 150mM sodium chloride, and 0.001% polysorbate-80, adjusted to a pH of 5.8 administered intravenously over a time period of approximately 4 hours once weekly for 26 weeks.

Primary outcomes

  1. Overall Change From Baseline to Week 26 in Percent Predicted Forced Vital Capacity (FVC)

    Time frame: Baseline to Week 26

    Percent Predicted Forced Vital Capacity (FVC): the maximal exhaled breathe volume following a maximal inhaled breath. Overall Change from Baseline to Week 26 in percent predicted FVC = (observed value)/(predicted value) * 100%). A higher value indicates a greater response.

  2. Overall Change From Baseline to Week 26 in Six Minute Walk Test (6MWT)

    Time frame: Baseline to Week 26

    Six Minute Walk Test (6MWT): Distance walked (measured in meters) in 6 minutes. A longer distance indicates a greater response.

Secondary outcomes

  1. Overall Change From Baseline to Week 26 in Apnea/Hypopnea Index (AHI)

    Time frame: Baseline to Week 26

    Apnea/Hypopnea Index (AHI): Number of absent (apnea) and shallow (hypopnea) breaths per hour of sleep. Overall change from Baseline to Week 26 in AHI. A greater decrease in events indicates a greater response.

  2. Overall Percent Change From Baseline to Week 26 in Liver Volume

    Time frame: Baseline to Week 26

    Liver Organ Volume: Volume of liver measured by Magnetic Resonance Imaging (MRI). Greater decrease in volume indicates a greater response.

  3. Overall Change From Baseline to Week 26 in Child Health Assessment Questionnaire/Health Assessment Questionnaire (CHAQ/HAQ) Disability Index Score

    Time frame: Baseline to week 26

    CHAQ/HAQ) = Patient questionnaire that measures the degree of disability on a scale of 0 (no disability) to 3 (maximal disability). A lower score indicates a greater response.

  4. Overall Change From Baseline to Week 26 in Active Joint Range of Motion (ROM)

    Time frame: Baseline to Week 26

    Active Joint Range of Motion (ROM): Shoulder Flexion Ability to maximally raise one's arm overhead without assistance. Shoulder range of motion (mean of left and right arms) measured in degrees (0-180) by goniometry. Greater degree of flexion indicates greater response.

  5. Overall Percent Change From Baseline to Week 26 in Urinary Glycosaminoglycan (GAG) Levels

    Time frame: Baseline to Week 26

    Urinary Glycosaminoglycan (GAG) Levels: Concentration of GAG relative to creatinine in urine. A greater decrease in GAG level indicates a greater response.

Sponsors and collaborators

Lead sponsor

Genzyme, a Sanofi Company

Industry

Collaborators

  • BioMarin/Genzyme LLC

Registry information

Official study title

A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Multinational, Clinical Study of Recombinant Human Alpha L-Iduronidase In Patients With Mucopolysaccharidosis I

Important dates

Study start
2000
Primary completion
2001
Study completion
2001
First posted
Jun 3, 2009
Registry last updated
Apr 7, 2015

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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