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Completed

NCT Number: NCT00146770

Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients

This study is being conducted to collect additional long-term efficacy and safety data of Aldurazyme® (laronidase) patients with MPS I disease. Patients who were previously enrolled in the Phase 3 Double-Blind Study will be enrolled in this study.

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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's Syndrome Hurler-Scheie Syndrome Lysosomal Storage Diseases Metabolic Diseases Metabolism, Inborn Errors Mucinoses Mucopolysaccharidoses Nutritional and Metabolic Diseases Scheie Syndrome Skin and Connective Tissue Diseases

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Hospital Infantil Joana de Gusmao, Florianópolis, Brazil

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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 or patient's legal guardian must provide written informed consent prior to any protocol-related procedures being performed.
  • The patient must have successfully completed Study ALID-003-99 (who received 21 of 26 consecutive weekly infusions).
  • The patient has not experienced any safety issues that would contraindicate participation in the Extension study.
  • A female patient of childbearing potential must have a negative pregnancy test at entry

Exclusion criteria

  • The patient is pregnant or lactating.
  • The patient has received an investigational drug within 30 days prior to the study enrollment.
  • The patient has a medical condition, serious intercurrent illness, or other extenuating circumstance that may significantly interfere with study compliance including all prescribed evaluations and follow-up activities

Treatment and study plan

Aldurazyme

Biological

Placebo for 26 weeks then 0.58 mg/kg Aldurazyme every week for 182 weeks

Placebo

Biological

Placebo for 26 weeks

Primary outcomes

  1. Change From Baseline to Week 182 in Percent Predicted Forced Vital Capacity (FVC)

    Time frame: Baseline to Week 182

    Percent Predicted Forced Vital Capacity: the maximal exhaled breath volume following a maximal inhaled breath. Overall change from Baseline to Week 182 in percent predicted FVC = (observed value)/(predicted value) * 100%). A higher value indicates a greater response.

  2. Change From Baseline to Week 182 in Six Minute Walk Test (6MWT)

    Time frame: Baseline to Week 182

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

Secondary outcomes

  1. Change From Baseline to Week 182 in Apnea/Hypopnea Index (AHI)

    Time frame: Baseline to Week 182

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

  2. Change From Baseline to Week 182 in Liver Volume

    Time frame: Baseline to Week 182

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

  3. Change From Baseline to Week 182 in Child Health Assessment Questionnaire/Health Assessment Questionnaire (CHAQ/HAQ) Disability Index Score

    Time frame: Baseline to Week 182

    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. Change From Baseline to Week 182 in Active Joint Range of Motion (ROM)

    Time frame: Baseline to Week182

    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.

Other outcomes

  1. Change From Baseline to Week 182 in Urinary GAG Level

    Time frame: Baseline to Week 182

    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 Multicenter, Multinational, Open-Label Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I

Important dates

Study start
2001
Primary completion
2005
Study completion
2005
First posted
Sep 7, 2005
Registry last updated
Apr 3, 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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