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

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Trial Status Completed

Trial Identifier

NCT00912925

Condition

MPS 1, Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

Trial Summary

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

Eligibility Criteria

Birth Sex

All

Age

From 5 Years

Healthy Volunteers

No

Drug/Treatment:

rhIDU (recombinant human-Alpha-L-Iduronidase), Placebo

Phase:

Phase 3

Study Type:

Interventional

Number of Participants:

45

Study Started:

2000-12

Study Updated:

2015-04-07

Trial Locations

  • New York, New York, United States

  • Chapel Hill, North Carolina, United States

  • Vancouver, British Columbia, Canada

  • Mainz, , Germany

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

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