Back to Results

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

Interested in this Trial?

Contact Us

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

Contact BioMarin Clinical Trials

Please complete and submit the following form to express interest in participating and request more information about one of our active clinical trials

*required fields

"*" indicates required fields

Please select the trial that you are interested in from the dropdown below. Note that this list only includes currently active trials. If you cannot find the trial you are seeking, please enter the study identifier and title in the comments box below, along with any other details you would like us to know to help address your inquiry.
Consent*
Consent*
For more Information about BioMarin's privacy obligations or to exercise your privacy rights, please visit BioMarin's Data Privacy Center.
This field is hidden when viewing the form
This field is hidden when viewing the form
This field is hidden when viewing the form
This field is hidden when viewing the form

What’s Next?

Visit the following links to learn more about clinical trials.

Discuss this trial with your care team.

You may wish to print this page to share the details with your healthcare providers and discuss whether you may be eligible.

Print

Get in touch.

Contact Us