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

Interested in this Trial?

Contact Us

Trial Status Completed

Trial Identifier

NCT00146770

Condition

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

Eligibility Criteria

Birth Sex

All

Age

Any

Healthy Volunteers

No

Drug/Treatment:

Aldurazyme, Aldurazyme, placebo

Phase:

Phase 3

Study Type:

Interventional

Number of Participants:

45

Study Started:

2001-05

Study Updated:

2015-04-03

Trial Locations

  • University of South Alabama

    Mobile, Alabama, United States

  • Emory University School of Medicine

    Atlanta, Georgia, United States

  • University of Rochester

    Rochester, New York, United States

  • University of North Carolina at Chapel Hill

    Chapel Hill, North Carolina, United States

  • Toledo Children's Hospital

    Toledo, Ohio, United States

  • Merle West Medical Center

    Klamath Falls, Oregon, United States

  • The Childrens Hospital of Philadelphia

    Philadelphia, Pennsylvania, United States

  • Rhode Island Hospital

    Providence, Rhode Island, United States

  • Hospital Universatario de Universidade Federal de Santa Catarina

    Florianopolis, , Brazil

  • Hospital Infantil Joana de Gusmao

    Florianopolis, , Brazil

  • The Hospital for Sick Children

    Toronto, Ontario, Canada

  • Alberta Children's Hospital

    Calgary, , Canada

  • Children's Hospital Klinikum Nord Heidberg

    Hamburg, , Germany

  • Medizinishe Hochshule Hannover

    Hannover, , Germany

  • Children's Hospital at the University Hospital of Heidelberg

    Heidelberg, , Germany

  • Children's Hospital Klinikum der F.S. Universitat

    Jena, , Germany

  • Catholic University Sacro Cuore

    Rome, , Italy

  • Academisch Ziekenhuis Rotterdam

    Rotterdam, , Netherlands

  • Blackpool Victoria Hospital

    Blackpool, Lancashire, United Kingdom

  • Belfast City Hospital

    Belfast, , United Kingdom

  • Birmingham Children's Hospital

    Birmingham, , United Kingdom

  • Bristol Royal Hospital for Children and Frenchay Hospital

    Bristol, , United Kingdom

  • Gartnavel Hospital

    Glasgow, , United Kingdom

  • Great Ormond Street Hospital for Sick Children and NHS Trust

    London, , United Kingdom

  • Royal Victoria Hospital

    Newcastle upon Tyne, , United Kingdom

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 activitie

Contact BioMarin Clinical Trials

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

By completing this form and clicking the send button, you understand and hereby consent to storage of your personal information, including within the United States and Europe, which will be accessible by BioMarin for purposes such as responding to your request, quality control, fulfilling compliance obligations, and assisting with products, services, or clinical trials. Depending on where you live, you may have the right to request access to, removal, or correction of your personal information held by BioMarin. You may submit your request via the contact information and/or webform located within the full BioMarin Privacy Policy.

*required fields

"*" indicates required fields

This field is hidden when viewing the form
This field is hidden when viewing the form