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MPS I Disease Registry

  • Status
    Accepting Candidates
  • Age
    N/A - N/A
  • Sexes
    All
  • Healthy Volunteers
    No
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Objective

The Mucopolysaccharidosis I (MPS I) Registry is an ongoing, observational database that tracks the outcomes of patients with MPS I. The data collected by the MPS I Registry will provide information to better characterize the natural history and progression of MPS I as well as the clinical responses of patients receiving enzyme replacement therapy, such as Aldurazyme (Recombinant Human Alpha-L-Iduronidase), or other treatment modalities.

The objectives of the Registry are:

  • To evaluate the long-term effectiveness and safety of Aldurazyme® (laronidase)

  • To characterize and describe the MPS I population as a whole, including the variability, progression, and natural history of MPS I

  • To help the MPS I medical community with the development of recommendations for monitoring patients and reports on patient outcomes to optimize patient care

Description

The MPS I Registry is an international program; in addition to the central contact information provided under the "Location" heading, patients may contact:

  • In Asia-Pacific * Vivian Liu, +65-6431-2548, Vivian.liu@genzyme.com

  • In Europe * +31-35-699-1232, europe@mpsiregistry.com

  • In Latin America * +617-591-5500, help@mpsiregistry.com

  • In North America * +617-591-5500, help@mpsiregistry.com

Details

Full study title Rare Disease Registry - Cross-Registry Protocol
Protocol number OCR41895
ClinicalTrials.gov ID NCT00144794

Eligibility

Inclusion Criteria:

  • All patients with a confirmed diagnosis of MPS I are eligible for inclusion. Confirmed

Diagnosis is defined as: A. documented biochemical evidence of a deficiency in alpha

(a)-L-iduronidase enzyme activity and/or B. mutation(s) in the gene coding for

a-L-iduronidase, or measurable clinical signs and symptoms of MPS I

  • For all patients there should be a completed patient authorization form

Exclusion Criteria:

  • No exclusion criteria for participation in the MPS I Registry. NOTE: Registry participation does not exclude participation in other clinical studies.

Participate in a study

Here are some general steps to consider when participating in a research study:

  1. Step
    1

    Contact the research team

    Call or email the research team listed within the specific clinical trial or study to let them know that you're interested. A member of the research team, such as the researcher or study coordinator, will be available to tell you more about the study and to answer any questions or concerns you may have.

    Primary contact

  2. Step
    2

    Get screened to confirm eligibility

    You may be asked to take part in prescreening to make sure you are eligible for a study. The prescreening process ensures it is safe for you to participate. During the prescreening process, you will be asked some questions and you may also be asked to schedule tests or procedures to confirm your eligibility.

  3. Step
    3

    Provide your consent to participate

    If you are eligible and want to join the clinical trial or study, a member of the research team will ask for your consent to participate. To give consent, you will be asked to read and sign a consent form for the study. This consent form explains the study's purpose, procedures, risks, benefits and provides other important information, such as the study team's contact information.

  4. Step
    4

    Participate

    If you decide to participate in a clinical trial or study, the research team will keep you informed of the study requirements and what you will need to do to throughout the study. For some trials or studies, your health care provider may work with the research team to ensure there are no conflicts with other medications or treatments.