Explore an MPS I Research Study

Adolescents ages 10 – 17 and adults with Hurler-Scheie or Scheie syndrome may be eligible to participate.

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About MPS I

About Mucopolysaccharidosis type I (MPS I)

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Mucopolysaccharidosis type I (MPS I) is a condition that can affect many parts of the body. MPS I is caused by changes in the IDUA gene that result in low levels of an enzyme called alpha-L-iduronidase. Without enough of this enzyme, complex sugar molecules can build up within cellular structures called lysosomes. Over time, this buildup causes the lysosomes and cells to enlarge, affecting many different organs and tissues which leads to health problems associated with MPS I.

MPS I can vary in severity and is divided into three types: Hurler, Hurler-Scheie, or Scheie syndrome.

About This Study

This research study is seeking adolescents ages 10 – 17 as well as adults who have been diagnosed with Hurler-Scheie syndrome (MPS IH/S) or Scheie syndrome (MPS IS).

Enzyme replacement therapy, also called ERT, is a primary treatment for people with Hurler-Scheie and Scheie syndrome. However, people receiving ERT may continue to experience disease-related complications. Researchers are therefore studying an investigational treatment to learn more about its safety and tolerability in people with MPS I.

Research studies depend on volunteers. If you or your child participates, it may help researchers learn more about investigational treatments for MPS I that may help shape care in the future. If you think participation may be right for you or your child, answer a few brief questions to see whether the study may be an option. Travel costs associated with study participation will be covered by the study Sponsor.

Short questionnaire

See if this study is right for you

How it works:

Illustration of a study coordinator talking with a visitor at a desk

Answer a few questions (2–3 minutes)

Find out if you may qualify

If eligible, choose to share your contact information with a Study Center

Pre-Qualifying Questionnaire

To see if you qualify for this research study, please complete this Pre-Qualifying Questionnaire.

If you are interested in learning more, the first step is a short questionnaire. Your answers will help determine whether this study may be a good fit for you.

If you complete the questionnaire and prequalify based on your answers, you will have the opportunity to send your contact information to the Study Center.
We will contact you to discuss potential participation and next steps.
Your contact information will be saved only with your consent after completing the questionnaire.

What to Expect

If you or your child decides to move forward, the study team will explain the study and review the appropriate informed consent documents before any study procedures are performed.

Screening & Enrollment

Screening must be completed within 28 days and may include a review of medical and surgical history, current medications and treatments, physical examinations, blood and urine tests, heart and lung assessments, imaging, mobility tests, and other study-related evaluations. These evaluations will help the study team determine whether all eligibility requirements are met.

Cell Collection and Preparation

Study Treatment and Early Monitoring

Long-Term Follow-Up

Clinic information

Participating Study Locations

University of California San Francisco

Study Coordinator

(707) 318-7657

747 52nd Street
Oakland, California 94609 (map)

University of Minnesota

Study Coordinator

(612) 624-3769

425 East River Parkway
Minneapolis, Minnesota 55455 (map)

FAQs

Frequently Asked Questions (FAQs)

Doctors and scientists are constantly discovering new medications and devices that may help prevent, detect, diagnose, control, and treat illnesses. A research study or clinical trial is a scientific study that tests these new discoveries for safety and how well they work. Research studies depend upon the participation of volunteers.

Participation in a research study is completely voluntary. In all research studies, qualified potential participants are first informed about the research study in detail. After receiving this information, all participants must give their consent in writing to proceed. Some research studies involve more tests and doctor visits than the participant would normally have for an illness or condition.

This research study may yield new information that could benefit people in the future by helping to answer questions about the investigational drug. While participants may not receive direct medical benefit from participating in this study, they may be compensated for their time and travel.

Before participating in a research study, volunteers must meet specific requirements. These requirements are based on various factors, such as age, medical history, current health status, current medications, and allergies. The circumstances that allow a person to participate are called Inclusion Criteria (requirements to join), and those that prevent participation are called Exclusion Criteria (reasons you may not be able to join). These requirements are used to ensure the accuracy of the study as well as the safety of all participants. We will not know whether you satisfy all study requirements until we speak with you and, if appropriate, complete a screening assessment at our facilities.

There is no cost to participate, receive the investigational drug, visit the study center, or undergo study-related procedures, tests, and exams. Participants who meet study requirements may be compensated for study-related expenses. Study-related travel costs will be covered by the study Sponsor.

Take the First Step

Could an MPS I Study be an Option?

Start the Questionnaire