Mucopolysaccharidosis type 1 (MPS I) is a condition that affects many parts of the body. It is
caused by a genetic alteration of the IDUA gene, resulting in reduced levels of the IDUA
enzyme. Without proper amounts of this enzyme, sugar molecules accumulate within cellular
structures called lysosomes. This accumulation causes the lysosomes and cells to enlarge,
affecting many different organs and tissues of the body. This leads to the medical problems seen
in this condition.
MPS I is divided into different categories according to severity. This research study is seeking
participants who have either the Hurler-Scheie (MPS IH/S) or Scheie (MPS IS) categories.
Currently, there is no cure for MPS I. While enzyme replacement therapy (ERT) is the primary
treatment for MPS I Hurler-Scheie and Scheie syndromes, it cannot address all the body systems
affected by the disease. This treatment also requires frequent dosing which can interfere with
daily activities.
This is why it is so important to conduct research studies seeking to learn more about potential medications for MPS I. These research studies would be impossible to conduct without volunteer participants, which is why we ask for your help. If you provide your contact information, we will reach out to you to provide more information about the study.
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To join this research study, potential participants must: |
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* Be 18 years of age or above |
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* Have Mucopolysaccharidosis type 1, Hurler-Scheie or Scheie syndrome |
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* Be currently receiving enzyme replacement (IDUA) therapy |
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This is not a complete list of requirements to participate. Please provide us with your contact
information so we can discuss these and additional requirements with you.
Your contact information will remain confidential. It will only be used to contact you regarding
participation in this study.
Immusoft will cover travel costs.
If you are interested in participating or just wish to learn more, please provide your contact information. We will contact you after your submission.
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. Research studies depend upon the participation of volunteers.
There is no guarantee that subjects will receive any benefit from participating in this study. However, this research study may yield new information that could benefit people in the future by helping to answer questions about the effectiveness and safety of the study treatment.
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.
Health Insurance is NOT required to participate.
You will not be charged for the study drug, study-related procedures and tests, or study visits.
Before participating in a trial volunteers must meet specific requirements. These requirements are based on various factors, such as your age, medical history, current health status, current medications, and allergies. The circumstances that allow one to participate are called Inclusion Criteria and those that prevent participation are called Exclusion Criteria. These requirements are used to ensure the accuracy of the trial 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.
Immusoft is staffed by experienced medical personnel that are committed to each patient's health as well as to helping find treatments that can help patients in the future.
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