Registry for Hurler Syndrome
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial focuses on understanding Mucopolysaccharidosis I (MPS I), a rare genetic disorder that affects the body's ability to break down certain sugars. Researchers aim to gather information on the condition's progression and the long-term effectiveness of treatments like Aldurazyme, an enzyme replacement therapy. The goal is to enhance patient care by identifying the best methods for monitoring and treating MPS I. Individuals with a confirmed diagnosis of MPS I may find this trial suitable. As a Phase 4 trial, this research seeks to understand how the already FDA-approved and effective treatment benefits more patients, offering valuable insights for potential participants.
Do I have to stop taking my current medications for this trial?
The trial information does not specify whether you need to stop taking your current medications. It seems to focus on observing patients with MPS I and their treatments, so you may not need to change your current medications.
What is the safety track record for Aldurazyme?
Previous studies have linked Aldurazyme to serious allergic reactions, including anaphylaxis, a severe and life-threatening condition. About 1% of patients in these studies experienced severe or serious allergic reactions. Other common side effects included fever, pneumonia, cough, flu-like symptoms, and skin reactions like hives. Although these side effects can be serious, they are known and monitored during treatment. The FDA has approved Aldurazyme for treating MPS I, providing a good understanding of its safety in humans.12345
Why are researchers excited about this trial?
Aldurazyme is unique because it provides an enzyme replacement therapy specifically for Mucopolysaccharidosis I (MPS I), a condition caused by a deficiency of the enzyme alpha-L-iduronidase. Most treatments for MPS I focus on managing symptoms, but Aldurazyme works by directly replacing the missing enzyme, addressing the root cause of the disorder. Researchers are excited about this treatment because it has the potential to improve patients' quality of life by reducing the accumulation of harmful substances in the body, something traditional symptom management can't achieve.
What is the effectiveness track record for Aldurazyme in treating MPS I?
Research has shown that Aldurazyme, a type of enzyme replacement therapy, helps treat Mucopolysaccharidosis I (MPS I). In one study, patients who received Aldurazyme walked 38 meters farther, indicating better physical ability. Another study found that lung function, measured by forced vital capacity (FVC), improved significantly after using Aldurazyme. Evidence suggests that starting treatment early may lead to better results for patients. Overall, Aldurazyme has shown promising results in reducing symptoms and improving the quality of life for people with MPS I.678910
Who Is on the Research Team?
Medical Monitor
Principal Investigator
Genzyme, a Sanofi Company
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Data Collection
Ongoing collection of data to track outcomes and characterize the natural history and progression of MPS I
Follow-up
Participants are monitored for safety and effectiveness of treatments such as enzyme replacement therapy
What Are the Treatments Tested in This Trial?
Interventions
- Aldurazyme
Find a Clinic Near You
Who Is Running the Clinical Trial?
Genzyme, a Sanofi Company
Lead Sponsor
David Meeker
Genzyme, a Sanofi Company
Chief Executive Officer since 2011
MD from the University of Vermont Medical School, Advanced Management Program at Harvard Business School
Jean-Paul Kress
Genzyme, a Sanofi Company
Chief Medical Officer since 2015
MD from Faculte Necker-Enfants Malades, Paris