Registry for Hurler Syndrome

Recruiting at 693 trial locations

Overseen By MPS I Registry HelpLine

Age: Any Age

Sex: Any

Trial Phase: Academic

Sponsor: Genzyme, a Sanofi Company

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

1500 Participants Needed

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. ^{1 2 3 4 5}

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. ^{6 7 8 9 10}

Who Is on the Research Team?

Medical Monitor

Principal Investigator

Genzyme, a Sanofi Company

Are You a Good Fit for This Trial?

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

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

Ongoing

Follow-up

Participants are monitored for safety and effectiveness of treatments such as enzyme replacement therapy

Long-term

What Are the Treatments Tested in This Trial?

Interventions

• Aldurazyme

Who Is Running the Clinical Trial?

Genzyme, a Sanofi Company

Lead Sponsor

Trials

528

Recruited

186,000+

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

Citations

1.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC6581069/

Enzyme replacement therapy with laronidase (Aldurazyme ...

The study included all of the key outcome measures we wished to look at. It demonstrated that laronidase is efficacious in relation to reducing biochemical ...

2.

pro.campus.sanofi

pro.campus.sanofi/us/products/aldurazyme/clinical-trial-results

Aldurazyme® (laronidase) Clinical Trial Results

In the 26-week, randomized, double-blind, placebo-controlled study, patients treated with Aldurazyme (n=22) showed a mean 38-meters increase in distance walked ...

3.

sciencedirect.com

sciencedirect.com/science/article/pii/S0022347616307004

Outcomes of Long-Term Treatment with Laronidase in ...

Data suggest that early treatment may result in better outcomes. Previous ... analysis and efficacy of recombinant human alpha-L-iduronidase (laronidase).

4.

nature.com

nature.com/articles/s41598-025-16351-4

Efficacy and safety of a biosimilar laronidase versus the ...

In addition, the mean (± SD) predicted FVC results increased from 57.50 ± 16.53% at week 12 (related to Aldurazyme) to 70.56 ± 35.01% at week 24 ...

5.

clinicaltrials.gov

clinicaltrials.gov/study/NCT00418821?cond=(HURLER-SCHEIE%20SYNDROME)%20OR%20(IDUA)&rank=6&tab=results

A Study of the Effect of Aldurazyme® (Laronidase ...

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.

6.

pro.campus.sanofi

pro.campus.sanofi/us/products/aldurazyme/safety

Aldurazyme® (laronidase) Safety Profile | For US HCPs

Serious adverse reactions reported with Aldurazyme treatment during clinical trials were anaphylactic and hypersensitivity reactions.

7.

accessdata.fda.gov

accessdata.fda.gov/drugsatfda_docs/label/2010/125058s0186lbl.pdf

ALDURAZYME (laronidase) - accessdata.fda.gov

In clinical studies and postmarketing safety experience with ALDURAZYME, approximately 1% of patients experienced severe or serious allergic reactions. In ...

8.

aldurazyme.com

aldurazyme.com/patient/about-aldurazyme/potential-side-effects

Potential Side Effects | Aldurazyme Patient

ALDURAZYME may cause serious side effects, including: Anaphylaxis, Hypersensitivity, and Infusion-Associated Reactions (IARs).

9.

aldurazyme.com

aldurazyme.com/patient/

Aldurazyme - For Patients

SERIOUS ALLERGIC REACTIONS: Patients treated with ALDURAZYME have experienced life-threatening severe reactions, including anaphylaxis. Appropriate medical ...

10.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC12405411/

Safety assessment of laronidase: real-world adverse event ...

The frequently reported AEs align with the labeled safety profile of laronidase, including pyrexia, pneumonia, cough, influenza, urticaria, ...