Mucopolysaccharidosis I (MPS I) Registry
Public ClinicalTrials.gov record NCT00144794. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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
Study identification
- NCT ID
- NCT00144794
- Recruitment status
- Completed
- Study type
- Observational
- Phase
- Not listed
- Enrollment
- 1,406 participants
Conditions and interventions
Conditions
Interventions
Not listed
Eligibility (public fields only)
- Age range
- Not listed
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Nov 19, 2003
- Primary completion
- Dec 30, 2024
- Completion
- Dec 30, 2024
- Last update posted
- Feb 26, 2026
2003 – 2024
United States locations
- U.S. sites
- 53
- U.S. states
- 32
- U.S. cities
- 47
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Phoenix Children's Hospital- Site Number : 840003 | Phoenix | Arizona | 85016 | — |
| Arkansas Children's Hospital- Site Number : 840109 | Little Rock | Arkansas | 72202 | — |
| Southern California Permanente Medical Group- Site Number : 840108 | Los Angeles | California | 90027 | — |
| USC Health Sciences Center Dept of Genetics- Site Number : 840082 | Los Angeles | California | 90033 | — |
| UCSF Benioff Children's Hospital - Oakland- Site Number : 840029 | Oakland | California | 94609 | — |
| Children's Hospital of Orange County- Site Number : 840074 | Orange | California | 92868 | — |
| Stanford Unviersity MC Dept of Genetics- Site Number : 840022 | Palo Alto | California | 94305 | — |
| UC Davis MIND Institute- Site Number : 840010 | Sacramento | California | 95817 | — |
| University of California at San Diego- Site Number : 840007 | San Diego | California | 92093 | — |
| University of California at San Francisco- Site Number : 840051 | San Francisco | California | — | — |
| Children's Hospital Colorado - Aurora- Site Number : 840069 | Aurora | Colorado | 80045 | — |
| University of Colorado at Denver Genetics Site Number : 840068 | Aurora | Colorado | 80045 | — |
| Yale University School of Medicine- Site Number : 840047 | New Haven | Connecticut | 06510 | — |
| Children's National Medical Center - Washington- Site Number : 840067 | Washington D.C. | District of Columbia | 20010 | — |
| University of Florida Dept of Genetics- Site Number : 840083 | Gainesville | Florida | 32610 | — |
| University Of Miami SOM- Site Number : 840006 | Miami | Florida | 33136 | — |
| Pediatric Endocrine Associates- Site Number : 840017 | Tampa | Florida | 33612 | — |
| Emory University School of Medicine- Human Genetics- Site Number : 840060 | Decatur | Georgia | 30033 | — |
| Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 840011 | Chicago | Illinois | 60611 | — |
| Rush University Medical Center Genetics- Site Number : 840079 | Chicago | Illinois | 60612 | — |
| Indianapolis University School of Medicine- Site Number : 840027 | Indianapolis | Indiana | 46202 | — |
| University of Iowa- Site Number : 840032 | Iowa City | Iowa | 52242 | — |
| University of Louisville- Genetics- Site Number : 840030 | Louisville | Kentucky | 40202 | — |
| John Hopkins- Site Number : 840044 | Baltimore | Maryland | — | — |
| Massachusetts General Hospital-Genetics- Site Number : 840062 | Boston | Massachusetts | 02114 | — |
| Boston Children's Hospital - PIN- Site Number : 840093 | Boston | Massachusetts | 2115 | — |
| Baystate Medical Center- Site Number : 840002 | Springfield | Massachusetts | 01199 | — |
| University of Michigan Pediatrics- Site Number : 840107 | Ann Arbor | Michigan | 48109 | — |
| University of Minnesota Medical Center Pediatrics- Site Number : 840076 | Minneapolis | Minnesota | 55455 | — |
| Washington University- Site Number : 840100 | St Louis | Missouri | 63110 | — |
| University of Nebraska Medical Center- Site Number : 840084 | Omaha | Nebraska | 68198 | — |
| Children's Specialty Center - Las Vegas- Site Number : 840008 | Las Vegas | Nevada | 89109 | — |
| Joseph M. Sanzari Children's Hospital- Site Number : 840101 | Hackensack | New Jersey | 07601 | — |
| Saint Peter's University Hospital - New Brunswick- Site Number : 840016 | New Brunswick | New Jersey | 08901 | — |
| Northwell Health - Site Number : 840102 | Manhasset | New York | 11030 | — |
| New York University School Of Medicine- Site Number : 840040 | New York | New York | 10016 | — |
| State University of New York- Site Number : 840052 | Syracuse | New York | 13210 | — |
| Duke University Medical Center Genetics Dept- Site Number : 840037 | Durham | North Carolina | 27710 | — |
| Cincinnati Children's Hospital-Genetics- Site Number : 840041 | Cincinnati | Ohio | — | — |
| Nationwide Children's Hospital- Site Number : 840091 | Columbus | Ohio | 43205 | — |
| Oregon Health & Science University (OHSU)- Site Number : 840080 | Portland | Oregon | 97239 | — |
| Children's Hospital of Philadelphia HUP Medical Genetics- Site Number : 840089 | Philadelphia | Pennsylvania | 19104 | — |
| Children's Hospital of Philadelphia- Site Number : 840034 | Philadelphia | Pennsylvania | — | — |
| University of Pittsburgh- Site Number : 840023 | Pittsburgh | Pennsylvania | 15224 | — |
| Rhode Island Hospital Genetics- Site Number : 840053 | Providence | Rhode Island | 02903 | — |
| Greenwood Genetic Center - Greenville- Site Number : 840055 | Greenville | South Carolina | 29605 | — |
| Baylor Institute of Metabolic Disease- Site Number : 840058 | Dallas | Texas | 75226 | — |
| University of Utah Health Hospital- Site Number : 840092 | Salt Lake City | Utah | 84132 | — |
| University of Virginia School of Medicine-Pediatrics- Site Number : 840078 | Charlottesville | Virginia | 22908 | — |
| Children's Hospital of The King's Daughters- Site Number : 840072 | Norfolk | Virginia | 23507 | — |
| Seattle Children's Hospital- Site Number : 840028 | Seattle | Washington | 98105 | — |
| University Of Washington Medical Center- Site Number : 840059 | Seattle | Washington | 98195 | — |
| Children's Hospital of Wisconsin-Pediatrics- Site Number : 840054 | Milwaukee | Wisconsin | 53226 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 88 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT00144794, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Feb 26, 2026 · Synced Sep 10, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete protocol, eligibility criteria, and contact information for NCT00144794 live on ClinicalTrials.gov.