Mucopolysaccharidosis I (MPS I) Clinical Trial
Official title:
Mucopolysaccharidosis I (MPS I) Registry
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
| Status | Recruiting |
| Enrollment | 1500 |
| Est. completion date | January 31, 2034 |
| Est. primary completion date | January 31, 2034 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | N/A and older |
| Eligibility | 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 Exclusion Criteria: - No exclusion criteria for participation in the MPS I Registry. NOTE: Registry participation does not exclude participation in other clinical studies. |
| Country | Name | City | State |
|---|---|---|---|
| Argentina | Investigational Site Number : 153130 | Buenos Aires | |
| Argentina | Investigational Site Number : 153032 | Ciudad Autónoma de Buenos Aires | Buenos Aires |
| Australia | Investigational Site Number : 152096 | Brisbane | Queensland |
| Australia | Investigational Site Number : 151069 | Westmead | New South Wales |
| Belgium | Investigational Site Number : 150944 | Brussel | |
| Belgium | Investigational Site Number : 150517 | Bruxelles | |
| Belgium | Investigational Site Number : 151668 | Leuven | |
| Belgium | Investigational Site Number : 154008 | Roeselare | |
| Brazil | PUC Campinas - Sociedade Campineira de Educaçao e Instruçao Site Number : 076007 | Campinas | São Paulo |
| Brazil | Centro de Pesquisa Clínica Prédio 21, 2o andar Hospital de Clínicas de Porto Alegre Site Number : 076001 | Porto Alegre | Rio Grande Do Sul |
| Brazil | Instituto Fernandes Figueira - FIOCRUZ Site Number : 076004 | Rio de Janeiro | |
| Brazil | Instituto de Genética e Erros Inatos do Metabolismo - IGEIM Site Number : 076002 | São Paulo | |
| Canada | Investigational Site Number : 124014 | Calgary | Alberta |
| Canada | Investigational Site Number : 124002 | Edmonton | Alberta |
| Canada | Investigational Site Number : 124021 | London | Ontario |
| Canada | Investigational Site Number : 124022 | Moncton | New Brunswick |
| Canada | Investigational Site Number : 124018 | Montreal | Quebec |
| Canada | Investigational Site Number : 124001 | Sherbrooke | Quebec |
| Canada | Investigational Site Number : 124006 | Toronto | Ontario |
| Canada | Investigational Site Number : 124011 | Vancouver | British Columbia |
| Canada | Investigational Site Number : 124019 | Winnipeg | Manitoba |
| Chile | Investigational Site Number : 152003 | Los Angeles | |
| Chile | Investigational Site Number : 152001 | Talcahuano | |
| Colombia | Investigational Site Number : 170001 | Bogotá | |
| Croatia | Investigational Site Number : 1910001 | Zagreb | |
| Czechia | Investigational Site Number : 150659 | Praha 2 | |
| Denmark | Investigational Site Number : 151229 | København Ø | |
| Egypt | Investigational Site Number : 818001 | Cairo | |
| Egypt | Investigational Site Number : 818002 | Cairo | |
| France | Investigational Site Number : 250004 | Bordeaux | |
| France | Investigational Site Number : 250001 | BRON Cedex | |
| France | Investigational Site Number : 250002 | Marseille Cedex 05 | |
| France | Investigational Site Number : 250003 | Paris | |
| France | Investigational Site Number : 250005 | Paris Cedex 12 | |
| France | Investigational Site Number : 250006 | Vandoeuvre Les Nancy Cedex | |
| Germany | Investigational Site Number : 151368 | Hamburg | |
| Germany | Investigational Site Number : 150031 | Mainz | |
| Hong Kong | Investigational Site Number : 344001 | Kowloon | |
| Hungary | Investigational Site Number : 3480005 | Pécs | |
| India | Investigational Site Number : 356003 | Ahmedabad | |
| India | Investigational Site Number : 356001 | Kolkata | |
| India | Investigational Site Number : 3560004 | Secunderabad | |
| India | Investigational Site Number : 356002 | Vellore | |
| Indonesia | Investigational Site Number : 360001 | Jakarta | |
| Ireland | Investigational Site Number : 154161 | Dublin | |
| Italy | Investigational Site Number : 380002 | Ancona | |
| Italy | Investigational Site Number : 380004 | Cusano Milanino | |
| Italy | Investigational Site Number : 380009 | Firenze | |
| Italy | Investigational Site Number : 380003 | Genova | |
| Italy | Investigational Site Number : 380007 | Milano | |
| Italy | Investigational Site Number : 380005 | Napoli | |
| Italy | Investigational Site Number : 380008 | Padova | |
| Italy | Investigational Site Number : 380006 | Roma | |
| Italy | Investigational Site Number : 380001 | Udine | |
| Japan | Investigational Site Number : 392102 | Nangoku-shi | Kochi |
| Japan | Investigational Site Number : 392101 | Setagaya | Tokyo |
| Korea, Republic of | Investigational Site Number : 154286 | Changwon-si | Gyeongsangnam-do |
| Korea, Republic of | Investigational Site Number : 153191 | Daejeon | |
| Korea, Republic of | Investigational Site Number : 153225 | Jeonnam | |
| Korea, Republic of | Investigational Site Number : 151149 | Seoul | |
| Korea, Republic of | Investigational Site Number : 153180 | Seoul | |
| Korea, Republic of | Investigational Site Number : 153192 | Seoul | |
| Korea, Republic of | Investigational Site Number : 153196 | Seoul | |
| Korea, Republic of | Investigational Site Number : 153212 | Seoul | |
| Korea, Republic of | Investigational Site Number : 153222 | Seoul | |
| Korea, Republic of | Investigational Site Number : 154148 | Yangsan | Gyeongsangnam-do |
| Korea, Republic of | Investigational Site Number : 153178 | Yangsan-si | Gyeongsangnam-do |
| Kuwait | Investigational Site Number : 154225 | Kuwait | |
| Lebanon | Investigational Site Number : 4220004 | Beirut | |
| Malaysia | Investigational Site Number : 458001 | Jalan Pahang | |
| Malaysia | Investigational Site Number : 458002 | Kuala Lumpur | |
| Netherlands | Investigational Site Number : 151171 | Amsterdam | |
| Netherlands | Investigational Site Number : 152029 | Rotterdam | |
| Pakistan | Investigational Site Number : 154264 | Lahore | |
| Pakistan | Investigational Site Number : 154266 | Lahore | |
| Philippines | Investigational Site Number : 608001 | Manila | |
| Poland | Investigational Site Number : 150575 | Warszawa | |
| Portugal | Investigational Site Number : 6200002 | Coimbra | |
| Romania | Investigational Site Number : 154177 | Cluj Napoca | |
| Russian Federation | Investigational Site Number : 643001 | Moscow | |
| Russian Federation | Investigational Site Number : 643002 | Moscow | |
| Saudi Arabia | Investigational Site Number : 682004 | Makkah | |
| Saudi Arabia | Investigational Site Number : 682001 | Qatif | |
| Singapore | Investigational Site Number : 702001 | Singapore | |
| Slovakia | Investigational Site Number : 703001 | Bratislava | |
| Sweden | Investigational Site Number : 7520001 | Stockholm | |
| Taiwan | Investigational Site Number : 154093 | Chiayi | |
| Taiwan | Investigational Site Number : 152152 | Hualien | |
| Taiwan | Investigational Site Number : 152244 | Kaohsiung | |
| Taiwan | Investigational Site Number : 152246 | Kaohsiung Hsien, | |
| Taiwan | Investigational Site Number : 151534 | New Taipei City | |
| Taiwan | Investigational Site Number : 151500 | Putz City, Chia-yi | |
| Taiwan | Investigational Site Number : 151181 | Taichung | |
| Taiwan | Investigational Site Number : 151179 | Taichung City | |
| Taiwan | Investigational Site Number : 152153 | Tainan | |
| Taiwan | Investigational Site Number : 153248 | Tainan | |
| Taiwan | Investigational Site Number : 151182 | Taipei | |
| Taiwan | Investigational Site Number : 152321 | Taipei | |
| Taiwan | Investigational Site Number : 153158 | Tao Yuan County | |
| Thailand | Investigational Site Number : 764001 | Bangkok | |
| Thailand | Investigational Site Number : 764002 | Bangkok | |
| Thailand | Investigational Site Number : 764003 | Bangkok | |
| Thailand | Investigational Site Number : 764004 | Bangkok | |
| Thailand | Investigational Site Number : 764005 | Khon Kaen | |
| United Kingdom | Investigational Site Number : 152287 | Birmingham | |
| United Kingdom | Investigational Site Number : 154240 | Birmingham | |
| United Kingdom | Investigational Site Number : 152156 | Glasgow | |
| United Kingdom | Investigational Site Number : 150303 | London | |
| United Kingdom | Investigational Site Number : 151123 | London | |
| United Kingdom | Investigational Site Number : 151646 | London | |
| United Kingdom | Investigational Site Number : 150455 | Manchester | |
| United Kingdom | Investigational Site Number : 152361 | Salford | |
| United States | University of Michigan Pediatrics Site Number : 840107 | Ann Arbor | Michigan |
| United States | Children's Hospital IMD Clinic Site Number : 840069 | Aurora | Colorado |
| United States | University of Colorado at Denver Genetics Site Number : 840068 | Aurora | Colorado |
| United States | John Hopkins Site Number : 840044 | Baltimore | Maryland |
| United States | Boston Children's Hospital Site Number : 840093 | Boston | Massachusetts |
| United States | Massachusetts General Hospital-Genetics Site Number : 840062 | Boston | Massachusetts |
| United States | University of Virginia School of Medicine-Pediatrics Site Number : 840078 | Charlottesville | Virginia |
| United States | Ann and Robert Lurie Children's Hospital Site Number : 840011 | Chicago | Illinois |
| United States | Rush University Medical Center Genetics Site Number : 840079 | Chicago | Illinois |
| United States | Cincinnati Children's Hospital-Genetics Site Number : 840041 | Cincinnati | Ohio |
| United States | UHCMC Site Number : 840119 | Cleveland | Ohio |
| United States | Nationwide Children's Hospital Site Number : 840091 | Columbus | Ohio |
| United States | Baylor Research Center Site Number : 840058 | Dallas | Texas |
| United States | Emory University School of Medicine- Human Genetics Site Number : 840060 | Decatur | Georgia |
| United States | Children's Hospital of Michigan Site Number : 840066 | Detroit | Michigan |
| United States | LSD Data Registry Site LLC Site Number : 840094 | Dublin | Ohio |
| United States | Duke University Medical Center Genetics Dept Site Number : 840037 | Durham | North Carolina |
| United States | University of Florida Dept of Genetics Site Number : 840083 | Gainesville | Florida |
| United States | Greenwood Genetcs Site Number : 840055 | Greenville | South Carolina |
| United States | Joseph M. Sanzari Children's Hospital Site Number : 840101 | Hackensack | New Jersey |
| United States | Indianapolis University School of Medicine Site Number : 840027 | Indianapolis | Indiana |
| United States | University of Iowa Site Number : 840032 | Iowa City | Iowa |
| United States | The Atwal Clinic Site Number : 840112 | Jacksonville | Florida |
| United States | University of Florida Pediatrics Genetics Site Number : 840121 | Jacksonville | Florida |
| United States | Children's Specialty Center of Nevada Site Number : 840008 | Las Vegas | Nevada |
| United States | Arkansas Childrens Hospital Site Number : 840109 | Little Rock | Arkansas |
| United States | Southern California Permanente Medical Group Site Number : 840108 | Los Angeles | California |
| United States | USC Health Sciences Center Dept of Genetics Site Number : 840082 | Los Angeles | California |
| United States | University of Louisville- Genetics Site Number : 840030 | Louisville | Kentucky |
| United States | Northwell Health Site Number : 840102 | Manhasset | New York |
| United States | University Of Miami SOM Site Number : 840006 | Miami | Florida |
| United States | Children's Hospital of Wisconsin-Pediatrics Site Number : 840054 | Milwaukee | Wisconsin |
| United States | Children's Health Care Site Number : 840114 | Minneapolis | Minnesota |
| United States | University of Minnesota Medical Center Pediatrics Site Number : 840076 | Minneapolis | Minnesota |
| United States | Atlantic Health System Site Number : 840099 | Morristown | New Jersey |
| United States | St. Peter's University Hospital Site Number : 840016 | New Brunswick | New Jersey |
| United States | Yale Site Number : 840047 | New Haven | Connecticut |
| United States | Ochsner Medical Center Site Number : 840120 | New Orleans | Louisiana |
| United States | Metropolitan Hospital Center Site Number : 840110 | New York | New York |
| United States | New York University School Of Medicine Site Number : 840040 | New York | New York |
| United States | Children's Hospital of the Kings Daughters Site Number : 840072 | Norfolk | Virginia |
| United States | Children's Hospital Oakland Site Number : 840029 | Oakland | California |
| United States | University of Nebraska Medical Center- Pediatrics Site Number : 840084 | Omaha | Nebraska |
| United States | Children's Hospital of Orange County Site Number : 840074 | Orange | California |
| United States | Stanford Unviersity MC Dept of Genetics Site Number : 840022 | Palo Alto | California |
| United States | St. Joseph's Children's Hospital Site Number : 840057 | Paterson | New Jersey |
| United States | Children's Hospital of Philadelphia HUP Medical Genetics Site Number : 840089 | Philadelphia | Pennsylvania |
| United States | Children's Hospital of Philadelphia Site Number : 840034 | Philadelphia | Pennsylvania |
| United States | Phoenix Children's Hospital Site Number : 840003 | Phoenix | Arizona |
| United States | University of Pittsburgh Site Number : 840023 | Pittsburgh | Pennsylvania |
| United States | Oregon Health & Science University Site Number : 840080 | Portland | Oregon |
| United States | Rhode Island Hospital Genetics Site Number : 840053 | Providence | Rhode Island |
| United States | University of Rochester Medical Center SOM Site Number : 840105 | Rochester | New York |
| United States | UC Davis MIND Institute Site Number : 840010 | Sacramento | California |
| United States | Washington University of St. Louis Site Number : 840100 | Saint Louis | Missouri |
| United States | University of Utah Medical Center Site Number : 840092 | Salt Lake City | Utah |
| United States | Loma Linda University Children's Hospital Site Number : 840117 | San Bernardino | California |
| United States | University of California at San Diego Site Number : 840007 | San Diego | California |
| United States | University of California at San Francisco Site Number : 840051 | San Francisco | California |
| United States | Seattle Children's Hospital Site Number : 840028 | Seattle | Washington |
| United States | University Of Washington Medical Center Site Number : 840059 | Seattle | Washington |
| United States | Baystate Health Site Number : 840002 | Springfield | Massachusetts |
| United States | State University of New York Site Number : 840052 | Syracuse | New York |
| United States | Multicare Health System ¿ Mary Bridge Childrens Hospital Site Number : 840115 | Tacoma | Washington |
| United States | Pediatric Endocrine Associates Site Number : 840017 | Tampa | Florida |
| United States | Children's National Medical Center Site Number : 840067 | Washington | District of Columbia |
| Vietnam | Investigational Site Number : 154212 | Hanoi | |
| Vietnam | Investigational Site Number : 704002 | Ho Chi Minh City |
| Lead Sponsor | Collaborator |
|---|---|
| Genzyme, a Sanofi Company |
United States, Vietnam, Argentina, Australia, Belgium, Brazil, Canada, Chile, Colombia, Croatia, Czechia, Denmark, Egypt, France, Germany, Hong Kong, Hungary, India, Indonesia, Ireland, Italy, Japan, Korea, Republic of, Kuwait, Lebanon, Malaysia, Netherlands, Pakistan, Philippines, Poland, Portugal, Romania, Russian Federation, Saudi Arabia, Singapore, Slovakia, Sweden, Taiwan, Thailand, United Kingdom,
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Beck M, Arn P, Giugliani R, Muenzer J, Okuyama T, Taylor J, Fallet S. The natural history of MPS I: global perspectives from the MPS I Registry. Genet Med. 2014 Oct;16(10):759-65. doi: 10.1038/gim.2014.25. Epub 2014 Mar 27. — View Citation
Viskochil D, Muenzer J, Guffon N, Garin C, Munoz-Rojas MV, Moy KA, Hutchinson DT. Carpal tunnel syndrome in mucopolysaccharidosis I: a registry-based cohort study. Dev Med Child Neurol. 2017 Dec;59(12):1269-1275. doi: 10.1111/dmcn.13545. Epub 2017 Sep 11. — View Citation
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | To evaluate the long-term effectiveness of Aldurazyme | Approximately 17 Years |