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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 19 October 2017
Main ID:  NCT00630747
Date of registration: 28/02/2008
Prospective Registration: No
Primary sponsor: Shire
Public title: Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase
Scientific title: An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement Therapy
Date of first enrolment: September 2004
Target sample size: 94
Recruitment status: Completed
URL:  https://clinicaltrials.gov/show/NCT00630747
Study type:  Interventional
Study design:   
Phase:  Phase 2/Phase 3
Countries of recruitment
Brazil Canada France Germany Italy Romania Spain Sweden
United Kingdom United States
Contacts
Name:     Angelina Acosta, MD
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Affiliation:  c-HUPES/UFBA
Name:     Uma Ramaswami, MD
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Affiliation:  Cambridge University Hospitals NHS Foundation Trust
Name:     Ronald Kline, MD
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Affiliation:  Comprehensive Cancer Centers of Nevada
Name:     Mal Ratnayaka, MD
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Affiliation:  Derbyshire Children's Hospital
Name:     Ashok Vellodi, MD
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Affiliation:  Great Ormond Street Hospital for Sick Children
Name:     Francis Gaches, MD
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Affiliation:  Hospital Ducuing
Name:     Gunilla Malm, MD
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Affiliation:  Karolinska University Hospital
Name:     Peter H. Robinson, MD
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Affiliation:  Royal Hospital for Sick Children
Name:     Pilar Munguira Aguado, MD
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Affiliation:  Servicio de Pediatria
Name:     Bjorn Hoffmann, MD
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Affiliation:  Universitatsklinik Dusseldorf Kinderklinik
Name:     Rolf Mertens, MD
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Affiliation:  Universitatsklinikum Aachen Kinderklinik
Name:     Sara Copeland, MD
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Affiliation:  University of Iowa
Name:     Dave Viskochil, MD
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Affiliation:  University of Utah Hospital
Name:     J. Edmond Wraith, MRCP, FRCP
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Affiliation:  Royal Manchester Children's Hospital
Name:     Stuart Murray, MD
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Affiliation:  Bath and NE Somerset Primary Care Trust
Name:     Paul Uvebrant, MD
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Affiliation:  Drottning Silvias Barnsjukhus
Name:     Nathalie Guffon, MD
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Affiliation:  Hopital Edouard Herriot
Name:     Joachim Kreuder, MD
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Affiliation:  Justus-Liebig Universitat
Name:     Antonia Patrocinio Leon Asensio, MD
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Affiliation:  Servicio de Pediatria
Name:     Ana Maria Martins, MD
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Affiliation:  UNIFESP Instituto de Oncologia Pediatrica
Name:     Emmanuelle Lemyre, MD
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Affiliation:  University of Montreal / Hopital Ste-Justine
Name:     Joseph Muenzer, MD, PhD
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Affiliation:  University of North Carolina, Chapel Hill
Name:     Christine Eng, MD
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Affiliation:  Baylor College of Medicine Texas Children's Hospital
Name:     Paul Harmatz, MD
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Affiliation:  Pediatric Clinical Research Center, Children's Hospital Oakland
Name:     Rick Martin, MD
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Affiliation:  Saint Louis University Cardinal Glennon Children's Hospital
Name:     Generoso Andria, MD
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Affiliation:  Universita degli Studi di Napoli Federico II
Name:     Kurt Ullrich, MD
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Affiliation:  Universitatsklinikum Hamburg Eppendorf
Name:     Edward Neilan, MD
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Affiliation:  Boston Children’s Hospital
Name:     Emerson Santos, MD
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Affiliation:  Fundacao Universidade de Ciencias da Saude de Alagoas Governador Lamenha Filho / UNCISAL
Name:     Dilip Patel, MD
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Affiliation:  Harbin Clinic
Name:     Paula Grigorescu Sido, MD
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Affiliation:  Spitalul Clinic de Copii
Name:     Chong Kim, MD
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Affiliation:  Instituto da Crianca / Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo
Name:     Marcia Ribeiro, MD
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Affiliation:  Instituto de Puericultura e Pediatria Martagao Gesteira / Hospital Pediatrico
Name:     Michael Ryalls, MD
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Affiliation:  Royal Surrey County Hospital
Name:     Kirk Aleck, MD
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Affiliation:  St. Joseph's Hospital
Name:     Maurizio Scarpa, MD
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Affiliation:  Universita di Padova
Name:     Robert Steinfeld, MD, PhD
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Affiliation:  Universitatsklinikum Gottingen
Name:     Roberto Garcia De Lima, MD
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Affiliation:  Clinica Casa de Saude Sao Joao
Name:     Roberto Giugliani, MD
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Affiliation:  Hospital de Clinicas de Porto Alegre, Servico de Genetica Medica
Name:     Luigi Besenzon, MD
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Affiliation:  Ospedale S. S. Annunziata
Name:     Janet Thomas, MD
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Affiliation:  The Children's Hospital
Name:     Joe Clarke, MD
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Affiliation:  The Hospital for Sick Children Research Institute
Name:     Irene Cherrick, MD
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Affiliation:  Upstate Medical University, State University of New York (SUNY)
Name:     Paige Kaplan, MD
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Affiliation:  Children's Hospital of Philadelphia
Name:     Michael Beck, MD
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Affiliation:  Children's University Hospital Mainz AG
Name:     David Capelli, MD
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Affiliation:  Franciscan Skemp Healthcare
Name:     Michel Fischbach, MD
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Affiliation:  Hôpital de Hautepierre
Name:     Durval Batista Palhares, MD
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Affiliation:  Hospital Universitario da Faculdade de Medicina da Universidade Federal de Mato Grosso do Sul
Name:     Hwan Jeong, MD
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Affiliation:  Mid-Illinois Hematology and Oncology Associates
Name:     Nicola Leech, MD
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Affiliation:  Royal Victoria Infirmary
Name:     Rossella Parini, MD
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Affiliation:  Universita Milano Bicocca / Ospedale S. Gerardo
Name:     Guillem Pintos-Morell, MD
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Affiliation:  University Hospital Germans Trias i Pujol
Name:     G. Bradley Schaefer, MD
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Affiliation:  University of Nebraska
Key inclusion & exclusion criteria

Inclusion Criteria:

- Patient must have completed the double-blind phase of Study TKT024, defined as
completing the Week 53 final evaluations.

- Patient, patient's parent(s), or legally authorized representative must have
voluntarily signed an Institutional Review Board (IRB)/Independent Ethics Committee
(IEC)-approved informed consent form after all relevant aspects of the study have been
explained and discussed with the patient.

Exclusion Criteria:

- Patient has received treatment with an investigational therapy other than
iduronate-2-sulfatase in Study TKT024 within the past 60 days.

- Patient is unable to comply with the protocol (e.g., due to a medical condition such
as cervical cord compression or uncooperative attitude) or is unlikely to complete the
study, as determined by the investigator.

- Patient has experienced an adverse reaction to study drug in Study TKT024, which
contraindicates further treatment with idursulfase.

- Patient with known hypersensitivity to any of the components of idursulfase.



Age minimum: 5 Years
Age maximum: N/A
Gender: Male
Health Condition(s) or Problem(s) studied
Hunter Syndrome
Mucopolysaccharidosis II (MPS II)
Intervention(s)
Biological: Idursulfase
Primary Outcome(s)
Change From Baseline in Mean Distance Walked in the 6-minute Walk Test (6MWT) at Week 105 [Time Frame: Baseline and at Week 105]
Change From Baseline in Mean Percent Predicted Forced Vital Capacity (FVC) at Week 105 [Time Frame: Baseline and at Week 105]
Secondary Outcome(s)
Change From Baseline in Mean Normalized Urine Glycosaminoglycans (GAG) Levels at Week 105 [Time Frame: Baseline and at Week 105]
Change From Baseline in Mean Passive Joint Range of Motion (JROM) at Week 105 [Time Frame: Baseline and at Week 105]
Change From Baseline in Mean Combined Liver and Spleen Volume at Week 105 [Time Frame: Baseline and at Week 105]
Change From Baseline in Mean Cardiac Left Ventricular Mass Index (LVMI) at Week 105 [Time Frame: Baseline and at Week 105]
Secondary ID(s)
2004-002743-27
TKT024EXT
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results
Results available: Yes
Date Posted: 17/03/2014
Date Completed:
URL: https://clinicaltrials.gov/ct2/show/results/NCT00630747
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