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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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