Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase

Completed · Phase 2/Phase 3

Conditions studied: Hunter Syndrome, Mucopolysaccharidosis II (MPS II)

In brief

Study TKT024EXT was a long-term, single-arm, open-label extension of Study TKT024, a one year Phase 2/Phase 3 registration study. The primary objective of this extension study was to collect long-term safety and clinical outcome data in Mucopolysaccharidosis II (MPS II), also known as Hunter Syndrome, from the Phase 2/Phase 3 Study TKT024. All patients enrolling into this study received weekly active treatment with idursulfase, the primary dosing regimen investigated in Study TKT024. Hunter Syndrome is an X-linked recessive lysosomal storage disease caused by a deficiency of iduronate-2-sulfatase, an enzyme required to catabolize glycosaminoglycans (GAGS) in cells. As a result, GAGs accumulate in the lysosomes leading to cellular engorgement, organomegaly, tissue destruction, and organ system dysfunction. Hunter Syndrome is a rare disease with an estimated incidence of 1 in 162,000 live births.

Key facts

Study ID: NCT00630747
Run by: Shire
People needed: 94
Starts: 2004-09-13
Expected to finish: 2008-01-31
Last updated by the study team: 2021-06-10

Who can join

Age: 5 and older. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

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.

You may not qualify if…

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.

Where it is running

Pediatric Clinical Research Center, Children's Hospital Oakland — Oakland, California, United States
The Children's Hospital — Denver, Colorado, United States
Harbin Clinic — Rome, Georgia, United States
Mid-Illinois Hematology and Oncology Associates — Normal, Illinois, United States
University of Iowa Hospitals and Clinics — Iowa City, Iowa, United States
Children's Hospital Boston — Boston, Massachusetts, United States
Saint Louis University Cardinal Glennon Children's Hospital — St Louis, Missouri, United States
University of Nebraska Medical Center — Omaha, Nebraska, United States
Comprehensive Cancer Centers of Nevada — Las Vegas, Nevada, United States
Upstate Medical University, State University of New York (SUNY) — Syracuse, New York, United States
University of North Carolina at Chapel Hill — Chapel Hill, North Carolina, United States
Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
Baylor College of Medicine Texas Children's Hospital — Houston, Texas, United States
University of Utah Hospital — Salt Lake City, Utah, United States
Franciscan Skemp Healthcare — La Crosse, Wisconsin, United States
Fundacao Universidade de Ciencias da Saude de Alagoas Governador Lamenha Filho / UNCISAL — Maceió, Alagoas, Brazil
Clinica Casa de Saude Sao Joao — Barreiras, Estado de Bahia, Brazil
c-HUPES/UFBA — Salvador, Estado de Bahia, Brazil
Hospital Universitario da Faculdade de Medicina da Universidade Federal de Mato Grosso do Sul — Campo Grande, Mato Grosso do Sul, Brazil
Instituto de Puericultura e Pediatria Martagao Gesteira / Hospital Pediatrico — Rio de Janeiro, Rio de Janeiro, Brazil
Hospital de Clinicas de Porto Alegre, Servico de Genetica Medica — Porto Alegre, Rio Grande do Sul, Brazil
UNIFESP Instituto de Oncologia Pediatrica — São Paulo, São Paulo, Brazil
Instituto da Crianca / Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo — São Paulo, São Paulo, Brazil
The Hospital for Sick Children Research Institute — Toronto, Ontario, Canada
St. Joseph's Hospital — Phoenix, Arizona, United States

Full record on ClinicalTrials.gov

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