Dettagli per singolo studio

DEC-NET Serial number ES531

Published online	06/04/2006 13.53.00
Last updated	24/04/2006 9.11.44

Other protocol ID number	TKT024EXT
Current trial status	Closed to recruitment of participants: follow-up continuing
Major Disease (ICD9 class)	MUCOPOLYSACCHARIDOSIS
Experimental drug
Idursulfase Treatment regimen (dosage and duration) idursulfase 0.5 mg/kg weekly for 2 years
Idursulfase Treatment regimen (dosage and duration) idursulfase 0.5 mg/kg weekly for 2 years
Gender	Male
Age (range)	Children (2-11 years) to Adults (18-65 years)
Eligibility criteria
Inclusion criteria
Patient must have completed the double-blind phase of Study TKT024, defined as completing the Week 53 final evaulations of the study. Patient, patient's parent(s), or legally authorized representaive 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, according to the local study site requirements.
Exclusion criteria
Patient has received treatment with an investigational therapy other than the study drug 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 expereinced an adverse reaction to the study drug in Study TKT024, which contraindicates further treatment with idursulfase. Patient with known hypersensitivity to any of the components of idursulfase
Trial design/methodology
Phase	3
Kind of study	Efficacy Safety Pharmacokinetics
Design	Abierto
Purpose of study
The primary objective of this open label extension study is to collect long-term safety and clinical outcome data in patients with Mucopolysaccharidosis II (MPS II or Hunter syndrome) who are receiving idursulfase (DRX006A) enzyme replacement therapy. The secondary objective of the study is to collect safety data on idursulfase manufactured at commercial scale
Primary outcomes
Safety of iduronate-2-sulfatase in male patients with Hunter syndrome
Secondary outcomes
Clinical outcomes (Efficacy)
Summary of study design, objectives, and ongoing research findings
This is an open label extension study. To qualify, patients will have completed the double-blind, placebo-controlled phase II/III study, TKT024. Patients eligible to continue receving treatment with iduronate-2-sulfatase will receive 0.5 mg/kg infusions each week for two years. The primary endpoint of this study will be an assessment of safety.