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

• Register: EUCTR
• Last refreshed on: 19 March 2012
• Main ID: EUCTR2004-002743-27-SE
• Date of registration: 17/03/2005
• Prospective Registration: Yes
• Primary sponsor: Shire Human Genetic Therapies INC.
• Public 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
• 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: 11/05/2005
• Target sample size: 96
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2004-002743-27
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: no Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no

Countries of recruitment

Germany, Italy, Spain, Sweden, United Kingdom

Key inclusion & exclusion criteria

Inclusion criteria:
1. Patient must have completed the double-blind phase of Study TKT024, defined as completing the week 53 final evaluations of the study.

2. Patient, patient's parent(s), or legally authorized representative must have voluntarily signed an Institutional Review Board (IRB)/Independant 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.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1. Patient has received treatment with an investigational therapy other than the study drug in Study TKT024 within the past 60 days.

2. 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.

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

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


Age minimum:
Age maximum:
Gender:
Female: no
Male: yes

Health Condition(s) or Problem(s) studied

Mucopolysaccharidosis Type II (MPS II or Hunter Syndrome)
MedDRA version: 6.1 Level: PT Classification code 10056889

Intervention(s)

Product Name: Idursulfase (I2S)
Pharmaceutical Form: Concentrate for solution for infusion
Current Sponsor code: DRX006A
Other descriptive name: Idursulfase
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 2-

Primary Outcome(s)

Primary end point(s): Forced vital capacity and the six-minute walk test were the primary efficacy endpoints in the double-blind phase of Study TKT024. These variables will continue to be the primary clinical outcomes investigated in the open-label extension of Study TKT024.

Main Objective: The primary objective of this open-label extension study is to collect long-term safety and clinical outcome data in patients with Mucopolysaccharidosis Type II (MPS II or Hunter Syndrome) who are receiving idursulfase (DRX006A) enzyme replacement therapy.

Secondary Objective: The secondary objective of the study is to collect safety data on idursulfase manufactured at commercial-scale.

Secondary ID(s)

TKT024EXT

2004-002743-27-DE