A single center, open-label, non-randomized, uncontrolled, multiple-dose study of the efficacy and long-term safety of Metazym (recombinant human arylsulfatase A or rhASA) for the treatment of patients with late infantile metachromatic leukodystrophy
- Conditions
- Metachromatic Leukodystrophy (MLD) in late infantile patientsMedDRA version: 9.1Level: LLTClassification code 10024381Term: Leukodystrophy
- Registration Number
- EUCTR2007-006345-40-DK
- Lead Sponsor
- Shire Pharmaceutical Ireland Limited
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- Not specified
The patients from the phase I trial (EudraCT number: 2006-005341-11) must meet the following criteria to be enrolled in the study:
1. Subject’s legally authorized guardian(s) must provide signed, informed consent prior to performing any study-related activities (trial-related activities are any procedures that would not have been performed during normal management of the subject)
2. The subject and his/her guardian(s) must have the ability to comply with the clinical protocol
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) no
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
Patients will be excluded from this study if they do not meet the specific inclusion criteria, or if any of the following criteria apply:
1. Spasticity so severe to inhibit transportation
2. Presence of known clinically significant cardiovascular, hepatic, pulmonary or renal disease or other medical condition that, in the opinion of the Investigator, would preclude participation in the trial
3. Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial
4. Use of any investigational product other than rhASA within 30 days prior to study enrolment or currently enrolled in another study which involves clinical investigations
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method