Metachromatic Leukodystrophy (MLD) in late infantile patients MedDRA version: 9.1 Level: LLT Classification code 10024381 Term: Leukodystrophy
Conditions
Interventions
Product Name: Metazym
Product Code: rhASA
Pharmaceutical Form: Injection*
Current Sponsor code: rhASA
Other descriptive name: recombinant human arylsulfatase A
Concentration unit: U unit(s)
Concentrat
Sponsors
Shire Pharmaceuticals Ireland Limited
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Patients 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 patient must have a confirmed diagnosis of MLD as defined by: · ASA activity =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 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. Known multiple sulfatase deficiency 3. Presence of major congenital abnormality 4. Presence of known chromosomal abnormality and syndromes affecting psychomotor development 5. History of stem cell transplantation 6. 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 7. Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial 8. 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The overall objective is to evaluate efficacy and safety of rhASA treatment in patients with late infantile MLD and high residual level of voluntary function. Determination of the optimal dose will be elucidated;Secondary Objective: Change in CSF biomarkers ;Primary end point(s): Relative change in GMFM after 26 weeks of treatment Absolute change in Mullen’s Scale of Early Learning after 26 weeks of treatment. | — |
Countries
Denmark
Outcome results
None listed