Status:
COMPLETED
Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
Lead Sponsor:
Shire
Conditions:
Metachromatic Leukodystrophy (MLD)
Eligibility:
All Genders
1-5 years
Phase:
PHASE1
Brief Summary
Objectives: The overall objective is to evaluate the safety, efficacy and pharmacokinetics (PK) of rhASA treatment in patients with late infantile MLD. Methodology: This is a single center, open-labe...
Detailed Description
Test product, dose, mode of administration, batch No.: The lowest dose level will be evaluated as a single dose of 25 U/kg. The three upper dose levels will be evaluated as repeated doses. Patients in...
Eligibility Criteria
Inclusion
- 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).
- The patient must have a confirmed diagnosis of MLD as defined by:
- ASA activity \< 10 nmol/h/mg in leukocytes Presence of elevated sulfatide in urine
- The patient must have a confirmed nerve conduction velocity \< 2 standard deviations (from the appropriate age level)
- The patient must have a residual level of voluntary function (as judged by the investigator), including presence of residual cognitive function (attention, executive and visual functions) as well as the presence of residual voluntary motor function in one upper or lower limb as a minimum.
- The patient must have an age at the time of screening ≥ 1 year and \< 6 years
- The patient must have had onset of symptoms before the age of 4 years
- The subject and his/her guardian(s) must have the ability to comply with the clinical protocol
- The patients' medical record must document that the legal guardian(s) has had independent counselling or a consultation regarding stem cell transplantation in order to assure that the guardian(s) is fully informed regarding the risks and benefits of this alternative
Exclusion
- Patients will be excluded from this study if they do not meet the specific inclusion criteria, or if any of the following criteria apply:
- Lack of voluntary function
- Presence of severe pseudo-bulbar signs (weakness and disco-ordination of tongue and swallowing muscles leading to severe difficulty with swallowing)
- Spasticity so severe to inhibit transportation
- Known multiple sulfatase deficiency
- Presence of major congenital abnormality
- Presence of known chromosomal abnormality and syndromes affecting psychomotor development
- History of stem cell transplantation
- Presence of known clinically significant cardiovascular, hepatic, pulmonary or renal disease or other medical condition
- Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial
- Use of any investigational product within 30 days prior to study enrolment or currently enrolled in another study which involves clinical investigations
- Received ERT with rhASA from any source
- Planned or anticipated initiation of antispastic treatment after trial initiation
Key Trial Info
Start Date :
January 22 2007
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 27 2008
Estimated Enrollment :
13 Patients enrolled
Trial Details
Trial ID
NCT00418561
Start Date
January 22 2007
End Date
March 27 2008
Last Update
June 25 2021
Active Locations (1)
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1
Rigshospitalet
Hvidovre, Denmark, DK-2650