Efficacy METAZYM for the Treatment Metachromatic Leukodystrophy Treated With Hematopoietic Stem Cell Transplantation

• Status: Completed
• Trial end date: 2010-04-01
• Target enrollment: 0
• Participant gender: All

Summary

There is currently no effective treatment for late infantile MLD once clinical symptoms are evident. METAZYM is a recombinant human arylsulfatase A developed for an intravenous ERT for the treatment of late infantile MLD. The overall objective of this study is to evaluate the efficacy and safety of intravenous rhASA treatment in a patient with late infantile MLD who had previously received hematopoietic stem cell transplantation (HCT).

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Assistance Publique - Hôpitaux de Paris
• Collaborators: European Leukodystrophy Association; Shire; Zymenex A/S

Criteria

Inclusion Criteria:

• Subject's legally authorized guardian(s) must provide signed, informed consent prior to performing any study-related activities.

• The patient must have a confirmed diagnosis of MLD as defined by:ARSA activity < 10 nmol/h/mg in leukocytes prior to HCT; Presence of elevated sulfatide in urine prior to HCT

• 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 ≥ 6 months

• 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 Criteria:

Patient will be excluded from this study if they do not meet the specific inclusion criteria, or if any of the following criteria apply:

• Presence of a gross motor function measure (GMFM < 25)

• 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

• 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