Efficacy METAZYM for the Treatment Metachromatic Leukodystrophy Treated With Hematopoietic Stem Cell Transplantation (Azylis)
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ClinicalTrials.gov Identifier: NCT01303146 |
Recruitment Status
:
Completed
First Posted
: February 24, 2011
Last Update Posted
: February 24, 2011
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Metachromatic Leukodystrophy | Drug: rhARSA | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 1 participants |
Allocation: | Non-Randomized |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | and Safety of METAZYM (Recombinant Human Arylsulfatase A or rhASA) for the Treatment of Patients With Late Infantile MLD Who Had Previously Hematopoietic Stem Cell Transplantation |
Study Start Date : | October 2008 |
Actual Primary Completion Date : | March 2010 |
Actual Study Completion Date : | April 2010 |

Arm | Intervention/treatment |
---|---|
Experimental: Enzyme replacement therapy
intravenous infusion 100U/kg every other week for 18 months
|
Drug: rhARSA
intravenous infusion 100U/kg every other week for 18 months
Other Names:
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- Efficacy of METAZYM on peripheral nerve function by electrophysiological studies (motor and sensory nerves conduction velocities) every 6 months; [ Time Frame: every 6 months ]
- Efficacy of METAZYM on peripheral nerve sulfatide storage and demyelination by nerve biopsy at baseline and week 26; [ Time Frame: week 26 ]
- Efficacy of METAZYM on functional capacity by assessing motor function (GMFM) every 6 months [ Time Frame: every 6 months ]
- Efficacy of METAZYM on central nervous system involvement by evaluation of cognitive and neurological function, somatosensory and auditory evoked potentials, and brain MRI every 6 months; [ Time Frame: every 6 months ]
- Safety profile of METAZYM by monitoring AE's, vital sign parameters and physical examination findings before each injection, as well as ECGs and routine clinical laboratory tests every 3 months. [ Time Frame: every 3 months ]

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Ages Eligible for Study: | 6 Months and older (Child, Adult, Senior) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01303146
France | |
Department of Pediatric Endocrinology and Neurology, Saint Vincent de Paul Hospital | |
Paris, France, 75014 |
Study Chair: | Patrick Aubourg, MD, PhD | Department of Pediatric Endocrinology and Neurology, Saint Vincent de Paul Hospital, Paris |
Responsible Party: | VACHER Yannick, Department of Clinical Research of developpement |
ClinicalTrials.gov Identifier: | NCT01303146 History of Changes |
Other Study ID Numbers: |
P070805 |
First Posted: | February 24, 2011 Key Record Dates |
Last Update Posted: | February 24, 2011 |
Last Verified: | February 2011 |
Keywords provided by Assistance Publique - Hôpitaux de Paris:
Metachromatic Leukodystrophy Enzyme replacement therapy Allogeneic hematopoietic stem cell transplantation |
Additional relevant MeSH terms:
Leukodystrophy, Metachromatic Hereditary Central Nervous System Demyelinating Diseases Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases Sulfatidosis Sphingolipidoses Lysosomal Storage Diseases, Nervous System |
Leukoencephalopathies Demyelinating Diseases Metabolism, Inborn Errors Genetic Diseases, Inborn Lipidoses Lipid Metabolism, Inborn Errors Lysosomal Storage Diseases Metabolic Diseases Lipid Metabolism Disorders |