Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD) (NCT00418561) | Clinical Trial Compass
CompletedPhase 1
Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
Denmark13 participantsStarted 2007-01-22
Plain-language 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-label study of patients with late infantile MLD. Twelve patients will be enrolled in this study receiving a total of thirteen intravenous infusions of Metazym. One infusion will be given every other week for a period of half a year. After the half year the subjects will continue treatment every other week until safety data is available. Safety (AE/SAE) will be monitored at every visit during this period.
Who can participate
Age range
1 Year – 5 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. 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:
. 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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Timeframe: From study drug administration up to Week 28
2
Change From Baseline in Gross Motor Function Measure (GMFM) at Week 26
Timeframe: Baseline, Week 26
3
Change From Baseline in Cerebrospinal Fluid (CSF) Sulfatide at Week 26
Timeframe: Baseline, Week 26
4
Number of Participants With Shift From Baseline to Week 26 in Sulfatide Levels in Urine
Timeframe: Baseline up to Week 26
5
Change From Baseline in Mullen's Scales of Early Learning at Week 26
Timeframe: Baseline, Week 26
6
Maximum Plasma Drug Concentration (Cmax) of Recombinant Human Arylsulphatase A (rhASA)
Timeframe: Pre-dose and post-dose at 20, 40, 90 minutes, 3, 6 and 8 hours on Day 0, 40 minutes post-dose at Week 4, Pre-dose and post-dose at 20, 40, 90 minutes, 3, 6 and 8 hours at Week 8
. 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
. 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
Arylsulfatase A (ASA) Activity in Leukocytes
Timeframe: Pre-dose and post-dose at 24 hours on Day 0 and at Weeks 8 and 26