The Natural History of Metachromatic Leukodystrophy (NCT00639132) | Clinical Trial Compass
WithdrawnNot Applicable
The Natural History of Metachromatic Leukodystrophy
Stopped: Merged with a broader study of rare diseases once moved to Pittsburgh
0Started 2008-03-11
Plain-language summary
There have not been longitudinal studies which track patients' neurologically or developmentally in a systematic manner. By simultaneously tracking patients' neurodevelopment along with neuroimaging and neurophysiologic studies it becomes much easier to draw conclusions on the differential effects of the disease process and any available treatments that patients might receive. In addition, many of the gene mutations, which cause MLD have not been linked to the age of onset or the expected disease course.
Who can participate
Age range6 Years
SexALL
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
✓. The patient must have a confirmed diagnosis of MLD as defined by:
✓. The patient must have voluntary function (as judged by the investigator), including cognitive and motor function that is no more than 3 standard deviations below normal at the time of enrollment.
✓. The patient must have an age at the time of screening birth to \< 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
Exclusion criteria
✕. Known multiple sulfatase deficiency
✕. Presence of major congenital abnormality
✕. Presence of known chromosomal abnormality and other neurological conditions unrelated to MLD that can affect psychomotor development
✕. History of hematopoietic 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 principal investigator, would preclude participation in the trial
✕. Use of any investigational product within 30 days prior to study enrollment or currently enrolled in another study which involves clinical investigations.
What they're measuring
1
Results of cognitive and motor testing
Timeframe: baseline, 6 months, 12 months and then yearly