Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-24 @ 12:09 PM
Ignite Modification Date:
2025-12-24 @ 12:09 PM
NCT ID:
NCT00418561
Eligibility Criteria:
Inclusion Criteria:
1. 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).
2. 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
3. The patient must have a confirmed nerve conduction velocity \< 2 standard deviations (from the appropriate age level)
4. 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.
5. The patient must have an age at the time of screening ≥ 1 year and \< 6 years
6. The patient must have had onset of symptoms before the age of 4 years
7. The subject and his/her guardian(s) must have the ability to comply with the clinical protocol
8. 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:
Patients will be excluded from this study if they do not meet the specific inclusion criteria, or if any of the following criteria apply:
1. Lack of voluntary function
2. Presence of severe pseudo-bulbar signs (weakness and disco-ordination of tongue and swallowing muscles leading to severe difficulty with swallowing)
3. Spasticity so severe to inhibit transportation
4. Known multiple sulfatase deficiency
5. Presence of major congenital abnormality
6. Presence of known chromosomal abnormality and syndromes affecting psychomotor development
7. History of stem cell transplantation
8. Presence of known clinically significant cardiovascular, hepatic, pulmonary or renal disease or other medical condition
9. Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial
10. Use of any investigational product within 30 days prior to study enrolment or currently enrolled in another study which involves clinical investigations
11. Received ERT with rhASA from any source
12. Planned or anticipated initiation of antispastic treatment after trial initiation
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
1 Year
Maximum Age:
5 Years
Study:
NCT00418561
Study Brief:
Protocol Section:
NCT00418561