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:
2025-12-25 @ 3:02 AM
Ignite Modification Date:
2025-12-25 @ 3:02 AM
NCT ID:
NCT00654433
Eligibility Criteria:
Inclusion Criteria:
* confirmed diagnosis of inherited metabolic diseases; including the following:
* Hurler Syndrome (MPS I)
* Hurler-Scheie Syndrome
* Hunter Syndrome (MPS II)
* Sanfilippo Syndrome A and B(MPS III)
* Maroteaux-Lamy Syndrome (MPS VI)
* Krabbe Disease (Globoid Leukodystrophy)
* Metachromatic Leukodystrophy (MLD)
* Adrenoleukodystrophy (ALD and AMN)
* Sandhoff Disease
* Tay Sachs Disease
* Pelizaeus Merzbacher (PMD)
* Niemann-Pick Disease
* Alpha-mannosidosis
* I-Cell Disease (ML II)
* Fucosidosis
* GM I Gangliosidosis
* Canavan Disease
* must be \<16 years of age at the time of study enrollment
* must have a good performance status (Lansky ≥80%)
* must have adequate function of other organ systems including: kidney, liver, heart and lungs
* must have given valid written informed consent
* must have a minimum life expectancy of at least 6 months
* must be determined to be a good candidate for a standard umbilical cord blood transplant
* must have an IQ \>70 or if too young for IQ testing the potential to reach this endpoint by age 5
Exclusion Criteria:
* HIV, Hepatitis B and/or Hepatitis C positive
* concurrently involved in any other clinical study that affects engraftment or immune reconstitution
* uncontrolled seizures, apnea, evidence of aspiration pneumonia, or evidence of brain stem involvement
* uncontrolled infections
* prior allogeneic stem cell transplant with cytoreduction preparative therapy within 12 months of enrollment
Healthy Volunteers:
False
Sex:
ALL
Maximum Age:
16 Years
Study:
NCT00654433
Study Brief:
Protocol Section:
NCT00654433