Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism (NCT00668564) | Clinical Trial Compass
TerminatedPhase 2
Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism
Stopped: Replaced by another study
United States18 participantsStarted 2008-03
Plain-language summary
The primary objective of this clinical trial is to evaluate the ability to achieve and sustain donor engraftment in patients with lysosomal and peroxisomal inborn errors of metabolism undergoing hematopoietic stem cell transplantation (HCT).
Who can participate
Age range21 Years
SexALL
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Inclusion Criteria:
* Mucopolysaccharidosis (MPS) Disorders:
* MPS IH (Hurler syndrome)
* MPS-VI (Maroteaux-Lamy syndrome)
* MPS VII (Sly syndrome).
* Glycoprotein metabolic disorders:
* Alpha mannosidosis
* Fucosidosis
* Aspartylglucosaminuria
* Sphingolipidoses and Recessive Leukodystrophies: Presymptomatic patients with globoid cell leukodystrophy (GLD, also known as Krabbe disease) and metachromatic leukodystrophy (MLD) will be eligible for treatment on this protocol. White matter disease by magnetic resonance imaging (MRI) alone is not an exclusion if the patient is asymptomatic.
* Peroxisomal Disorders: Presymptomatic patients with inherited peroxisomal disorders associated with of very long chain fatty acids (VLCFA) elevation, identified by family history or laboratory testing (including neonatal screening), are eligible for this protocol. White matter disease by MRI alone is not an exclusion if the patient is asymptomatic.
* Other Inherited Diseases of Metabolism:
* Wolman syndrome (acid lipase deficiency)
* Niemann-Pick B patients (sphingomyelin deficiency)
* Niemann-Pick C subtype 2
* Donor Availability: Patients considered for transplantation must have a sufficient graft as based on current criteria of the University of Minnesota Blood and Marrow Transplantation Program: Priority will be as follows, although in circumstances in which timing is of the essence, cord blood grafts may be chosen over an unrelated graft, despite the priority listed …
What they're measuring
1
Number of Patients Achieving Engraftment
Timeframe: Day 100
Trial details
NCT IDNCT00668564
SponsorMasonic Cancer Center, University of Minnesota