MT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG

Trial Information
Who is this study for? Child to adult patients with Mucopolysaccharidosis Disorder, Glycoprotein Metabolic Disorder, Sphingolipidoses and Recessive Leukodystrophy, Peroxisomal Disorder, Osteopetrosis, Hereditary Leukoencephalopathy, or other Inherited Metabolic Disorders
What treatments are being studied? Stem Cell Transplant
Status: Recruiting
Location: See location...
Intervention Type: Drug, Biological
Study Type: Interventional
Study Phase: Phase 2

This single-institution, phase II study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-related mortality (TRM) using busulfan- and fludarabine-based conditioning regimens with busulfan therapeutic drug monitoring (TDM) for patients with various inherited metabolic disorders (IMD) and severe osteopetrosis (OP).

Am I eligible for this trial?
Participation Requirements
Maximum Age:
Healthy Volunteers:

• 0 through 55 years of age

• Adequate graft available

• Adequate organ function

• Eligible Diseases:

• Mucopolysaccharidosis Disorders:

• MPS IH (Hurler syndrome)

• MPS II (Hunter syndrome) if the patient has no or minimal evidence of symptomatic neurologic disease but is expected to have a neurologic phenotype

• MPS VI (Maroteaux-Lamy syndrome)

• MPS VII (Sly syndrome)

• Glycoprotein Metabolic Disorders:

• Alpha mannosidosis

• Fucosidosis

• Aspartylglucosaminuria

• Sphingolipidoses and Recessive Leukodystrophies:

• Globoid cell leukodystrophy

• Metachromatic leukodystrophy

• Niemann-Pick B patients (sphingomyelin deficiency)

• Niemann-Pick C subtype 2

• Peroxisomal Disorders:

• Adrenoleukodystrophy with cerebral involvement

• Zellweger syndrome

• Neonatal Adrenoleukodystrophy

• Infantile Refsum disease

• Acyl-CoA-Oxidase Deficiency

• D-Bifunctional enzyme deficiency

• Multifunctional enzyme deficiency

• Alpha-methylacyl-CoA Racmase Deficiency (AMACRD)

• Mitochondrial Neurogastrointestingal Encephalopathy (MNGIE)

• Severe Osteopetrosis (OP)

• Hereditary Leukoencephalopathy with axonal spheroids (HDLS; CSF1R mutation)

• Other Inherited Metabolic Disorders (IMD): Patients will also be considered who have other life-threatening, rare lysosomal, peroxisomal or other similar inherited disorders characterized by white matter disease or other neurologic manifestations for which there is rationale that transplantation would be of benefit, such as certain patients with Wolman's disease, GM1 gangliosidosis, I-cell disease, Tay-Sachs disease, Sandhoff disease or others.

• Voluntary written consent

Where is this trial taking place?
United States
Masonic Cancer Center, University of Minnesota
Who do I contact about this trial?
Lisa Burke
Troy Lund, M.D.Ph.D.
When is this trial taking place?
Start Date: July 10, 2014
Estimated Completion Date: December 2022
How many participants will be in this trial?
Target number of participants: 100
What treatment is being studied in this trial?
Experimental: IMD - Except Haplo-identical
Inherited Metabolic Disease (IMD) - Except Haplo-Identical~See intervention descriptions.
Experimental: OP - Except Haplo-Identical
Severe Osteoperosis (OP) - Except Haplo-Identical~See intervention descriptions.
Experimental: OP and IMD -Haplo-Identical Only
Severe Osteopetrosis (OP) and Inhterited Metabolic Disorders (IMD)~-Haplo-Identical Only~See intervention descriptions.
Experimental: cALD SR-A (Standard-Risk, Regimen A)
See intervention descriptions.
Experimental: cALD SR-B (Standard-Risk, Regimen B)
See intervention descriptions.
Experimental: cALD HR-C (High-Risk, Regimen C)
See intervention descriptions.
Experimental: cALD HR-D (High-Risk, Regimen D)
See intervention descriptions.
Who are the authors of this trial?

This content was sourced from

Phase II Study of Timed Sequential Busulfan in Combination With Fludarabine in Allogeneic Stem Cell Transplantation
Who is this study for:Patients with High-Risk Hematologic Malignancies
Status:Active, not recruiting
Start Date:April 2012
Study Drug:Stem Cell Transplantation
Study Type:Drug, Procedure
Phase: Phase 2
PRO#1278: A Phase III Study of Fludarabine and Busulfan Versus Fludarabine, Busulfan and Low Dose Total Body Irradiation in Patients Receiving an Allogeneic Hematopoietic Stem Cell Transplant
Who is this study for:Adult patients with Acute Myeloid Leukemia, Chronic Myeloid Leukemia, or Myelodysplastic Syndrome
Status:Active, not recruiting
Start Date:June 16, 2010
Study Type:Drug
Phase: Phase 3