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MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

Hunter Syndrome Clinical Trial

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

Clinical Trial Summary

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).

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

  • Acyl-CoA Oxidase Deficiency
  • Adrenoleukodystrophy
  • Adrenoleukodystrophy With Cerebral Involvement
  • Alpha-Mannosidosis
  • Alpha-methylacyl-CoA Racmase Deficiency
  • Aspartylglucosaminuria
  • Brain Diseases
  • D-Bifunctional Enzyme Deficiency
  • Fucosidosis
  • Globoid Cell Leukodystrophy
  • Glycoprotein Metabolic Disorders
  • Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation)
  • Hunter Syndrome
  • Hurler Syndrome
  • Infantile Refsum Disease
  • Inherited Metabolic Disorders
  • Leukodystrophy, Globoid Cell
  • Leukodystrophy, Metachromatic
  • Leukoencephalopathies
  • Maroteaux Lamy Syndrome
  • Metabolic Diseases
  • Metachromatic Leukodystrophy
  • Mitochondrial Neurogastrointestingal Encephalopathy
  • Mucopolysaccharidoses
  • Mucopolysaccharidosis Disorders
  • Mucopolysaccharidosis I
  • Mucopolysaccharidosis II
  • Mucopolysaccharidosis VI
  • Mucopolysaccharidosis VII
  • Multifunctional Enzyme Deficiency
  • Neonatal Adrenoleukodystrophy
  • Niemann-Pick B
  • Niemann-Pick C Subtype 2
  • Osteopetrosis
  • Peroxisomal Disorders
  • Recessive Leukodystrophies
  • Refsum Disease
  • Severe Osteopetrosis
  • Sly Syndrome
  • Sphingolipidoses
  • Sphingomyelin Deficiency
  • Syndrome
  • Zellweger Syndrome
Clinical Trial Details
NCT number NCT02171104
Study type Interventional
Source Masonic Cancer Center, University of Minnesota
Contact Lisa Burke
Phone 612-273-8482
Email lburke3@Fairview.org
Status Recruiting
Phase Phase 2
Start date July 10, 2014
Completion date July 14, 2028
View Details
See also
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Recruiting NCT05494593 - A Study of ELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis [MPS] II) Phase 4
Completed NCT00607386 - Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy Phase 4
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Completed NCT01506141 - An Extension Study of HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Idursulfase-IT in Conjunction With Elaprase in Pediatric Participants With Hunter Syndrome and Cognitive Impairment Phase 1/Phase 2