Clinical Trials Logo
  1. Home
  2. Hunter Syndrome
  3. NCT02171104

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
  Status Clinical Trial Phase
Recruiting NCT05422482 - A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients With MPS Ⅱ Phase 1
Completed NCT00630747 - Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase Phase 2/Phase 3
Completed NCT03292887 - Hunter Outcome Survey (HOS)
Active, not recruiting NCT02455622 - Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age Phase 4
Active, not recruiting NCT02412787 - Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094 Phase 2/Phase 3
Completed NCT00882921 - An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients
Completed NCT00920647 - A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase® Phase 1/Phase 2
Completed NCT01449240 - Collection and Study of Cerebrospinal Fluid in Patients With Hunter Syndrome
Completed NCT03920540 - A Study of GC1111 in Hunter Syndrom Patients Phase 3
Recruiting NCT06031259 - Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive Impairment Phase 2/Phase 3
Completed NCT02055118 - Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment Phase 2/Phase 3
Completed NCT01645189 - Safety and Efficacy of Hunterase Phase 3
Completed NCT00937794 - Screening Study to Identify Pediatric Patients With Hunter Syndrome Who Demonstrate Evidence of Central Nervous System (CNS) Involvement and Who Are Currently Receiving Treatment With Elaprase®
Recruiting NCT02044692 - The Long-term Safety Study of Idursulfase-beta in Hunter Syndrome(Mucopolysaccharidosis II) Patients N/A
Completed NCT03582449 - Intensive Pharmacovigilance Program for Elaprase (SHP ELA-701)
Completed NCT01822184 - Observational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II)
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
Completed NCT01043640 - Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders Phase 2
Terminated NCT01330277 - Biomarkers for Hunter Syndrome