ACTIVE_NOT_RECRUITING

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

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Conditions

Mucopolysaccharidosis DisordersHurler SyndromeHunter SyndromeMaroteaux Lamy SyndromeSly SyndromeAlpha-MannosidosisFucosidosisAspartylglucosaminuriaGlycoprotein Metabolic DisordersSphingolipidosesRecessive LeukodystrophiesGloboid Cell LeukodystrophyMetachromatic LeukodystrophyNiemann-Pick BNiemann-Pick C Subtype 2Sphingomyelin DeficiencyPeroxisomal DisordersAdrenoleukodystrophy With Cerebral InvolvementZellweger SyndromeNeonatal AdrenoleukodystrophyInfantile Refsum DiseaseAcyl-CoA Oxidase DeficiencyD-Bifunctional Enzyme DeficiencyMultifunctional Enzyme DeficiencyAlpha-methylacyl-CoA Racmase DeficiencyMitochondrial Neurogastrointestingal EncephalopathySevere OsteopetrosisHereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation)Inherited Metabolic Disordersallogeneic hematopoietic cell transplantationbone marrow transplantationIMDAMACRDMNGIEHDLSOPALD

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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).

Official Title

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

Quick Facts

Study Start:2014-07-10
Study Completion:2029-07-14
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:ACTIVE_NOT_RECRUITING

Study ID

NCT02171104

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:Not specified to 55 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT
Inclusion CriteriaExclusion Criteria
  1. * 0 through 55 years of age
  2. * Adequate graft available
  3. * Adequate organ function
  4. * Eligible Diseases:
  5. * Mucopolysaccharidosis Disorders:
  6. * MPS IH (Hurler syndrome)
  7. * MPS II (Hunter syndrome) if the patient has no or minimal evidence of symptomatic neurologic disease but is expected to have a neurologic phenotype
  8. * MPS VI (Maroteaux-Lamy syndrome)
  9. * MPS VII (Sly syndrome)
  10. * Glycoprotein Metabolic Disorders:
  11. * Alpha mannosidosis
  12. * Fucosidosis
  13. * Aspartylglucosaminuria
  14. * Sphingolipidoses and Recessive Leukodystrophies:
  15. * Globoid cell leukodystrophy
  16. * Metachromatic leukodystrophy
  17. * Niemann-Pick B patients (sphingomyelin deficiency)
  18. * Niemann-Pick C subtype 2
  19. * Peroxisomal Disorders:
  20. * Adrenoleukodystrophy with cerebral involvement
  21. * Zellweger syndrome
  22. * Neonatal Adrenoleukodystrophy
  23. * Infantile Refsum disease
  24. * Acyl-CoA-Oxidase Deficiency
  25. * D-Bifunctional enzyme deficiency
  26. * Multifunctional enzyme deficiency
  27. * Alpha-methylacyl-CoA Racmase Deficiency (AMACRD)
  28. * Mitochondrial Neurogastrointestingal Encephalopathy (MNGIE)
  29. * Severe Osteopetrosis (OP)
  30. * Hereditary Leukoencephalopathy with axonal spheroids (HDLS; CSF1R mutation)
  31. * 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.
  32. * Voluntary written consent
  1. * Pregnancy - menstruating females must have a negative serum or urine pregnancy test within 14 days of study treatment start
  2. * Prior myeloablative chemotherapy exposure within 4 months of the start of conditioning on this protocol (patients excluded for this reason may be eligible for other institutional protocols)
  3. * Uncontrolled bacterial, fungal or viral infections including HIV (including active infection with Aspergillus or other mold within 30 days)

Contacts and Locations

Principal Investigator

Paul Orchard, M.D.
PRINCIPAL_INVESTIGATOR
Masonic Cancer Center, University of Minnesota

Study Locations (Sites)

Masonic Cancer Center, University of Minnesota
Minneapolis, Minnesota, 55455
United States

Collaborators and Investigators

Sponsor: Masonic Cancer Center, University of Minnesota

  • Paul Orchard, M.D., PRINCIPAL_INVESTIGATOR, Masonic Cancer Center, University of Minnesota

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2014-07-10
Study Completion Date2029-07-14

Study Record Updates

Study Start Date2014-07-10
Study Completion Date2029-07-14

Terms related to this study

Keywords Provided by Researchers

  • allogeneic hematopoietic cell transplantation
  • bone marrow transplantation
  • IMD
  • AMACRD
  • MNGIE
  • HDLS
  • OP
  • ALD

Additional Relevant MeSH Terms

  • Mucopolysaccharidosis Disorders
  • Hurler Syndrome
  • Hunter Syndrome
  • Maroteaux Lamy Syndrome
  • Sly Syndrome
  • Alpha-Mannosidosis
  • Fucosidosis
  • Aspartylglucosaminuria
  • Glycoprotein Metabolic Disorders
  • Sphingolipidoses
  • Recessive Leukodystrophies
  • Globoid Cell Leukodystrophy
  • Metachromatic Leukodystrophy
  • Niemann-Pick B
  • Niemann-Pick C Subtype 2
  • Sphingomyelin Deficiency
  • 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
  • Mitochondrial Neurogastrointestingal Encephalopathy
  • Severe Osteopetrosis
  • Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation)
  • Inherited Metabolic Disorders