Treatment Trials

2 Clinical Trials for Various Conditions

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

      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 Disorders
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      TERMINATED
      Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism
      Description

      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).

      Conditions
      Hurler's SyndromeMaroteaux-Lamy SyndromeSly SyndromeAlpha MannosidosisFucosidosisAspartylglucosaminuriaSphingolipidosesKrabbe DiseaseWolman's DiseaseNiemann-Pick Disease Type BNiemann-Pick Disease, Type C
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