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Peroxisomal Biogenesis Disorders - 25 Studies Found

Status Study
Completed Study Name: Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders
Condition:
  • Lysosomal Storage Disease
  • Peroxisomal Disorder

Date: 2012-06-20
Interventions:
  • Drug: Campath-1H A daily dose

Not yet recruiting Study Name: Evaluation of a New Strategy for the Diagnosis of Peroxisomal Diseases
Condition:
  • Peroxisomal Disorders
  • Diagnoses Disease

Date: 2017-05-22
Not yet recruiting Study Name: Study of Protein Translocation in Patients With Beta-Oxidation Disorders
Condition:
  • Beta-Oxidation Disorder
  • Peroxisomal Disorders

Date: 1999-10-18
Terminated Study Name: Study of Bile Acids in Patients With Peroxisomal Disorders
Condition:
  • Infantile Refsum's Disease
  • Zellweger Syndrome
  • Bifunctional
    Date: 1999-10-18
    Interventions:
    • Drug: chenodeoxycholic acid
    • Drug: cholic a
Completed Study Name: HSCT for High Risk Inherited Inborn Errors
Condition:
  • Adrenoleukodystrophy
  • Metachromatic Leukodystrophy
  • Globoid
    Date: 2006-09-29
    Interventions:
    • Drug: Clofarabine days -7 thro
Completed Study Name: Betaine and Peroxisome Biogenesis Disorders
Condition: Peroxisome Biogenesis Disorders
Date: 2013-03-29
Interventions: Drug: Betaine Betaine will be given orally (mixed with foo
Recruiting Study Name: Longitudinal Natural History Study of Patients With Peroxisome Biogenesis Disorders (PBD)
Condition: Peroxisome Biogenesis Disorders
Date: 2012-08-10
Interventions:
  • Genetic: Next-generation panel

Completed Study Name: Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid
Condition:
  • Infantile Refsum's Disease
  • Zellweger Syndrome
  • Adrenoleukod
    Date: 2000-12-06
    Interventions: Drug: Cholic Acids 10-15 mg/kg body weight/day taken orally.
Recruiting Study Name: MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
Condition:
  • Mucopolysaccharidosis Disorders
  • Hurler Syndrome
  • Hunter Syn
    Date: 2014-06-20
    Interventions:
    • Biological: Stem Cell Transplantation

Active, not recruiting Study Name: Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
Condition:
  • Mucopolysaccharidosis
  • Hurler Syndrome
  • Hunter Syndrome

    • Date: 2010-01-05
    Interventions:
    • Drug: Campath-1H Administered

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