At a glance
ClinicalIndex Comparison Record- ✓Diagnosis of one of the eligible lysosomal or peroxisomal storage diseases (adrenoleukodystrophy, metachromatic leukodystrophy, globoid cell leukodystrophy, Gaucher's disease, Fucosidosis, Wolman disease, Niemann-Pick disease, Batten disease, GM1 gangliosidosis, Tay Sachs disease, or Sandhoff disease)
- ✓HLA-identical or partially mismatched (1-3 antigens for related, 1 antigen for unrelated) related donor marrow, unrelated peripheral blood donor, or umbilical cord blood (1-2 units)
- ✓Adrenoleukodystrophy patients: MRI findings and neuropsychometric function consistent with diagnosis; boys with parietal-occipital dysmyelination must have performance IQ ≥80 (protocol committee may allow transplant with lower IQ if clinical condition acceptable)
- ✓Metachromatic leukodystrophy patients: presymptomatic late infantile, juvenile, or adult form with acceptable neurological and neuropsychometric function
- ✕Symptomatic late infantile form of metachromatic leukodystrophy
- ✕Symptomatic infantile globoid leukodystrophy
- ✕Hurler syndrome, mucopolysaccharidosis (MPS) VI, or Mannosidosis disease
- ✕Pregnancy
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
Treatment of Lysosomal and Peroxisomal Inborn Errors of Metabolism by Bone Marrow Transplantation
In Brief
A Phase 3 clinical trial evaluating Stem Cell Transplant and Busulfan, Cyclophosphamide, Antithymocyte Globulin for Adrenoleukodystrophy and 10 related conditions. Completed, enrolled 135 participants across 1 site.
Detailed Summary
The purpose of this study is to determine the safety and engraftment of donor hematopoietic cells using this conditioning regimen in patients undergoing a hematopoietic (blood forming) cell transplant for an inherited metabolic storage disease.
Study Details
Timeline
Interventions
The purpose of hematopoietic cell transplantation is to introduce hematopoietic cells from a normal donor that contains an enzyme able to get rid of the substances that have accumulated in the body of patients with storage diseases. Hematopoietic cells can come from bone marrow, peripheral blood (i.e., the blood circulating in our body's blood vessels) or umbilical cord blood (i.e., blood taken from the umbilical cord after a baby is born and umbilical cord is cut).
Subjects will receive BUSULFAN intravenously (IV)- patients \< or= 12 kg 1.1 mg/kd/dose IV every 6 hours for 16 doses; patients \> 12kg 0.8 mg/kg/dose IV every 6 hours for 16 doses - via the Hickman line four times daily for 4 days, CYCLOPHOSPHAMIDE intravenously (50 mg/kg/day IV over 2 hours) via the Hickman line once a day for 4 days, and ANTI-THYMOCYTE GLOBULIN IV (15 mg/kg/day over 2 hours) via the Hickman line twice daily for three days before the transplant. These three drugs are being given to help the new marrow "take" and grow. METHYLPREDNISOLONE will be given as a pre-medication for the ATG.