| Status | Study |
| Recruiting |
Study Name: Biomarker for Metachromatic Leukodystrophy Disease Condition: Leukodystrophy, Metachromatic Hereditary Central Nervous System Date: 2012-02-21 |
| Completed |
Study Name: Multicenter Study of HGT-1110 Administered Intrathecally in Children With Metachromatic Leukodystrophy (MLD) Condition: Metachromatic Leukodystrophy (MLD) Date: 2011-12-14 Interventions: Biological: Recombinant human arylsulfatase A 6 patients treated with HGT-1110 EOW by IT injection |
| Active, not recruiting |
Study Name: Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders Condition: Hurler Syndrome (MPS I) Hurler-Scheie Syndrome With Early Neurologic Involvement and/or Sens Date: 2011-06-10 Interventions: Biological: FCRx infusion Enriched hematopoetic stem cell infusion |
| Completed |
Study Name: Imaging Study of the White Matter Lesions in Children With Metachromatic Leucodystrophy Condition: Late Infantile Metachromatic Leukodystrophy Date: 2011-03-09 Interventions: Other: High-field MRI (3 Teslas) |
| Completed |
Study Name: Efficacy METAZYM for the Treatment Metachromatic Leukodystrophy Treated With Hematopoietic Stem Cell Transplantation Condition: Metachromatic Leukodystrophy Date: 2011-02-23 Interventions: Drug: rhARSA intravenous infusion 100U/kg every other week for 18 months |
| 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 |
| Completed |
Study Name: Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy Condition: Metachromatic Leukodystrophy Date: 2008-05-21 Interventions: Drug: Warfarin Oral administration (QD), variable dosage: patients will undergo blood test for PT/INR at |
| Terminated |
Study Name: Open-Label Extension Study of Recombinant Human Arylsulfatase A (HGT-1111) in Late Infantile MLD Condition: Late Infantile Metachromatic Leukodystrophy Date: 2008-05-19 Interventions: Drug: HGT-1111 Patients currently dosed with 100 U/kg or 200 U/kg will continue this treatment. Patients |
| Active, not recruiting |
Study Name: The Natural History of Metachromatic Leukodystrophy Condition: Metachromatic Leukodystrophy Date: 2008-03-11 |
| Completed |
Study Name: Long-term Metazym Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD) Condition: Late Infantile Metachromatic Leukodystrophy Date: 2008-02-29 Interventions: Biological: Recombinant human Arylsulfatase A (rhASA) intravenous infusion, every other week for 26 week |