metachromatic leukodystrophy
Showing 1 - 21 of 21
Baby Detect : Genomic Newborn Screening
Recruiting
- Congenital Adrenal Hyperplasia
- +126 more
-
Liege, Wallonia, BelgiumCRMN, Hôpital La Citadelle
Jan 24, 2023
Lysosomal Storage Diseases, Metachromatic Leukodystrophy Trial in Milan (OTL-200)
Recruiting
- Lysosomal Storage Diseases
- Metachromatic Leukodystrophy
- OTL-200
-
Milan, ItalyOspedale San Raffaele - Telethon Institute for Gene Therapy (OSR
Nov 16, 2022
Lysosomal Storage Disease, Metachromatic Leukodystrophy Trial in Milan (OTL-200 Gene Therapy)
Active, not recruiting
- Lysosomal Storage Disease
- Metachromatic Leukodystrophy
- OTL-200 Gene Therapy
-
Milan, ItalyOspedale San Raffaele - Telethon Institute for Gene Therapy (OSR
Nov 4, 2022
Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome Trial in Minneapolis (Stem Cell Transplantation, IMD
Recruiting
- Mucopolysaccharidosis Disorders
- +28 more
- Stem Cell Transplantation
- +7 more
-
Minneapolis, MinnesotaMasonic Cancer Center, University of Minnesota
Nov 1, 2022
Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Adrenoleukodystrophy Trial in Denver, Valhalla, Salt Lake City (Human
Completed
- Mucopolysaccharidosis I
- +15 more
- Human Placental Derived Stem Cell
-
Denver, Colorado
- +2 more
Oct 24, 2022
Macrophages Activation Pattern in X-linked Adrenoleukodystrophy,
Recruiting
- Adrenoleukodystrophy
- +3 more
- Blood sample collection
-
Le Kremlin-Bicêtre, France
- +1 more
Oct 10, 2022
Metachromatic Leukodystrophy Trial in Durham (Enriched Hematopoetic Stem Cell Infusion)
No longer available
- Metachromatic Leukodystrophy
- Enriched Hematopoetic Stem Cell Infusion
-
Durham, North CarolinaDuke University
Jul 15, 2022
Metachromatic Leukodystrophy, Adrenoleukodystrophy Trial in Shenzhen (transduced CD34+ hematopoietic stem cell)
Recruiting
- Metachromatic Leukodystrophy
- Adrenoleukodystrophy
- transduced CD34+ hematopoietic stem cell
-
Shenzhen, Guangdong, ChinaShenzhen Second People's Hospital, The First Affiliated Hospital
May 25, 2022
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn
Enrolling by invitation
- Acid Sphingomyelinase Deficiency
- +13 more
- Confirmatory Testing
-
Bronx, New York
- +9 more
May 9, 2022
Metachromatic Leukodystrophy Trial in Le Kremlin-Bicêtre (intracerebral administration of AAVrh.10cuARSA)
Active, not recruiting
- Metachromatic Leukodystrophy
- intracerebral administration of AAVrh.10cuARSA
-
Le Kremlin-Bicêtre, FranceBicêtre Hospital - Paris Sud
Jan 10, 2022
Leukodystrophy, White Matter Disease, Leukoencephalopathies Trial in United States
Recruiting
- Leukodystrophy
- +21 more
-
Palo Alto, California
- +8 more
Dec 21, 2021
Metachromatic Leukodystrophy Study (HOME Study)
Recruiting
- Metachromatic Leukodystrophy
-
Danbury, ConnecticutNational Organization for Rare Disorders
Oct 28, 2021
Lysosomal Storage Disease, Metachromatic Leukodystrophy Trial in Milan (OTL-200)
Active, not recruiting
- Lysosomal Storage Disease
- Metachromatic Leukodystrophy
- OTL-200
-
Milan, ItalyOspedale San Raffaele - Telethon Institute for Gene Therapy (OSR
Sep 29, 2021
Metachromatic Leukodystrophy Trial in Durham (Enriched Hematopoetic Stem Cell Infusion)
No longer available
- Metachromatic Leukodystrophy
- Enriched Hematopoetic Stem Cell Infusion
-
Durham, North CarolinaDuke University
Oct 6, 2020
Hurler Syndrome, Inherited Metabolic Disorder, Lysosomal Storage Disorder Trial in Los Angeles, Palo Alto (rivogenlecleucel,
No longer available
- Hurler Syndrome
- +4 more
- rivogenlecleucel
- rimiducid
-
Los Angeles, California
- +1 more
Oct 1, 2020
Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy Trial in Minneapolis (drug, procedure,
Completed
- Adrenoleukodystrophy
- +8 more
- Clofarabine
- +7 more
-
Minneapolis, MinnesotaMasonic Cancer Center, University of Minnesota
Jul 10, 2019
Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy Trial in Minneapolis (Stem Cell Transplant,
Completed
- Adrenoleukodystrophy
- +10 more
- Stem Cell Transplant
- Busulfan, Cyclophosphamide, Antithymocyte Globulin
-
Minneapolis, MinnesotaMasonic Cancer Center, University of Minnesota
Dec 3, 2017
Metachromatic Leukodystrophy Trial in Camden (Warfarin)
Completed
- Metachromatic Leukodystrophy
-
Camden, New JerseyCooper University Hospital
Mar 18, 2011
Metachromatic Leukodystrophy Trial in Paris (rhARSA)
Completed
- Metachromatic Leukodystrophy
-
Paris, FranceDepartment of Pediatric Endocrinology and Neurology, Saint Vince
Feb 23, 2011
Thrombocytopenia, Metachromatic Leukodystrophy, Fanconi's Anemia Trial in Los Angeles (Stem Cell Transplantation)
Completed
- Thrombocytopenia
- +5 more
- Stem Cell Transplantation
-
Los Angeles, CaliforniaUniversity of California Los Angeles Medical Center
Jun 23, 2005
Pulmonary Complications in Pediatric Storage Disorders
Unknown status
- I Cell Disease
- +11 more
-
Minneapolis, MinnesotaFairview University Medical Center
Jun 23, 2005