Niemann-Pick Diseases
Showing 1 - 25 of >10,000
Genomic and Phenotypic Database for Niemann-Pick Disease, Type C
Recruiting
- Niemann-Pick Disease, Type C
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Bethesda, MarylandNational Institutes of Health Clinical Center
Feb 2, 2023
Asmd, Visceral Type Trial in Amsterdam (MR Spectroscopy (MRS), MR Elastography (MRE))
Recruiting
- Asmd, Visceral Type
- MR Spectroscopy (MRS)
- MR Elastography (MRE)
-
Amsterdam, Noord-Holland, NetherlandsAmsterdam UMC
Jun 6, 2023
PREDIGA 2: Spanish Acronym of "Educational and Diagnostic
Not yet recruiting
- Gaucher Disease
- +4 more
- Patient with splenomegaly or splenectomy
-
Barcelona, SpainHospital Vall d'Hebron
Dec 13, 2022
GM2 Gangliosidosis, Niemann-Pick Disease, Type C Trial in Curitiba, Porto Alegre, Rio De Janeiro (AZ-3102 (Dose 1), Placebo,
Not yet recruiting
- GM2 Gangliosidosis
- Niemann-Pick Disease, Type C
- AZ-3102 (Dose 1)
- +2 more
-
Curitiba, Brazil
- +2 more
Feb 24, 2023
Functional Near-Infrared Spectroscopy Combined With Diffuse
Recruiting
- Juvenile Neuronal Ceroid Lipofuscinosis
- +3 more
-
Bethesda, MarylandNational Institutes of Health Clinical Center
Jan 30, 2023
Sphingomyelin Lipidosis Trial (olipudase alfa (GZ402665))
Approved for marketing
- Sphingomyelin Lipidosis
- olipudase alfa (GZ402665)
- (no location specified)
Sep 15, 2022
Niemann-Pick Disease, Type C1 Trial in Worldwide (Hydroxypropyl-beta-cyclodextrin, Placebo)
Recruiting
- Niemann-Pick Disease, Type C1
- Hydroxypropyl-beta-cyclodextrin
- Placebo
-
Oakland, California
- +13 more
Jan 9, 2023
Adrenoleukodystrophy, Batten Disease, Mucopolysaccharidosis II Trial in Durham (DUOC-01)
Recruiting
- Adrenoleukodystrophy
- +11 more
- DUOC-01
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Durham, North CarolinaDuke University Medical Center
Oct 24, 2022
Acid Sphingomyelinase Deficiency Disease and Gaucher Disease in
Recruiting
- Gaucher Disease
- Acid SphingoMyelinase Deficiency
-
Ponferrada, León, Spain
- +11 more
Aug 8, 2023
Adult and Pediatric Acid Sphingomyelinase Deficiency on Early
Recruiting
- Acid Sphingomyelinase Deficiency (ASMD)
- Olipudase alfa
-
France, FranceInvestigational site number France
Jun 13, 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
Niemann-Pick Disease, Type C1 Trial run by the (VTS-270)
Completed
- Niemann-Pick Disease, Type C1
-
Bethesda, MarylandNational Institutes of Health Clinical Center, 9000 Rockville Pi
Jul 19, 2021
The Natural History of Metachromatic Leukodystrophy
Recruiting
- MLD
- +26 more
- Palliative Care
- Hematopoetic Stem Cell Transplantation
-
Pittsburgh, PennsylvaniaThe Program for the Study of Neurodevelopment in Rare Disorders,
Nov 2, 2021
Complement Activation in the Lysosomal Storage Disorders
Withdrawn
- Fabry Disease
- +3 more
- Complement measurements
-
Melbourne, Victoria, AustraliaThe Royal Melbourne Hospital
Jun 2, 2021
Sphingomyelin Lipidosis Trial in Worldwide (Olipudase alfa)
Completed
- Sphingomyelin Lipidosis
- Olipudase alfa
-
New York, New York
- +5 more
Mar 15, 2022
Biomarker for Niemann Pick Type C Disease (BioNPC)
Active, not recruiting
- Niemann-Pick Disease
- Niemann-Pick Disease, Type C
-
Rostock, Germany
- +3 more
May 12, 2021
Niemann-Pick Disease, Type C1 Trial in Oakland (Hydroxypropyl-ß-cyclodextrin)
Active, not recruiting
- Niemann-Pick Disease, Type C1
-
Oakland, CaliforniaUCSF Benioff Children's Hospital Oakland
Jun 16, 2021
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
Health Insurance Literacy and Challenges in Accessing Health
Completed
- Niemann-Pick Diseases
-
New York, New YorkIcahn School of Medicine at Mount Sinai
Oct 5, 2020
Pediatric Patients With Unexplained Enlarged Spleen
Recruiting
- Gaucher Disease, Splenomegaly
- Acid SphingoMyelinase Deficiency
-
Amiens, France
- +39 more
Aug 19, 2022
Niemann-Pick Disease, Type C1 Trial in Oakland, Morristown (Hydroxypropyl-beta-cyclodextrin)
Completed
- Niemann-Pick Disease, Type C1
-
Oakland, California
- +1 more
Feb 17, 2021
Niemann-Pick Disease, Type C Trial in Worldwide (Parts A/B: Adrabetadex, Parts A/B: Sham Control)
Active, not recruiting
- Niemann-Pick Disease, Type C
- Parts A/B: Adrabetadex
- Parts A/B: Sham Control
-
Birmingham, Alabama
- +28 more
Jul 23, 2021
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