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Showing 1–24 of 49 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Niemann-Pick Diseases Clear all
Terminated Phase 2 Interventional Results available

Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency

NCT02193867
View directory record Official record
Conditions
Lysosomal Acid Lipase Deficiency
Interventions
Sebelipase Alfa
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
Up to 8 Months
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
1
States / cities
Phoenix, Arizona
Source: ClinicalTrials.gov public record
Updated Nov 17, 2019 · Synced Sep 26, 2026, 2:27 AM EDT
Completed Phase 1 Interventional

Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1)

NCT02939547
View directory record Official record
Conditions
Niemann-Pick Disease, Type C1
Interventions
Hydroxypropyl-beta-cyclodextrin
Drug
Lead sponsor
Cyclo Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
2
States / cities
Oakland, California • Morristown, New Jersey
Source: ClinicalTrials.gov public record
Updated Feb 20, 2021 · Synced Sep 26, 2026, 2:27 AM EDT
Terminated Phase 1Phase 2 Interventional

Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders

NCT01372228
View directory record Official record
Conditions
Hurler Syndrome (MPS I), Hurler-Scheie Syndrome, Hunter Syndrome (MPS II), Sanfilippo Syndrome (MPS III), Krabbe Disease (Globoid Leukodystrophy), Metachromatic Leukodystrophy (MLD), Adrenoleukodystrophy (ALD and AMN), Sandhoff Disease, Tay Sachs Disease, Pelizaeus Merzbacher (PMD), Niemann-Pick Disease, Alpha-mannosidosis
Interventions
hematopoietic stem cell infusion
Biological
Lead sponsor
Talaris Therapeutics Inc.
Industry
Eligibility
Not listed
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Apr 11, 2023 · Synced Sep 26, 2026, 2:27 AM EDT
Completed Phase 1 Interventional

Tolerability and Safety Study of Recombinant Human Acid Sphingomyelinase in Acid Sphingomyelinase Deficiency Patients

NCT01722526
View directory record Official record
Conditions
Human Acid Sphingomyelinase Deficiency
Interventions
Recombinant human acid sphingomyelinase
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years to 65 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 29, 2015 · Synced Sep 26, 2026, 2:27 AM EDT
Recruiting Phase 3 Interventional

A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C

NCT05163288
View directory record Official record
Conditions
Niemann-Pick Disease, Type C
Interventions
N-Acetyl-L-Leucine, Placebo
Drug · Other
Lead sponsor
IntraBio Inc
Industry
Eligibility
4 Years and older
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2030
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 2, 2026 · Synced Sep 26, 2026, 2:27 AM EDT
Terminated Phase 2Phase 3 Interventional Results available

Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease

NCT04958642
View directory record Official record
Conditions
Niemann-Pick Disease, Type C
Interventions
Adrabetadex
Drug
Lead sponsor
Mandos LLC
Industry
Eligibility
4 Years to 21 Years
Enrollment
66 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2022
U.S. locations
15
States / cities
Birmingham, Alabama • Orange, California • San Francisco, California + 12 more
Source: ClinicalTrials.gov public record
Updated Aug 28, 2023 · Synced Sep 26, 2026, 2:27 AM EDT
Terminated Phase 3 Interventional

ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases

NCT00654433
View directory record Official record
Conditions
Inherited Metabolic Diseases, Lysosomal Storage Disorders, Peroxisomal Storage Diseases, Inborn Errors of Metabolism, Mucopolysaccharidosis
Interventions
ALD-101
Biological
Lead sponsor
Aldagen
Industry
Eligibility
Up to 16 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
3
States / cities
Los Angeles, California • New York, New York • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 7, 2014 · Synced Sep 26, 2026, 2:27 AM EDT
Completed No phase listed Observational

Longitudinal Study of Cognition With Niemann-Pick Disease, Type C

NCT01899950
View directory record Official record
Conditions
Niemann-Pick Disease, Type C
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
2 Years to 99 Years
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 5, 2016 · Synced Sep 26, 2026, 2:27 AM EDT
Active, not recruiting Phase 2 Interventional

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

NCT02171104
View directory record Official record
Conditions
Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome, Maroteaux Lamy Syndrome, Sly Syndrome, Alpha-Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Glycoprotein Metabolic Disorders, Sphingolipidoses, Recessive Leukodystrophies, Globoid Cell Leukodystrophy, Metachromatic Leukodystrophy, Niemann-Pick B, Niemann-Pick C Subtype 2, Sphingomyelin Deficiency, Peroxisomal Disorders, Adrenoleukodystrophy With Cerebral Involvement, Zellweger Syndrome, Neonatal Adrenoleukodystrophy, Infantile Refsum Disease, Acyl-CoA Oxidase Deficiency, D-Bifunctional Enzyme Deficiency, Multifunctional Enzyme Deficiency, Alpha-methylacyl-CoA Racmase Deficiency, Mitochondrial Neurogastrointestingal Encephalopathy, Severe Osteopetrosis, Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation), Inherited Metabolic Disorders
Interventions
Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen, Osteopetrosis Haploidentical Only Preparative Regimen, cALD SR-A (Standard-Risk, Regimen A), cALD SR-B (Standard-Risk, Regimen B), cALD HR-D (High-Risk, Regimen C), cALD HR-D (High-Risk, Regimen D)
Biological · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
149 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2029
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 6, 2026 · Synced Sep 26, 2026, 2:27 AM EDT
Completed Phase 2 Interventional Results available

A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

NCT02004704
View directory record Official record
Conditions
Sphingomyelin Lipidosis
Interventions
GZ402665
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2023
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 2, 2024 · Synced Sep 26, 2026, 2:27 AM EDT
Completed Phase 1Phase 2 Interventional

Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells

NCT00730314
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Anemia, Granuloma, Wiskott-Aldrich Syndrome, Chediak Higashi Syndrome, Osteopetrosis, Neutropenia, Thrombocytopenia, Hurler Disease, Niemann-Pick Disease, Fucosidosis
Interventions
Hematopoietic stem cell transplantation
Procedure
Lead sponsor
Children's Hospital Los Angeles
Other
Eligibility
Up to 21 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jun 22, 2016 · Synced Sep 26, 2026, 2:27 AM EDT
Terminated Phase 3 Interventional Results available

Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System

NCT03643562
View directory record Official record
Conditions
Niemann-Pick Type C Disease
Interventions
Adrabetadex
Drug
Lead sponsor
Mandos LLC
Industry
Eligibility
4 Years and older
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
17
States / cities
Little Rock, Arkansas • Loma Linda, California • Los Angeles, California + 13 more
Source: ClinicalTrials.gov public record
Updated Jan 16, 2024 · Synced Sep 26, 2026, 2:27 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

PET Scan of Brain Metabolism in Relation to Age and Disease

NCT00001972
View directory record Official record
Conditions
Alzheimer's Disease, Brain Neoplasm, Niemann Pick Disease
Interventions
15 O Water
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
123 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1994 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Sep 26, 2026, 2:27 AM EDT
Recruiting Phase 1 Interventional

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

NCT02254863
View directory record Official record
Conditions
Adrenoleukodystrophy, Batten Disease, Mucopolysaccharidosis II, Leukodystrophy, Globoid Cell, Leukodystrophy, Metachromatic, Neimann Pick Disease, Pelizaeus-Merzbacher Disease, Sandhoff Disease, Tay-Sachs Disease, Brain Diseases, Metabolic, Inborn, Alpha-Mannosidosis, Sanfilippo Mucopolysaccharidoses
Interventions
DUOC-01
Biological
Lead sponsor
Joanne Kurtzberg, MD
Other
Eligibility
1 Week to 22 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2027
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Sep 26, 2026, 2:27 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Stem Cell Transplant for Inborn Errors of Metabolism

NCT00176904
View directory record Official record
Conditions
Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Gaucher's Disease, Fucosidosis, Wolman Disease, Niemann-Pick Disease, Batten Disease, GM1 Gangliosidosis, Tay Sachs Disease, Sandhoff Disease
Interventions
Stem Cell Transplant, Busulfan, Cyclophosphamide, Antithymocyte Globulin
Procedure · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Not listed
Enrollment
135 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1995 – 2010
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 27, 2017 · Synced Sep 26, 2026, 2:27 AM EDT
Not listed Phase 1 Interventional

Open-Label Study of Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann-Pick Disease Type C

NCT03893071
View directory record Official record
Conditions
Niemann-Pick Disease, Type C1
Interventions
Hydroxypropyl-β-cyclodextrin
Drug
Lead sponsor
Cyclo Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Jun 17, 2021 · Synced Sep 26, 2026, 2:27 AM EDT
Completed Phase 2 Interventional Results available

N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)

NCT03759639
View directory record Official record
Conditions
Niemann-Pick Disease, Type C
Interventions
IB1001
Drug
Lead sponsor
IntraBio Inc
Industry
Eligibility
6 Years and older
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Nov 27, 2023 · Synced Sep 26, 2026, 2:27 AM EDT
Recruiting No phase listed Observational

A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD)

NCT06192576
View directory record Official record
Conditions
Niemann-Pick Diseases, Acid Sphingomyelinase Deficiency
Interventions
Olipudase alfa
Drug
Lead sponsor
Sanofi
Industry
Eligibility
Up to 2 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
6
States / cities
Chicago, Illinois • Cincinnati, Ohio • Columbus, Ohio + 3 more
Source: ClinicalTrials.gov public record
Updated Sep 15, 2026 · Synced Sep 26, 2026, 2:27 AM EDT
Completed Phase 1 Interventional

Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease

NCT01747135
View directory record Official record
Conditions
Niemann-Pick Disease, Type C1
Interventions
VTS-270
Drug
Lead sponsor
Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company
Industry
Eligibility
2 Years to 25 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 22, 2021 · Synced Sep 26, 2026, 2:27 AM EDT
Terminated Phase 2 Interventional Results available

Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism

NCT00668564
View directory record Official record
Conditions
Hurler's Syndrome, Maroteaux-Lamy Syndrome, Sly Syndrome, Alpha Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Sphingolipidoses, Krabbe Disease, Wolman's Disease, Niemann-Pick Disease Type B, Niemann-Pick Disease, Type C
Interventions
Stem Cell Transplantation, Cyclophosphamide, Campath-1H, Busulfan
Procedure · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 21 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 27, 2017 · Synced Sep 26, 2026, 2:27 AM EDT
Terminated No phase listed Observational

A Study to Identify and Characterize LAL-D Patients in High-risk Populations

NCT02345421
View directory record Official record
Conditions
Lysosomal Acid Lipase Deficiency
Interventions
Not listed
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
2 Years and older
Enrollment
640 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
15
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 12 more
Source: ClinicalTrials.gov public record
Updated May 23, 2016 · Synced Sep 26, 2026, 2:27 AM EDT
Not listed No phase listed Observational

Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

NCT00005900
View directory record Official record
Conditions
I Cell Disease, Fucosidosis, Globoid Cell Leukodystrophy, Adrenoleukodystrophy, Mannosidosis, Niemann-Pick Disease, Pulmonary Complications, Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Metachromatic Leukodystrophy, Gaucher's Disease, Wolman Disease
Interventions
Not listed
Lead sponsor
Fairview University Medical Center
Other
Eligibility
0 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Sep 26, 2026, 2:27 AM EDT
Completed Phase 2Phase 3 Interventional Accepts healthy volunteers Results available

Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

NCT02004691
View directory record Official record
Conditions
Sphingomyelin Lipidosis
Interventions
placebo (saline), Olipudase alfa
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
Enrollment
36 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2023
U.S. locations
3
States / cities
San Francisco, California • Decatur, Georgia • The Bronx, New York
Source: ClinicalTrials.gov public record
Updated Nov 6, 2024 · Synced Sep 26, 2026, 2:27 AM EDT
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

NCT05368038
View directory record Official record
Conditions
Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2, Cerebrotendinous Xanthomatosis, Fabry Disease, GM1 Gangliosidosis, Gaucher Disease, Lysosomal Acid Lipase Deficiency, Metachromatic Leukodystrophy, Mucopolysaccharidosis II, Mucopolysaccharidosis III-B, Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI, Mucopolysaccharidosis VII, Niemann-Pick Disease, Type C
Interventions
Confirmatory Testing
Diagnostic Test
Lead sponsor
Albert Einstein College of Medicine
Other
Eligibility
Up to 4 Weeks
Enrollment
100,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2029
U.S. locations
9
States / cities
Brooklyn, New York • Manhasset, New York • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 27, 2026 · Synced Sep 26, 2026, 2:27 AM EDT