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ClinicalTrials.gov public records Last synced Oct 2, 2026, 6:00 AM EDT

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Showing 1–24 of 29 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Niemann-Pick Disease, Type C Clear all
Recruiting No phase listed Observational Accepts healthy volunteers

Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C

NCT00344331
View directory record Official record
Conditions
Niemann-Pick Disease, Type C
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
1 Day to 120 Years
Enrollment
900 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2006
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 28, 2026 · Synced Oct 2, 2026, 6:00 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 29, 2023 · Synced Oct 2, 2026, 6:00 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 7, 2026 · Synced Oct 2, 2026, 6:00 AM EDT
Completed Phase 2Phase 3 Interventional Results available

VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease

NCT02534844
View directory record Official record
Conditions
Niemann-Pick Disease, Type C
Interventions
Parts A/B: Adrabetadex, Parts A/B: Sham Control
Drug · Other
Lead sponsor
Mandos LLC
Industry
Eligibility
4 Years to 21 Years
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
14
States / cities
Birmingham, Alabama • Orange, California • San Francisco, California + 11 more
Source: ClinicalTrials.gov public record
Updated Feb 22, 2023 · Synced Oct 2, 2026, 6:00 AM EDT
Not listed Phase 3 Interventional

Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1

NCT04860960
View directory record Official record
Conditions
Niemann-Pick Disease, Type C1
Interventions
Hydroxypropyl-beta-cyclodextrin, Placebo
Drug
Lead sponsor
Cyclo Therapeutics, Inc.
Industry
Eligibility
3 Years and older
Enrollment
94 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
7
States / cities
Oakland, California • Jacksonville, Florida • Atlanta, Georgia + 4 more
Source: ClinicalTrials.gov public record
Updated Jun 4, 2024 · Synced Oct 2, 2026, 6:00 AM EDT
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 18, 2019 · Synced Oct 2, 2026, 6:00 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 28, 2026 · Synced Oct 2, 2026, 6:00 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 21, 2021 · Synced Oct 2, 2026, 6:00 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C

NCT03471143
View directory record Official record
Conditions
Niemann-Pick Disease, Type C
Interventions
VTS-270
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
Up to 6 Months
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Jan 5, 2026 · Synced Oct 2, 2026, 6:00 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 6, 2016 · Synced Oct 2, 2026, 6:00 AM EDT
Completed Phase 1 Interventional

Saccadic Eye Movements in Patients With Niemann-Pick Type C Disease

NCT00316498
View directory record Official record
Conditions
Niemann Pick Diseases
Interventions
OGT918
Drug
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
12 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2007
U.S. locations
2
States / cities
Bethesda, Maryland • New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 2, 2026, 6:00 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine

NCT00975689
View directory record Official record
Conditions
Niemann-Pick Disease, Type C
Interventions
N-Acetyl Cysteine
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
12 Months and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 6, 2013 · Synced Oct 2, 2026, 6:00 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 18, 2021 · Synced Oct 2, 2026, 6:00 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 17, 2024 · Synced Oct 2, 2026, 6:00 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1

NCT03887533
View directory record Official record
Conditions
Niemann-Pick Disease, Type C1
Interventions
VTS-270
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
3 Years to 60 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 30, 2022 · Synced Oct 2, 2026, 6:00 AM EDT
Active, not recruiting Phase 3 Interventional

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis

NCT07082543
View directory record Official record
Conditions
Gangliosidoses, GM2, Gangliosidosis, GM1
Interventions
AZ-3102, Placebo
Drug
Lead sponsor
Azafaros B.V.
Industry
Eligibility
4 Years and older
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
5
States / cities
Oakland, California • Minneapolis, Minnesota • Rochester, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 22, 2026 · Synced Oct 2, 2026, 6:00 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 3, 2026 · Synced Oct 2, 2026, 6:00 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 23, 2021 · Synced Oct 2, 2026, 6:00 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Phase 1/2 Study of Vorinostat Therapy in Niemann-Pick Disease, Type C1

NCT02124083
View directory record Official record
Conditions
Neimann-Pick Disease
Interventions
Vorinostat
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
18 Years to 60 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Feb 22, 2018 · Synced Oct 2, 2026, 6:00 AM EDT
Completed No phase listed Observational

Functional Near-Infrared Spectroscopy (fNIRS) Combined With Diffuse Correlation Spectroscopy (DCS) in Neurocognitive Disease as Compared to Healthy Neurotypical Controls

NCT05642221
View directory record Official record
Conditions
Juvenile Neuronal Ceroid Lipofuscinosis, Smith-Lemli-Opitz Syndrome, Creatine Transporter Deficiency, Niemann-Pick Disease Type C1
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
6 Months to 110 Years
Enrollment
73 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 16, 2026 · Synced Oct 2, 2026, 6:00 AM EDT
Recruiting Phase 3 Interventional

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)

NCT07082725
View directory record Official record
Conditions
Niemann-Pick Type C Disease
Interventions
Nizubaglustat, Placebo
Drug
Lead sponsor
Azafaros B.V.
Industry
Eligibility
4 Years and older
Enrollment
72 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
5
States / cities
Oakland, California • Minneapolis, Minnesota • Rochester, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 28, 2026 · Synced Oct 2, 2026, 6:00 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C

NCT02612129
View directory record Official record
Conditions
Niemann-Pick Disease, Type C
Interventions
arimoclomol, Placebo
Drug
Lead sponsor
ZevraDenmark
Industry
Eligibility
2 Years to 18 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2024
U.S. locations
2
States / cities
Oakland, California • Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Nov 29, 2024 · Synced Oct 2, 2026, 6:00 AM EDT
Recruiting Phase 3 Interventional

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis

NCT07054515
View directory record Official record
Conditions
Niemann-Pick Type C Disease, GM1 Gangliosidosis, GM2 Gangliosidosis
Interventions
AZ-3102, Placebo
Drug
Lead sponsor
Azafaros B.V.
Industry
Eligibility
4 Years and older
Enrollment
147 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
5
States / cities
Oakland, California • Minneapolis, Minnesota • Rochester, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 24, 2026 · Synced Oct 2, 2026, 6:00 AM EDT