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ClinicalTrials.gov public records Last synced Nov 7, 2026, 7:49 AM EST

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Showing 1–24 of 47 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Neimann-Pick Disease Clear all

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

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

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

Conditions: Lysosomal Acid Lipase Deficiency, Wolman Disease
Interventions: Sebelipase alfa (SBC-102); Drug
Lead sponsor: Alexion Pharmaceuticals, Inc.; Industry
Eligibility: Up to 24 Months
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2018
U.S. locations: 1
States / cities: Irvine, California

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

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

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

Conditions: Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Adrenoleukodystrophy, Niemann-Pick Disease, Metachromatic Leukodystrophy, Wolman Disease, Krabbe's Disease, Gaucher's Disease, Fucosidosis, Batten Disease, Severe Aplastic Anemia, Diamond-Blackfan Anemia, Amegakaryocytic Thrombocytopenia, Myelodysplastic Syndrome, Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia
Interventions: Human Placental Derived Stem Cell; Drug
Lead sponsor: New York Medical College; Other
Eligibility: 0 Years to 55 Years
Enrollment: 43 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2022
U.S. locations: 3
States / cities: Denver, Colorado • Valhalla, New York • Salt Lake City, Utah

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

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

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

Conditions: Sphingomyelin Lipidosis
Interventions: Investigational Procedures; Procedure
Lead sponsor: Sanofi; Industry
Eligibility: Not listed
Enrollment: 84 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2023
U.S. locations: 3
States / cities: Atlanta, Georgia • The Bronx, New York • Valhalla, New York

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

Conditions: Niemann-Pick Diseases
Interventions: Not listed
Lead sponsor: National Niemann-Pick Disease Foundation; Other
Eligibility: 18 Years and older
Enrollment: 76 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020
U.S. locations: 1
States / cities: New York, New York

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

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

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

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

Conditions: MLD, Krabbe Disease, ALD, MPS I, MPS II, MPS III, Vanishing White Matter Disease, GM3 Gangliosidosis, PKAN, Tay-Sachs Disease, NP Deficiency, Osteopetrosis, Alpha-Mannosidosis, Sandhoff Disease, Niemann-Pick Diseases, MPS IV, Gaucher Disease, GAN, GM1 Gangliosidoses, Morquio Disease, S-Adenosylhomocysteine Hydrolase Deficiency, Batten Disease, Pelizaeus-Merzbacher Disease, Leukodystrophy, Lysosomal Storage Diseases, Purine Nucleoside Phosphorylase Deficiency, Multiple Sulfatase Deficiency Disease
Interventions: Palliative Care, Hematopoetic Stem Cell Transplantation; Other · Biological
Lead sponsor: University of Pittsburgh; Other
Eligibility: Not listed
Enrollment: 1,500 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2035
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

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

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

Conditions: Lysosomal Storage Disorders, Gaucher Disease, Fabry Disease, Pompe Disease, Niemann-Pick Disease
Interventions: Not listed
Lead sponsor: O & O Alpan LLC; Other
Eligibility: 1 Day to 100 Years
Enrollment: 20,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2014 – 2018
U.S. locations: 1
States / cities: Fairfax, Virginia

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

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

Search by objective public record fields.

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

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

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

Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency

Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease

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

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

Human Placental-Derived Stem Cell Transplantation

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

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

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

A Prospective and Retrospective Cohort Study in Patients With Chronic Forms of Acid Sphingomyelinase Deficiency (ASMD)

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

Health Insurance Literacy and Challenges in Accessing Health Services in Niemann-Pick

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)

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

Stem Cell Transplant for Inborn Errors of Metabolism

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

Longitudinal Study of Neurodegenerative Disorders

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

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

Investigating Lysosomal Storage Diseases in Minority Groups

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

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