16 completed, 6 terminated, 1 active not recruiting, 1 not yet recruiting. 25 Phase 2, 8 Phase 1, 8 Phase 3.
Follow Niemann-Pick Diseases: new trials by email Literature Review of Niemann-Pick Diseases trials Research Report on Niemann-Pick Diseases
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Long-Term Safety and Tolerability of Nizubaglustat in Participants With Rare Lysosomal Disorders AZ-3102 - Niemann-Pick Disease Type C, GM2 Gangliosidosis | Phase 2 / Phase 3 | Not yet recruiting | Azafaros B.V. | 218 | 2026-12-10 | 2026-10-21 |
| A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease AZ-3102 - GM2 Gangliosidosis, Niemann-Pick Type C Disease | Phase 2 | Active not recruiting | Azafaros B.V. | 21 | 2026-02-04 | 2026-10-21 |
| Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease AZ-3102 (Dose 1), Placebo, AZ-3102 (Dose 2) - GM2 Gangliosidosis, Niemann-Pick Disease, Type C | Phase 2 | Completed | Azafaros B.V. | 13 | 2023-04-24 | 2026-05-14 |
| A Study to Evaluate Safety and Tolerability of Olipudase Alfa in Pediatric and Adult Participants With Acid Sphingomyelinase Deficiency (ASMD) Who Completed the DFI12712 or the LTS13632 Study in France Olipudase alfa - Acid Sphingomyelinase Deficiency | Phase 2 | Completed | Sanofi | 3 | 2021-11-18 | 2026-04-13 |
| Safety and Tolerability Study of VTS-270 in Pediatric Participants With Niemann-Pick Type C (NPC) Disease VTS-270 - Niemann-Pick Disease, Type C | Phase 2 | Withdrawn | Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company | 2020-05-01 | 2020-07-22 | |
| Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1 VTS-270 - Niemann-Pick Disease, Type C1 | Phase 1 / Phase 2 | Terminated | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 2 | 2020-01-06 | 2022-08-30 |
| Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1 VTS-270 - Niemann-Pick Disease, Type C | Phase 2 / Phase 3 | Terminated | Mandos LLC | 2 | 2019-12-02 | 2023-12-26 |
| N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC) IB1001 - Niemann-Pick Disease, Type C | Phase 2 | Completed | IntraBio Inc | 33 | 2019-09-04 | 2023-11-28 |
| Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C VTS-270 - Niemann-Pick Disease, Type C | Phase 1 / Phase 2 | Completed | Washington University School of Medicine | 4 | 2019-02-22 | 2026-01-05 |
| Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C Patients Hydroxypropyl-beta-cyclodextrin - Niemann-Pick Disease, Type C1 | Phase 1 / Phase 2 | Completed | Cyclo Therapeutics, Inc. | 12 | 2017-03-20 | 2021-08-10 |
| Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C arimoclomol, Placebo - Niemann-Pick Disease, Type C | Phase 2 / Phase 3 | Completed | ZevraDenmark | 50 | 2016-06-14 | 2024-11-29 |
| Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease Adrabetadex - Niemann-Pick Disease, Type C | Phase 2 / Phase 3 | Terminated | Mandos LLC | 66 | 2015-12-23 | 2023-08-29 |
| Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency placebo (saline), Olipudase alfa - Sphingomyelin Lipidosis | Phase 2 / Phase 3 | Completed | Genzyme, a Sanofi Company | 36 | 2015-12-18 | 2024-11-07 |
| VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease Parts A/B: Adrabetadex, Parts A/B: Sham Control - Niemann-Pick Disease, Type C | Phase 2 / Phase 3 | Completed | Mandos LLC | 56 | 2015-10-01 | 2023-02-22 |
| Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency Olipudase alfa - Sphingomyelin Lipidosis | Phase 1 / Phase 2 | Completed | Genzyme, a Sanofi Company | 20 | 2015-05-01 | 2022-03-23 |
| Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency Sebelipase Alfa - Lysosomal Acid Lipase Deficiency | Phase 2 | Terminated | Alexion Pharmaceuticals, Inc. | 10 | 2014-06-06 | 2019-11-18 |
| Phase 1/2 Study of Vorinostat Therapy in Niemann-Pick Disease, Type C1 Vorinostat - Neimann-Pick Disease | Phase 1 / Phase 2 | Completed | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 12 | 2014-04-25 | 2018-02-22 |
| A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency GZ402665 - Sphingomyelin Lipidosis | Phase 2 | Completed | Genzyme, a Sanofi Company | 25 | 2013-12-04 | 2024-07-03 |
| Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency Sebelipase alfa (SBC-102) - Lysosomal Acid Lipase Deficiency, Wolman Disease | Phase 2 / Phase 3 | Completed | Alexion Pharmaceuticals, Inc. | 9 | 2011-05-04 | 2019-01-30 |
| Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders hematopoietic stem cell infusion - Hurler Syndrome (MPS I), Hurler-Scheie Syndrome | Phase 1 / Phase 2 | Terminated | Talaris Therapeutics Inc. | 3 | 2011-04-01 | 2023-04-12 |
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Niemann-Pick Disease, Type C (16) · Niemann-Pick Disease, Type A (4) · Wolman Disease (4) · Fucosidosis (3) · Gangliosidoses, GM2 (3) · Leukodystrophy, Globoid Cell (3) · Leukodystrophy, Metachromatic (3) · Mucopolysaccharidosis I (3) · Adrenoleukodystrophy (2) · Chanarin-Dorfman Syndrome (2) · Cholesterol Ester Storage Disease (2) · Cirrhosis, Cryptogenic (2) · Cognitive Dysfunction (2) · Deglutition Disorders (2)
Brain Diseases (25) · Brain Diseases, Metabolic (25) · Brain Diseases, Metabolic, Inborn (25) · Central Nervous System Diseases (25) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (25) · Hemic and Lymphatic Diseases (25) · Histiocytosis (25) · Histiocytosis, Non-Langerhans-Cell (25) · Lipid Metabolism Disorders (25) · Lipid Metabolism, Inborn Errors (25) · Lipidoses (25) · Lymphatic Diseases (25) · Lysosomal Storage Diseases, Nervous System (25) · Metabolic Diseases (25) · Nervous System Diseases (25)
2-Hydroxypropyl-beta-cyclodextrin (5) · olipudase alfa (4) · Cyclodextrins (3) · Stem Cell Transplantation (3) · Bone Marrow Transplantation (2) · Busulfan (2) · Cyclophosphamide (2) · Sebelipase alfa (2) · Acetylcysteine (1) · Alemtuzumab (1) · Antilymphocyte Serum (1) · Hematopoietic Stem Cell Transplantation (1) · IB1001 (1) · Sodium Chloride (1) · Vorinostat (1)
Azafaros B.V. (3) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (3) · Genzyme, a Sanofi Company (3) · Mandos LLC (3) · Alexion Pharmaceuticals, Inc. (2) · Masonic Cancer Center, University of Minnesota (2) · Actelion (1) · Children's Hospital Los Angeles (1) · Cyclo Therapeutics, Inc. (1) · IntraBio Inc (1) · Sanofi (1) · Talaris Therapeutics Inc. (1) · Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company (1) · Washington University School of Medicine (1) · ZevraDenmark (1)
United States (17) · United Kingdom (10) · France (8) · Germany (7) · Brazil (5) · Italy (5) · Spain (4) · Australia (3) · Turkey (Türkiye) (3) · Belgium (2) · New Zealand (2) · Singapore (2) · Switzerland (2) · Argentina (1) · Bulgaria (1)
Forbes D Porter, M.D. (3) · Paul Orchard, MD (2) · Claudia Chiriboga, Assoc. Prof. MD (1) · Hisham Abdel-Azim, MD (1) · Karl-Eugen Mengel (1) · Patricia I Dickson, MD (1) · Reena Sharma, MB BS (1) · Suzanne T Ildstad, MD (1)
PMID 21586746, PMID 29305734, PMID 32620536, PMID 33482890, PMID 33875845, PMID 34349180, PMID 34387740, PMID 34389161, PMID 34418116, PMID 37098529, PMID 37670901, PMID 38042851, PMID 39774103, PMID 40525490, PMID 40937531
2015 (3), U01HD090845 (1)