6 completed, 3 recruiting, 3 terminated, 1 not yet recruiting. 14 Phase 3, 8 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 14 | Completed 6, Recruiting 3, Terminated 3, Not yet recruiting 1, Unknown 1 |
| Phase 2 | 8 | Completed 5, Terminated 2, Not yet recruiting 1 |
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 Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis AZ-3102, Placebo - Niemann-Pick Type C Disease, GM1 Gangliosidosis | Phase 3 | Recruiting | Azafaros B.V. | 147 | 2025-06-30 | 2026-10-27 |
| 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) Nizubaglustat, Placebo - Niemann-Pick Type C Disease | Phase 3 | Recruiting | Azafaros B.V. | 72 | 2025-06-30 | 2026-10-28 |
| A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C N-Acetyl-L-Leucine, Placebo - Niemann-Pick Disease, Type C | Phase 3 | Recruiting | IntraBio Inc | 53 | 2022-06-30 | 2026-04-03 |
| Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1 Hydroxypropyl-beta-cyclodextrin, Placebo - Niemann-Pick Disease, Type C1 | Phase 3 | Unknown | Cyclo Therapeutics, Inc. | 94 | 2021-07-20 | 2024-06-04 |
| 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 |
| Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System Adrabetadex - Niemann-Pick Type C Disease | Phase 3 | Terminated | Mandos LLC | 27 | 2018-06-18 | 2024-01-17 |
| 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, 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 |
| Application of Miglustat in Patients With Niemann-Pick Type C Miglustat - Niemann-Pick Disease Type C | Phase 3 | Completed | National Taiwan University Hospital | 5 | 2008-01-01 | 2013-01-04 |
| Stem Cell Transplant for Inborn Errors of Metabolism Stem Cell Transplant, Busulfan, Cyclophosphamide, Antithymocyte Globulin - Adrenoleukodystrophy, Metachromatic Leukodystrophy | Phase 2 / Phase 3 | Completed | Masonic Cancer Center, University of Minnesota | 135 | 1995-01-01 | 2017-12-28 |
Niemann-Pick Disease, Type C (11) · Gangliosidosis, GM1 (3) · Cognitive Dysfunction (2) · Deglutition Disorders (2) · Disease (2) · Gangliosidoses, GM2 (2) · Gaucher Disease (2) · Mobility Limitation (2) · Wolman Disease (2) · Adrenoleukodystrophy (1) · Chanarin-Dorfman Syndrome (1) · Cholesterol Ester Storage Disease (1) · Cirrhosis, Cryptogenic (1) · Fucosidosis (1)
Brain Diseases (14) · Brain Diseases, Metabolic (14) · Brain Diseases, Metabolic, Inborn (14) · Central Nervous System Diseases (14) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (14) · Genetic Diseases, Inborn (14) · Hemic and Lymphatic Diseases (14) · Histiocytosis (14) · Histiocytosis, Non-Langerhans-Cell (14) · Lipid Metabolism Disorders (14) · Lipid Metabolism, Inborn Errors (14) · Lipidoses (14) · Lymphatic Diseases (14) · Lysosomal Storage Diseases, Nervous System (14) · Metabolic Diseases (14)
2-Hydroxypropyl-beta-cyclodextrin (5) · Cyclodextrins (3) · Antilymphocyte Serum (1) · Bone Marrow Transplantation (1) · Busulfan (1) · Cyclophosphamide (1) · IB1001 (1) · Sebelipase alfa (1) · Sodium Chloride (1) · Stem Cell Transplantation (1) · acetylleucine (1) · arimoclomol (1) · miglustat (1) · olipudase alfa (1)
Mandos LLC (4) · Azafaros B.V. (3) · Alexion Pharmaceuticals, Inc. (1) · Cyclo Therapeutics, Inc. (1) · Genzyme, a Sanofi Company (1) · IntraBio Inc (1) · Masonic Cancer Center, University of Minnesota (1) · National Taiwan University Hospital (1) · ZevraDenmark (1)
United States (11) · United Kingdom (9) · Germany (8) · Australia (7) · France (6) · Spain (6) · Turkey (Türkiye) (6) · Italy (5) · Switzerland (5) · Argentina (4) · Brazil (4) · Portugal (3) · Canada (2) · India (2) · Mexico (2)
Karen Mullen, MD (1) · Karl-Eugen Mengel (1) · Paul Orchard, MD (1) · Whu-Liang Hwu, MD, PhD (1)
PMID 21586746, PMID 22476655, PMID 30115089, PMID 33738443, PMID 34387740, PMID 34418116, PMID 36279795, PMID 38042851, PMID 38294974, PMID 39774103, PMID 40513057, PMID 40525490, PMID 40739200
2015 (3)