76 completed, 16 terminated, 14 active not recruiting, 8 recruiting. 136 Phase 2, 58 Phase 1, 12 Phase 3.
Follow Lipidoses: new trials by email Literature Review of Lipidoses trials Research Report on Lipidoses
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 |
|---|---|---|---|---|---|---|
| Study to Evaluate the Efficacy and Safety of Nizubaglustat (AZ-3102) in Patients With Gaucher Disease Type 3 (GD3) AZ-3102 - Gaucher Disease Type 3 | Phase 2 | Not yet recruiting | Azafaros B.V. | 20 | 2027-01-25 | 2026-11-12 |
| 4SCAR-T Therapy Targeting GD2, PSMA and CD276 for Treating Neuroblastoma GD2, PSMA and CD276 CAR-T cells - Neuroblastoma | Phase 1 / Phase 2 | Not yet recruiting | Shenzhen Geno-Immune Medical Institute | 100 | 2026-12-31 | 2026-10-30 |
| 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 |
| Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol) PLX-200 - Lysosomal Storage Disorders, Sandhoff Disease | Phase 2 | Not yet recruiting | Polaryx Therapeutics, Inc. | 24 | 2026-12-01 | 2026-07-31 |
| Gene Therapy Trial for CLN6 Batten Disease scAAV9.CB.CLN6 (dose: 1.5E14 vector genomes) - CLN6, Batten Disease | Phase 1 / Phase 2 | Not yet recruiting | The Charlotte and Gwenyth Gray Foundation | 12 | 2026-08-01 | 2026-05-14 |
| A Phase I/II Clinical Study to Evaluate the Safety and Efficacy of VGN-R08b in Patients With Type III Gaucher's Disease VGN-R08b - Gaucher Disease Type 3 | Phase 1 / Phase 2 | Not yet recruiting | Shanghai Vitalgen BioPharma Co., Ltd. | 12 | 2026-05-14 | 2026-05-22 |
| 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 |
| A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease PRX-102 1 mg/kg every two weeks - Fabry Disease | Phase 2 / Phase 3 | Recruiting | Chiesi Farmaceutici S.p.A. | 22 | 2025-07-29 | 2026-08-10 |
| The Safety and Efficacy of Intravenous EXG110 in Patients With Fabry Disease EXG110 Injection - Fabry Disease | Phase 1 / Phase 2 | Not yet recruiting | Guangzhou Jiayin Biotech Ltd | 16 | 2025-03-01 | 2025-02-11 |
| Efficacy and Safety of Eliglustat in Chinese Pediatric Patients With Gaucher Disease Type 1 and Type 3 Eliglustat Tartrate Capsules - Gaucher Disease | Phase 2 | Unknown | Peking Union Medical College Hospital | 5 | 2024-08-01 | 2024-07-26 |
| A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients With Type I Gaucher Disease LY-M001 - Gaucher Disease Type 1 | Phase 1 / Phase 2 | Recruiting | Lingyi Biotech Co., Ltd. | 12 | 2024-07-05 | 2026-01-26 |
| Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease AMT-191 - Fabry Disease | Phase 1 / Phase 2 | Recruiting | UniQure Biopharma B.V. | 20 | 2024-06-05 | 2026-09-24 |
| Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2) FLT201 - Gaucher Disease, Type 1 | Phase 1 / Phase 2 | Active not recruiting | Spur Therapeutics | 75 | 2024-05-13 | 2025-11-04 |
| Study of the Safety and Biologic Activity of AL01211 in Treatment Naive Males With Classic Fabry Disease AL01211 - Fabry Disease | Phase 2 | Unknown | AceLink Therapeutics, Inc. | 16 | 2023-10-25 | 2024-07-30 |
| An Efficacy and Safety Study of AVR-RD-02 Compared to Enzyme Replacement Therapy for Treatment of Gaucher Disease Type 3 Gene therapy, Enzyme Replacement Agent - Gaucher Disease, Type 3 | Phase 2 / Phase 3 | Withdrawn | AVROBIO | 2023-10-01 | 2023-08-09 | |
| Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease PRX-102 1 mg/kg every 2 weeks, PRX-102 2 mg/kg every 4 weeks - Fabry Disease | Phase 2 / Phase 3 | Recruiting | Chiesi Farmaceutici S.p.A. | 16 | 2023-08-01 | 2026-03-18 |
| Effects Of Sodium Glucose Cotranspoter 2 Inhibitors On Heart And Kidneys In Fabry Disease Patients Dapagliflozin 10mg Tab, Placebo - Fabry Disease | Phase 2 | Unknown | Albina Nowak, MD | 46 | 2023-08-01 | 2023-02-02 |
| Safety and Efficacy of SMART101 in Adult Patients With Hematological Malignancies After Haploidentical HSCT With Post-transplant Cyclophosphamide Allogeneic T cell progenitors, cultured ex-vivo - Hematological Malignancies | Phase 1 / Phase 2 | Unknown | Smart Immune SAS | 40 | 2023-06-06 | 2023-09-25 |
| A Registrational Study of TTX-381 to Treat Vision Loss in CLN2 Disease TTX-381 - Neuronal Ceroid Lipofuscinosis Type 2 | Phase 1 / Phase 2 | Recruiting | Tern Therapeutics, LLC | 26 | 2023-05-17 | 2026-10-14 |
| 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 |
Showing 20 of 136. Sign in to see all of them.
Fabry Disease (34) · Gaucher Disease (34) · Leukodystrophy, Metachromatic (19) · Lysosomal Storage Diseases (18) · Niemann-Pick Disease, Type C (16) · Neuronal Ceroid-Lipofuscinoses (13) · Leukodystrophy, Globoid Cell (11) · Sphingolipidoses (9) · Mucopolysaccharidosis I (8) · Sandhoff Disease (8) · Wolman Disease (8) · Adrenoleukodystrophy (7) · Gangliosidoses, GM2 (7) · Metabolism, Inborn Errors (7) · Tay-Sachs Disease (7)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (136) · Nutritional and Metabolic Diseases (136) · Lipid Metabolism, Inborn Errors (134) · Lipid Metabolism Disorders (132) · Metabolic Diseases (132) · Genetic Diseases, Inborn (131) · Metabolism, Inborn Errors (129) · Nervous System Diseases (128) · Brain Diseases (118) · Brain Diseases, Metabolic, Inborn (117) · Lysosomal Storage Diseases, Nervous System (117) · Brain Diseases, Metabolic (116) · Central Nervous System Diseases (116) · Sphingolipidoses (109)
agalsidase alfa (7) · larazotide acetate (6) · migalastat (6) · 2-Hydroxypropyl-beta-cyclodextrin (5) · Sebelipase alfa (5) · Stem Cell Transplantation (5) · Alemtuzumab (4) · Glucosylceramidase (4) · agalsidase beta (4) · cerliponase alfa (4) · olipudase alfa (4) · AT2101 (3) · Busulfan (3) · Cyclodextrins (3) · Cyclophosphamide (3)
Shire (11) · Genzyme, a Sanofi Company (10) · Amicus Therapeutics (9) · Alexion Pharmaceuticals, Inc. (5) · Masonic Cancer Center, University of Minnesota (5) · National Institute of Neurological Disorders and Stroke (NINDS) (5) · Azafaros B.V. (4) · AVROBIO (3) · BioMarin Pharmaceutical (3) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (3) · Mandos LLC (3) · Prevail Therapeutics (3) · Protalix (3) · Spur Therapeutics (3) · 4D Molecular Therapeutics (2)
United States (87) · United Kingdom (42) · France (25) · Germany (21) · Australia (15) · Brazil (15) · Canada (15) · Italy (15) · Spain (13) · Israel (12) · China (7) · Denmark (6) · Japan (6) · Poland (6) · Turkey (Türkiye) (6)
Paul Orchard, MD (4) · Forbes D Porter, M.D. (3) · Alan H Cohen, MD (2) · May Orfali, MD (2) · Aabha Nagral, MD (1) · Aaron Tward, MD, PhD (1) · An N. Dang Do, MD, PhD (1) · Anupam Sehgal, MBBS (1) · Ari Zimran (1)
PMID 27657681, PMID 21586746, PMID 23176611, PMID 23348766, PMID 29688815, PMID 33332761, PMID 33482890, PMID 33980287, PMID 34349180, PMID 36196048, PMID 36481125, PMID 37624739, PMID 38101904, PMID 38776275, PMID 40267426, PMID 11231629, PMID 11399225, PMID 18346516, PMID 19595619, PMID 19892975, PMID 20299511, PMID 20439622, PMID 20713962, PMID 23339116, PMID 23474038, PMID 23845948, PMID 23860343, PMID 23966770, PMID 24014509, PMID 24816856, PMID 24835462, PMID 25425121, PMID 25758611, PMID 26252393, PMID 27289174, PMID 27307708, PMID 27866808, PMID 29305734, PMID 30834538, PMID 31184505, PMID 32620536, PMID 32634238, PMID 32657505, PMID 32750127, PMID 32792226, PMID 33875845, PMID 34387740, PMID 34389161, PMID 34418116, PMID 35065785
2015 (3), 2014 (2), 2005 (1), 2008 (1), 2017 (1), 2018 (1), 2023 (1), 2025 (1), U01HD090845 (1), U54NS065768 (1)