48 completed, 9 terminated, 8 recruiting, 6 active not recruiting. 80 Phase 3, 12 Phase 2.
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 |
|---|---|---|---|---|---|---|
| The Efficacy and Safety of Venglustat Compared With Cerezyme in Pediatric Participants 2 to 11 Years of Age With Gaucher Disease Type 3 (GD3) Venglustat, Imiglucerase - Gaucher's Disease Type III | Phase 3 | Not yet recruiting | Sanofi | 18 | 2026-12-11 | 2026-11-10 |
| 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 Learn How Well Lucerastat Works and How Safe it is in Untreated Adult Male Participants With Fabry Disease Lucerastat - Fabry Disease | Phase 3 | Recruiting | Idorsia Pharmaceuticals Ltd. | 16 | 2026-12-01 | 2026-11-03 |
| Safety, Tolerability, and Efficacy of PLX-200 in Patients With CLN3 PLX-200, Placebo - Juvenile Neuronal Ceroid Lipofuscinosis | Phase 3 | Withdrawn | Polaryx Therapeutics, Inc. | 2026-11-01 | 2026-08-19 | |
| Study of Velaglucerase Beta ERT in Children With Gaucher Disease Velaglucerase beta - Gaucher Disease, Gaucher Disease Type 1 | Phase 3 | Not yet recruiting | CANbridge Life Sciences Ltd. | 10 | 2026-09-16 | 2026-09-29 |
| A Gaucher Disease Gene Therapy Trial With FLT201 FLT201 - Gaucher Disease Type 1 | Phase 3 | Recruiting | Spur Therapeutics | 45 | 2026-04-07 | 2026-10-14 |
| A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants Migalastat HCl 20 mg - Fabry Disease | Phase 3 | Recruiting | Amicus Therapeutics | 8 | 2026-01-08 | 2026-10-22 |
| 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 |
| 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 AZ-3102, Placebo - Gangliosidoses, GM2, Gangliosidosis, GM1 | Phase 3 | Active not recruiting | Azafaros B.V. | 75 | 2025-06-30 | 2026-09-22 |
| 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 |
| 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 |
| Evaluate the Safety and Efficacy of Fabagal® (Agalsidase Beta) in Patients With Fabry Disease Fabagal® (Agalsidase beta), Active comparator (Agalsidase beta) - Fabry Disease | Phase 3 | Unknown | ISU Abxis Co., Ltd. | 24 | 2023-04-14 | 2024-07-29 |
| Role of Vitamin E in Gaucher Disease Patients Vitamin E - Gaucher Disease | Phase 3 | Completed | Ain Shams University | 40 | 2023-03-03 | 2024-01-18 |
| A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease Velaglucerase Alfa - Gaucher Disease | Phase 3 | Completed | Takeda | 20 | 2023-01-03 | 2025-06-26 |
| Switch Over Study of Biosimilar Agalsidase Beta for Fabry Disease Recombinant human alpha galactosidase A (agalsidase beta), Recombinant human alpha-galactosidase A (agalsidase beta) - Fabry Disease | Phase 3 | Completed | Bio Sidus SA | 20 | 2022-12-13 | 2026-02-18 |
| A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease migalastat HCl 150 mg - Fabry Disease | Phase 3 | Active not recruiting | Amicus Therapeutics | 14 | 2022-10-31 | 2026-07-06 |
| 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 |
| A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease Venglustat (GZ402671), Agalsidase alfa, Agalsidase beta (GZ419828) - Fabry Disease | Phase 3 | Active not recruiting | Sanofi | 104 | 2022-05-03 | 2026-06-24 |
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Fabry Disease (30) · Gaucher Disease (28) · Niemann-Pick Disease, Type C (11) · Gangliosidosis, GM1 (6) · Lysosomal Storage Diseases (6) · Gangliosidoses, GM2 (5) · Sandhoff Disease (5) · Tay-Sachs Disease (4) · Cognitive Dysfunction (3) · Leukodystrophy, Metachromatic (3) · Proteinuria (3) · Wolman Disease (3) · Adrenoleukodystrophy (2) · Cholesterol Ester Storage Disease (2) · Deglutition Disorders (2)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (80) · Genetic Diseases, Inborn (80) · Lipid Metabolism Disorders (80) · Lipid Metabolism, Inborn Errors (80) · Metabolic Diseases (80) · Nutritional and Metabolic Diseases (80) · Metabolism, Inborn Errors (79) · Nervous System Diseases (78) · Brain Diseases (77) · Brain Diseases, Metabolic (77) · Brain Diseases, Metabolic, Inborn (77) · Central Nervous System Diseases (77) · Lysosomal Storage Diseases, Nervous System (77) · Sphingolipidoses (77)
migalastat (12) · imiglucerase (8) · larazotide acetate (8) · Glucosylceramidase (7) · agalsidase alfa (7) · agalsidase beta (7) · taliglucerase alfa (6) · 2-Hydroxypropyl-beta-cyclodextrin (5) · eliglustat (5) · miglustat (4) · venglustat (4) · Cyclodextrins (3) · Sebelipase alfa (2) · Sodium Chloride (2) · Antilymphocyte Serum (1)
Shire (10) · Amicus Therapeutics (8) · Genzyme, a Sanofi Company (8) · Pfizer (5) · Sanofi (5) · Azafaros B.V. (4) · Chiesi Farmaceutici S.p.A. (4) · Mandos LLC (4) · Protalix (4) · ISU Abxis Co., Ltd. (3) · Idorsia Pharmaceuticals Ltd. (3) · Alexion Pharmaceuticals, Inc. (2) · Takeda (2) · AVROBIO (1) · Actelion (1)
United States (50) · United Kingdom (45) · Spain (30) · Australia (26) · Canada (26) · France (25) · Italy (24) · Argentina (20) · Germany (20) · Turkey (Türkiye) (17) · Brazil (15) · Japan (15) · Poland (14) · Czechia (12) · Israel (11)
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