307 completed, 92 recruiting, 81 unknown, 50 terminated. 136 Phase 2, 108 Phase 1, 80 Phase 3, 59 Not applicable, 27 Phase 4.
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 |
|---|---|---|---|---|---|---|
| Lentiviral Hematopoietic Stem Cell Gene Therapy for MLD Lentiviral TYF-ARSA correction of patient's autologous HSCs - Metachromatic Leukodystrophy (MLD) | Not applicable | Not yet recruiting | Shenzhen Geno-Immune Medical Institute | 10 | 2027-06-01 | 2026-11-02 |
| Direct Lentiviral Injection Gene Therapy for MLD Intrathecal and intravenous LV gene therapy - Metachromatic Leukodystrophy (MLD) | Not applicable | Not yet recruiting | Shenzhen Geno-Immune Medical Institute | 10 | 2027-05-31 | 2026-11-02 |
| A Survey to Assess Participants', Caregivers', and Nurses' Use and Understanding of Educational Material on Velaglucerase Alfa (VPRIV) Home Infusion Gaucher Disease | Not yet recruiting | Takeda | 60 | 2027-01-31 | 2026-10-01 | |
| 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 |
| Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis Clinical Trial Gene Transfer with an AAV9 Vector Expressing Human ß-galactosidase - GM1 Gangliosidoses, GM1 Gangliosidosis, Type I | Phase 1 | Not yet recruiting | Tippi Mackenzie | 5 | 2027-01-01 | 2026-08-31 |
| 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 |
| 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 |
| 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 |
| 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 | |
| Therapeutic Implications of CMR Low Native T1 in Non-treated Non-hypertrophic Females With Fabry Disease (FD) Decision tree for initiating treatment - Fabry Disease | Not applicable | Not yet recruiting | University Hospital, Bordeaux | 60 | 2026-11-01 | 2026-10-14 |
| Nutritional Status of Gaucher Disease Patients Gaucher Disease | Not yet recruiting | Assiut University | 84 | 2026-09-26 | 2026-09-28 | |
| Parkinson's Disease in Type 1 Gaucher Disease Parkinson Disease (PD), Type 1 Gaucher Disease | Recruiting | Icahn School of Medicine at Mount Sinai | 100 | 2026-09-21 | 2026-11-05 | |
| 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 |
| Quality of Life in Adults With Untreated Fabry Disease in Sweden (QoLUF) Qualitative Interview - Fabry Disease | Enrolling by invitation | Vastra Gotaland Region | 35 | 2026-08-21 | 2026-08-27 | |
| 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 |
| Observational Study on Long-term Use of Pegunigalsidase Alfa in Fabry Patients in a Real-world Setting pegunigalsidase alfa - Fabry Disease | Recruiting | Chiesi Italia | 75 | 2026-06-15 | 2026-07-08 | |
| 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 |
| Advancing Integrated Therapies for Gaucher Disease Gaucher Disease | Recruiting | Yale University | 30 | 2026-05-06 | 2026-08-11 |
Showing 20 of 642. Sign in to see all of them.
Fabry Disease (243) · Gaucher Disease (176) · Lysosomal Storage Diseases (62) · Leukodystrophy, Metachromatic (49) · Neuronal Ceroid-Lipofuscinoses (49) · Niemann-Pick Disease, Type C (37) · Wolman Disease (33) · Leukodystrophy, Globoid Cell (31) · Niemann-Pick Diseases (27) · Gangliosidoses, GM2 (26) · Sandhoff Disease (24) · Tay-Sachs Disease (24) · Metabolism, Inborn Errors (23) · Sphingolipidoses (23) · Gangliosidosis, GM1 (22)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (642) · Nutritional and Metabolic Diseases (642) · Lipid Metabolism, Inborn Errors (639) · Lipid Metabolism Disorders (634) · Genetic Diseases, Inborn (628) · Metabolic Diseases (627) · Metabolism, Inborn Errors (619) · Nervous System Diseases (601) · Brain Diseases (566) · Central Nervous System Diseases (564) · Lysosomal Storage Diseases, Nervous System (562) · Brain Diseases, Metabolic (561) · Brain Diseases, Metabolic, Inborn (560) · Sphingolipidoses (540)
agalsidase beta (28) · migalastat (28) · agalsidase alfa (24) · larazotide acetate (19) · Glucosylceramidase (14) · eliglustat (14) · imiglucerase (14) · miglustat (11) · taliglucerase alfa (11) · 2-Hydroxypropyl-beta-cyclodextrin (9) · Blood Specimen Collection (9) · Sebelipase alfa (8) · Enzyme Replacement Therapy (7) · olipudase alfa (7) · venglustat (7)
Genzyme, a Sanofi Company (34) · Shire (34) · Amicus Therapeutics (29) · Sanofi (23) · National Institute of Neurological Disorders and Stroke (NINDS) (18) · CENTOGENE GmbH Rostock (15) · Alexion Pharmaceuticals, Inc. (12) · University of Minnesota (11) · Takeda (10) · Protalix (9) · Azafaros B.V. (8) · Chiesi Farmaceutici S.p.A. (8) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (8) · Pfizer (8) · Assistance Publique - Hôpitaux de Paris (7)
United States (318) · United Kingdom (112) · France (94) · Germany (79) · Italy (71) · Canada (68) · Spain (60) · Australia (49) · Brazil (44) · Israel (39) · China (34) · Turkey (Türkiye) (32) · Argentina (31) · Denmark (31) · Poland (29)
Ozlem Goker-Alpan, MD (7) · Peter Bauer, Prof. (7) · Forbes D Porter, M.D. (6) · Joanne Kurtzberg, MD (5) · Paul Orchard, MD (4) · Ari Zimran, MD (3) · Arndt Rolfs, MD (3)
PMID 27657681, PMID 21586746, PMID 27839979, PMID 33332761, PMID 36279795, PMID 37624739, PMID 39847314, PMID 40525490, PMID 18473686, PMID 23176611, PMID 23348766, PMID 23860343, PMID 25795794, PMID 26053270, PMID 27509102, PMID 28476546, PMID 29688815, PMID 31889231, PMID 32994552, PMID 33482890, PMID 33980287, PMID 34349180, PMID 34387740, PMID 36196048, PMID 36461157, PMID 36481125, PMID 36739645, PMID 37171121, PMID 38101904, PMID 38776275, PMID 39745261, PMID 39774103, PMID 40155993, PMID 40267426, PMID 40817303, PMID 41527340, PMID 10665494, PMID 11231629, PMID 11399225, PMID 14578207, PMID 15100373, PMID 15324318, PMID 1603628, PMID 16546416, PMID 17179052, PMID 17230068, PMID 17634524, PMID 17656478, PMID 17679671, PMID 18346516
U54NS065768 (12), 2019 (5), 2008 (4), 2014 (4), 2015 (4), 2009 (3), 2018 (3), 2017 (2), 2023 (2), 2025 (2), 2005 (1), 2016 (1), K12CA120780 (1), R01HD073292 (1), R01HD104814 (1)