58 completed, 13 active not recruiting, 12 terminated, 9 recruiting. 108 Phase 1, 58 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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 |
| 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 |
| A First-in-Human, Single- and Multiple-Ascending Dose Study of YH35995 in Healthy Adult Male Participants YH35995, Placebo - Healthy Participants | Phase 1 | Active not recruiting | Yuhan Corporation | 94 | 2024-07-29 | 2026-07-28 |
| 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 |
| 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 |
| Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCT FBX-101 - Krabbe Disease | Phase 1 / Phase 2 | Active not recruiting | Duke University | 9 | 2023-02-03 | 2026-11-04 |
| ADX-629 Therapy for Sjogren-Larsson Syndrome ADX-629 - Sjogren-Larsson Syndrome | Phase 1 / Phase 2 | Completed | University of Nebraska | 8 | 2023-01-02 | 2026-05-05 |
| A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED) LY3884961 - Gaucher Disease, Gaucher Disease, Type 1 | Phase 1 / Phase 2 | Active not recruiting | Prevail Therapeutics | 8 | 2022-12-20 | 2026-08-17 |
| Safety and Effect of Oral RVX000222 in Subjects With Fabry Disease RVX000222 - Fabry Disease | Phase 1 / Phase 2 | Withdrawn | Resverlogix Corp | 2022-11-22 | 2023-11-18 | |
| 4D-310 in Adults With Fabry Disease and Cardiac Involvement 4D-310 - Fabry Disease | Phase 1 / Phase 2 | Recruiting | 4D Molecular Therapeutics | 18 | 2022-10-16 | 2025-02-21 |
| Phase 1/2 Study of CAN103 in Subjects With Gaucher Disease Low-dose CAN103, High-dose CAN103 - Gaucher Disease, Type 1, Gaucher Disease, Type 3 | Phase 1 / Phase 2 | Unknown | CANbridge (Suzhou) Bio-pharma Co., Ltd. | 40 | 2022-07-11 | 2022-07-27 |
| A Gene Therapy Study in Patients With Gaucher Disease Type 1 FLT201 - Gaucher Disease, Type 1 | Phase 1 | Completed | Spur Therapeutics | 10 | 2022-04-15 | 2026-06-02 |
| An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of Subjects With Batten Ceroid Lipofuscinosis, Neuronal 3 (CLN3) Disease Miglustat 100 milligrams (mg) Oral Capsule - Batten Disease | Phase 1 / Phase 2 | Completed | Beyond Batten Disease Foundation | 6 | 2022-03-10 | 2025-09-09 |
| Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe Disease PBKR03 - Leukodystrophy, Globoid Cell | Phase 1 / Phase 2 | Suspended | Gemma Biotherapeutics | 24 | 2022-02-24 | 2026-06-30 |
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Gaucher Disease (32) · Fabry Disease (23) · Lysosomal Storage Diseases (17) · Neuronal Ceroid-Lipofuscinoses (17) · Leukodystrophy, Metachromatic (11) · Niemann-Pick Disease, Type C (8) · Sandhoff Disease (6) · Tay-Sachs Disease (6) · Gangliosidoses, GM2 (5) · Genetic Diseases, Inborn (5) · Leukodystrophy, Globoid Cell (5) · Metabolism, Inborn Errors (5) · Niemann-Pick Diseases (5) · Sphingolipidoses (5) · Adrenoleukodystrophy (4)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (108) · Nutritional and Metabolic Diseases (108) · Lipid Metabolism, Inborn Errors (107) · Lipid Metabolism Disorders (105) · Metabolic Diseases (104) · Genetic Diseases, Inborn (103) · Metabolism, Inborn Errors (103) · Nervous System Diseases (103) · Brain Diseases (90) · Lysosomal Storage Diseases, Nervous System (89) · Brain Diseases, Metabolic (88) · Brain Diseases, Metabolic, Inborn (88) · Central Nervous System Diseases (88) · Sphingolipidoses (85)
eliglustat (6) · migalastat (5) · 2-Hydroxypropyl-beta-cyclodextrin (4) · larazotide acetate (4) · Methylprednisolone (3) · Sirolimus (3) · cerliponase alfa (3) · Acetylcysteine (2) · Enzyme Replacement Therapy (2) · Glucosylceramidase (2) · Prednisone (2) · Pyrimethamine (2) · Sebelipase alfa (2) · agalsidase alfa (2) · agalsidase beta (2)
Amicus Therapeutics (6) · Shire (6) · Genzyme, a Sanofi Company (5) · Protalix (4) · Sanofi (4) · Spur Therapeutics (4) · Cyclo Therapeutics, Inc. (3) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (3) · Prevail Therapeutics (3) · Weill Medical College of Cornell University (3) · 4D Molecular Therapeutics (2) · AVROBIO (2) · BioMarin Pharmaceutical (2) · Exsar Corporation (2) · Gemma Biotherapeutics (2)
United States (70) · United Kingdom (21) · Germany (13) · Canada (10) · Australia (9) · Brazil (8) · France (8) · Israel (6) · Italy (6) · Spain (6) · China (5) · Denmark (4) · Taiwan (3) · Argentina (2) · Austria (2)
Forbes D Porter, M.D. (3) · Joanne Kurtzberg, MD (3) · Alan H Cohen, MD (2) · Caroline Hastings, MD (2) · May Orfali, MD (2) · Aaron Tward, MD, PhD (1) · An N. Dang Do, MD, PhD (1) · Anupam Sehgal, MBBS (1) · Arian Pano, MD, MPH (1) · Benjamin Greenberg, MD (1)
PMID 37624739, PMID 29688815, PMID 33332761, PMID 33980287, PMID 36196048, PMID 36279795, PMID 37171121, PMID 38101904, PMID 38776275, PMID 11231629, PMID 11399225, PMID 15324318, PMID 18473686, PMID 19277123, PMID 19892975, PMID 19948615, PMID 20299511, PMID 21637107, PMID 23348766, PMID 23510062, PMID 23581634, PMID 23845948, PMID 23860343, PMID 23966770, PMID 25758611, PMID 26000324, PMID 27136905, PMID 27289174, PMID 27536442, PMID 28088251, PMID 28374935, PMID 28657663, PMID 30337566, PMID 30834538, PMID 31187454, PMID 32102934, PMID 32792226, PMID 33633114, PMID 33875845, PMID 34389161, PMID 34810067, PMID 35065785, PMID 35249485, PMID 35333110, PMID 36631545, PMID 37035740, PMID 37592153, PMID 37614821, PMID 37670901, PMID 37942487
2025 (1), K12CA120780 (1), U01HD090845 (1), U54NS065768 (1), UL1TR000457-06 (1)