16 unknown. 16 Phase 2, 3 Phase 1, 3 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 16 | Unknown 16 |
| Phase 1 | 3 | Unknown 3 |
| Phase 3 | 3 | Unknown 3 |
Follow Lysosomal Storage Diseases: new trials by email Literature Review of Lysosomal Storage Diseases trials Research Report on Lysosomal Storage 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 |
|---|---|---|---|---|---|---|
| An Open Label Dose Escalation Study to Assess the Safety, Tolerability, and Pharmacologic Properties of High Dose Ambroxol Hydrochloride in Adult (≥ 18 Years of Age) Subjects With MPS III Ambroxol Hydrochloride 30 mg tablet - 9 mg/kg/day, Ambroxol Hydrochloride 30 mg tablet - 18 mg/kg/day, Ambroxol Hydrochloride 30 mg tablet - 27 mg/kg/day - Sanfilippo Syndrome, MPS3 | Phase 2 / Phase 3 | Unknown | Ozlem Goker-Alpan | 10 | 2024-12-01 | 2025-08-20 |
| 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 |
| Nutritional Therapy in Late-onset Pompe Disease Multi-ingredient supplement (PDT-MIS), Placebo (PLA) - Pompe Disease, Muscle Loss | Phase 2 | Unknown | McMaster University | 28 | 2024-04-01 | 2023-11-14 |
| 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 |
| 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 |
| 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 |
| Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA) LYS-SAF302 - Mucopolysaccharidosis Type IIIA | Phase 2 / Phase 3 | Unknown | LYSOGENE | 20 | 2018-12-17 | 2021-08-31 |
| Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI Losartan, Placebo - Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI | Phase 2 | Unknown | Hospital de Clinicas de Porto Alegre | 10 | 2018-11-07 | 2022-12-19 |
| A Treatment Extension Study of Mucopolysaccharidosis Type IIIB AX 250 - Mucopolysaccharidosis Type IIIB, MPS III B | Phase 2 | Unknown | Allievex Corporation | 20 | 2018-02-19 | 2023-01-18 |
| Study of Idursulfase-beta (GC1111) in Hunter Syndrome idursulfase beta, idursulfase - Mucopolysaccharidosis II | Phase 2 | Unknown | Green Cross Corporation | 20 | 2016-12-01 | 2016-01-26 |
| Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy transduced CD34+ hematopoietic stem cell - Metachromatic Leukodystrophy, Adrenoleukodystrophy | Phase 1 / Phase 2 | Unknown | Shenzhen Second People's Hospital | 50 | 2015-01-01 | 2022-05-31 |
| Intra-monocyte Imiglucerase Kinetics in Gaucher Disease Imiglucérase (drug) pharmacokinetics - Gaucher Disease | Phase 2 | Unknown | University Hospital, Clermont-Ferrand | 60 | 2012-11-01 | 2013-09-27 |
| Dose Finding Study of Recombinant Human Alpha-mannosidase for the Treatment of Patients With Alpha-mannosidosis Lamazym - Alpha Mannosidosis | Phase 2 | Unknown | Zymenex A/S | 10 | 2011-01-01 | 2012-09-26 |
| Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis Conditioning regimen, Graft-versus-host disease (GVHD) prophylaxis, Stem Cell Transplantation - Mucopolysaccharidosis | Phase 2 / Phase 3 | Unknown | Tehran University of Medical Sciences | 10 | 2009-09-01 | 2012-06-04 |
| Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease glucocerebrosidase - Gaucher's Disease | Phase 2 | Unknown | National Center for Research Resources (NCRR) | 24 | 1999-11-01 | 2005-06-24 |
Gaucher Disease (4) · Mucopolysaccharidosis III (3) · Fabry Disease (2) · Mucopolysaccharidoses (2) · Adrenoleukodystrophy (1) · Glycogen Storage Disease Type II (1) · Hematologic Neoplasms (1) · Leukemia, Myeloid, Acute (1) · Leukodystrophy, Metachromatic (1) · Malnutrition (1) · Metabolism, Inborn Errors (1) · Mucopolysaccharidosis II (1) · Mucopolysaccharidosis IV (1) · Mucopolysaccharidosis VI (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (16) · Genetic Diseases, Inborn (16) · Metabolic Diseases (16) · Nutritional and Metabolic Diseases (16) · Metabolism, Inborn Errors (15) · Nervous System Diseases (10) · Brain Diseases (9) · Brain Diseases, Metabolic (9) · Brain Diseases, Metabolic, Inborn (9) · Central Nervous System Diseases (9) · Lysosomal Storage Diseases, Nervous System (9) · Carbohydrate Metabolism, Inborn Errors (8) · Lipid Metabolism Disorders (8) · Lipid Metabolism, Inborn Errors (8)
Ambroxol (1) · Antilymphocyte Serum (1) · Cyclophosphamide (1) · Glucosylceramidase (1) · Losartan (1) · Pharmaceutical Preparations (1) · Pharmacokinetics (1) · Stem Cell Transplantation (1) · Tablets (1) · Transplantation Conditioning (1) · dapagliflozin (1) · eliglustat (1) · idursulfase (1) · idursulfase beta, mouse (1)
AceLink Therapeutics, Inc. (1) · Albina Nowak, MD (1) · Allievex Corporation (1) · CANbridge (Suzhou) Bio-pharma Co., Ltd. (1) · Green Cross Corporation (1) · Hospital de Clinicas de Porto Alegre (1) · LYSOGENE (1) · McMaster University (1) · National Center for Research Resources (NCRR) (1) · Ozlem Goker-Alpan (1) · Peking Union Medical College Hospital (1) · Shenzhen Second People's Hospital (1) · Smart Immune SAS (1) · Tehran University of Medical Sciences (1) · University Hospital, Clermont-Ferrand (1)
China (4) · United States (4) · France (3) · Germany (2) · United Kingdom (2) · Brazil (1) · Colombia (1) · Denmark (1) · Iran (1) · Netherlands (1) · Spain (1) · Taiwan (1) · Turkey (Türkiye) (1)
Allan M. Lund, MD (1) · Bing Han (1) · Bing Han, MD (1) · GuangFu Chen, M.D.,Ph.D (1) · Guilherme Baldo, PhD (1) · Hua Jiang, M.D,Ph.D (1) · Jens Fogh (1) · Jiacai Zhuo (1) · John Barranger (1) · Mark Tarnopololsky, MD/PhD (1) · Nan Chen, Dr. (1) · Ozlem Goker-Alpan, MD (1)
PMID 11231629, PMID 11399225, PMID 19892975, PMID 23845948, PMID 23966770