10 withdrawn. 10 Phase 2, 4 Phase 1, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 10 | Withdrawn 10 |
| Phase 1 | 4 | Withdrawn 4 |
| Phase 3 | 1 | Withdrawn 1 |
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 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of Gaucher Disease Type 3 Gene therapy, Enzyme Replacement Agent - Gaucher Disease, Type 3 | Phase 2 / Phase 3 | Withdrawn | AVROBIO | 2023-10-01 | 2023-08-09 | |
| Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease Clenbuterol, Placebos - Pompe Disease (Late-onset) | Phase 2 | Withdrawn | Duke University | 2023-01-01 | 2022-08-17 | |
| Safety and Effect of Oral RVX000222 in Subjects With Fabry Disease RVX000222 - Fabry Disease | Phase 1 / Phase 2 | Withdrawn | Resverlogix Corp | 2022-11-22 | 2023-11-18 | |
| Safety and Efficacy of Encapsulated Allogeneic MPS-1 Therapy SIG-005 (hIDUA Producing Spheres) - MPS I, MPS1-S | Phase 1 / Phase 2 | Withdrawn | Sigilon Therapeutics, Inc. | 2021-11-15 | 2023-04-11 | |
| Safety and Tolerability Study of VTS-270 in Pediatric Participants With Niemann-Pick Type C (NPC) Disease VTS-270 - Niemann-Pick Disease, Type C | Phase 2 | Withdrawn | Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company | 2020-05-01 | 2020-07-22 | |
| Safety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD) Umbilical cord blood transplantation with HSC835 - Inherited Metabolic Disorders IMD | Phase 1 / Phase 2 | Withdrawn | Novartis Pharmaceuticals | 2017-10-10 | 2018-11-28 | |
| Therapeutic Effect of Recombinant Human Growth Hormone (rhGH) on the Myopathy of Cystinosis rh Growth Hormone - Cystinosis, Myopathy | Phase 1 / Phase 2 | Withdrawn | National Human Genome Research Institute (NHGRI) | 2014-03-26 | 2017-10-20 | |
| High Protein and Exercise Therapy Plus Nocturnal Enteral Feeding in Juvenile-onset Pompe Disease High Protein and Exercise Therapy along-with Nocturnal Enteral Feeding - Glycogen Storage Disease Type II | Phase 2 | Withdrawn | Columbia University | 2012-10-01 | 2015-04-13 | |
| Safety and Efficacy of Gabapentin for Neuropathic Pain in Fabry Disease Gabapentin, placebo - Fabry Disease, Neuropathic Pain | Phase 2 | Withdrawn | University of Minnesota | 2012-04-01 | 2019-05-29 | |
| Providing Access to Cord Blood Units for Transplants Access to unlicensed cord blood units - Chronic Myelogous Leukemia/Other Leukemia, Acute Leukemias | Phase 2 | Withdrawn | National Cancer Institute (NCI) | 2011-10-27 | 2018-12-21 |
Fabry Disease (2) · Glycogen Storage Disease Type II (2) · Mucopolysaccharidosis I (2) · Cardiovascular Diseases (1) · Cystinosis (1) · Gaucher Disease (1) · Hematologic Neoplasms (1) · Kidney Diseases (1) · Leukemia (1) · Leukemia, Myelogenous, Chronic, BCR-ABL Positive (1) · Leukodystrophy, Metachromatic (1) · Lymphoma, Non-Hodgkin (1) · Multiple Myeloma (1) · Muscular Diseases (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (10) · Genetic Diseases, Inborn (10) · Metabolic Diseases (10) · Metabolism, Inborn Errors (10) · Nutritional and Metabolic Diseases (10) · Nervous System Diseases (8) · Brain Diseases (7) · Brain Diseases, Metabolic (7) · Brain Diseases, Metabolic, Inborn (7) · Central Nervous System Diseases (7) · Lysosomal Storage Diseases, Nervous System (7) · Lipid Metabolism Disorders (5) · Lipid Metabolism, Inborn Errors (5) · Lipidoses (5)
Clenbuterol (1) · Gabapentin (1) · Genetic Therapy (1) · Velaglucerase alfa, human (1) · apabetalone (1) · imiglucerase (1) · taliglucerase alfa (1)
AVROBIO (1) · Columbia University (1) · Duke University (1) · National Cancer Institute (NCI) (1) · National Human Genome Research Institute (NHGRI) (1) · Novartis Pharmaceuticals (1) · Resverlogix Corp (1) · Sigilon Therapeutics, Inc. (1) · University of Minnesota (1) · Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company (1)
United States (3) · Brazil (1) · Canada (1) · United Kingdom (1)
Alfred E Slonim, MD (1) · Dwight Koeberl, MD, PhD (1) · Galina V Nesterova, M.D. (1) · Jeanine R. Jarnes, PharmD (1) · Michael West, MD (1) · Milena Veselinovic, MD, PhD (1) · Ronald E Gress, M.D. (1)