3 completed, 2 terminated, 1 active not recruiting, 1 not yet recruiting. 4 Phase 2, 1 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 4 | Active not recruiting 1, Completed 1, Not yet recruiting 1, Terminated 1 |
| Phase 1 | 1 | Not yet recruiting 1 |
Follow Aspartylglucosaminuria: new trials by email Literature Review of Aspartylglucosaminuria trials Research Report on Aspartylglucosaminuria
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 |
|---|---|---|---|---|---|---|
| Safety and Efficacy of scAAV9/AGA Gene Therapy in Participants With Aspartylglucosaminuria (AGU) scAAV9/AGA - Aspartylglucosaminuria, Aspartylglucosamidase (AGA) Deficiency | Phase 1 / Phase 2 | Not yet recruiting | Rare Trait Hope | 9 | 2026-05-01 | 2026-04-15 |
| Baby Detect : Genomic Newborn Screening Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1 | Completed | Centre Hospitalier Universitaire de Liege | 6824 | 2022-09-01 | 2025-08-12 | |
| A Natural History Study of Aspartylglucosaminuria Aspartylglucosaminuria, Aspartylglucosamidase (AGA) Deficiency | Terminated | Neurogene Inc. | 8 | 2019-04-18 | 2022-04-12 | |
| MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen - Mucopolysaccharidosis Disorders, Hurler Syndrome | Phase 2 | Active not recruiting | Masonic Cancer Center, University of Minnesota | 149 | 2014-07-10 | 2026-01-07 |
| Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders Campath-1H, Cyclophosphamide, Busulfan - Mucopolysaccharidosis, Hurler Syndrome | Phase 2 | Completed | Masonic Cancer Center, University of Minnesota | 46 | 2009-12-01 | 2018-02-05 |
| Longitudinal Studies of the Glycoproteinoses Non-interventional. - Aspartylglucosaminuria, Fucosidosis | Completed | Greenwood Genetic Center | 100 | 2009-08-01 | 2023-09-13 | |
| Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism Stem Cell Transplantation, Cyclophosphamide, Campath-1H - Hurler's Syndrome, Maroteaux-Lamy Syndrome | Phase 2 | Terminated | Masonic Cancer Center, University of Minnesota | 18 | 2008-03-01 | 2017-12-28 |
Fucosidosis (4) · Leukodystrophy, Metachromatic (4) · Mucopolysaccharidosis I (4) · Mucopolysaccharidosis VI (4) · Mucopolysaccharidosis VII (4) · alpha-Mannosidosis (4) · Leukodystrophy, Globoid Cell (3) · Mucopolysaccharidosis II (3) · Peroxisomal Disorders (3) · Sphingolipidoses (3) · Adrenoleukodystrophy (2) · Aspartylglucosamidase (AGA) deficiency (2) · Lysosomal Storage Diseases (2) · Wolman Disease (2)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (7) · Genetic Diseases, Inborn (7) · Nutritional and Metabolic Diseases (7) · Metabolic Diseases (6) · Metabolism, Inborn Errors (6) · Brain Diseases (5) · Brain Diseases, Metabolic (5) · Brain Diseases, Metabolic, Inborn (5) · Carbohydrate Metabolism, Inborn Errors (5) · Central Nervous System Diseases (5) · Lysosomal Storage Diseases (5) · Lysosomal Storage Diseases, Nervous System (5) · Nervous System Diseases (5) · Connective Tissue Diseases (4) · Demyelinating Diseases (4)
Stem Cell Transplantation (3) · Alemtuzumab (2) · Busulfan (2) · Cyclophosphamide (2) · Bone Marrow Transplantation (1) · Cyclosporine (1) · Mycophenolic Acid (1) · Regimen B (1)
Masonic Cancer Center, University of Minnesota (3) · Centre Hospitalier Universitaire de Liege (1) · Greenwood Genetic Center (1) · Neurogene Inc. (1) · Rare Trait Hope (1)
United States (5) · Belgium (1)
Paul Orchard, MD (2) · Elise Beausoleil (1) · Laurent Servais (1) · Paul Orchard, M.D. (1) · Sara Cathey, MD (1)