2 completed, 2 recruiting, 1 active not recruiting, 1 unknown. 1 Not applicable, 1 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Not applicable | 1 | Completed 1 |
| Phase 1 | 1 | Recruiting 1 |
Follow Glutaric Acidemia I: new trials by email Literature Review of Glutaric Acidemia I trials Research Report on Glutaric Acidemia I
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 |
|---|---|---|---|---|---|---|
| A Study to Evaluate the Tolerability, Safety and Efficacy of VGM-R02b VGM-R02b - Glutaric Acidemia Type I, Glutaric Aciduria Type I | Phase 1 | Recruiting | Shanghai Vitalgen BioPharma Co., Ltd. | 12 | 2024-04-29 | 2026-05-13 |
| Selective Screening of Children for Hereditary Metabolic Diseases by Tandem Mass Spectrometry in Kazakhstan Obtaining Dry Blood Spots From Healthy Newborns (Aged 1-7 Days), Obtaining Dry Blood Spots From Healthy Children Aged 8 Days - 7 Years, Obtaining Dry Blood Spots From Healthy Children Aged 8-18 Years - Propionic/Methylmalonic Acidemias, Maple Syrup Urine Disease | Unknown | West Kazakhstan Medical University | 2250 | 2022-10-03 | 2023-06-18 | |
| 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 | |
| Evaluation of the Express Plus Range PKU express plus - Phenylketonurias, PKU | Not applicable | Completed | Vitaflo International, Ltd | 28 | 2021-06-14 | 2026-05-18 |
| Systemic Biomarkers of Brain Injury From Hyperammonemia Urea Cycle Disorder, Organic Acidemia | Recruiting | Children's National Research Institute | 24 | 2020-07-09 | 2024-02-07 | |
| Early Check: Expanded Screening in Newborns Confirmatory Testing - Spinal Muscular Atrophy, Fragile X Syndrome | Active not recruiting | RTI International | 30000 | 2018-10-15 | 2026-07-01 |
Argininosuccinic Aciduria (4) · Carbamoyl-Phosphate Synthase I Deficiency Disease (4) · Carnitine-Acylcarnitine Translocase Deficiency (4) · Citrullinemia (4) · Homocystinuria (4) · Hyperargininemia (4) · Maple Syrup Urine Disease (4) · Medium chain acyl CoA dehydrogenase deficiency (4) · Ornithine Carbamoyltransferase Deficiency Disease (4) · Trifunctional Protein Deficiency With Myopathy And Neuropathy (4) · Tyrosinemias (4) · 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency (3) · Acidemia, isovaleric (3) · Beta ketothiolase deficiency (3)
Amino Acid Metabolism, Inborn Errors (5) · Brain Diseases (5) · Brain Diseases, Metabolic (5) · Brain Diseases, Metabolic, Inborn (5) · Central Nervous System Diseases (5) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (5) · Genetic Diseases, Inborn (5) · Metabolic Diseases (5) · Nervous System Diseases (5) · Nutritional and Metabolic Diseases (5) · Connective Tissue Diseases (4) · Genetic Diseases, X-Linked (4) · Hyperhomocysteinemia (4) · Metabolism, Inborn Errors (4) · Mitochondrial Diseases (4)
Aging (1)
Centre Hospitalier Universitaire de Liege (1) · Children's National Research Institute (1) · RTI International (1) · Shanghai Vitalgen BioPharma Co., Ltd. (1) · Vitaflo International, Ltd (1) · West Kazakhstan Medical University (1)
United States (2) · Belgium (1) · China (1) · Kazakhstan (1) · United Kingdom (1)
Anita MacDonald (1) · Curt Scharfe, MD, PhD (1) · Guanping Dong (1) · Gulmira M. Zharmakhanova, MD, PhD (1) · Laurent Servais (1) · Ljubica Caldovic, PhD (1) · Lyazzat M. Syrlybayeva, MD (1) · Nicholas Ah Mew, MD (1) · Qiang Shu (1) · Rulai Yang (1) · Victoria I. Kononets, MD, MS (1)
PMID 31315600, PMID 35142618, PMID 38283151
R21TR003166 (1)