5 completed, 2 unknown, 1 not yet recruiting, 1 recruiting. 3 Not applicable, 1 Phase 2, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Not applicable | 3 | Completed 1, Not yet recruiting 1, Unknown 1 |
| Phase 2 | 1 | Completed 1 |
| Phase 3 | 1 | Completed 1 |
Follow Maple Syrup Urine Disease: new trials by email Literature Review of Maple Syrup Urine Disease trials Research Report on Maple Syrup Urine Disease
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 |
|---|---|---|---|---|---|---|
| MyRareDiet A Novel Diet Tracking Tool MDR diet assessment, 24 hour recall - Urea Cycle Disorder, Propionic Aciduria | Not applicable | Not yet recruiting | Oregon Health and Science University | 60 | 2024-11-15 | 2024-10-30 |
| Liquid Valine and Isoleucine in Maple Syrup Urine Disease Valine oral solution (50mg/ml) and isoleucine oral solution (25mg/ml) - Maple Syrup Urine Disease | Not applicable | Unknown | Meta Healthcare Ltd | 5 | 2024-10-01 | 2024-09-03 |
| 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 |
| Neurocognitive Outcomes and Quality of Life in Adults With Maple Syrup Urine Disease (MSUD) Neurocognitive testing - Maple Syrup Urine Disease | Completed | Children's Hospital of Philadelphia | 44 | 2021-06-12 | 2024-06-04 | |
| 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 | |
| Patient and Observer Reported Outcome Measurements in Inborn Errors of Metabolism Survey - Inborn Errors of Metabolism, Urea Cycle Disorder | Completed | University Children's Hospital, Zurich | 69 | 2019-08-01 | 2020-11-04 | |
| Phenylbutyrate Therapy for Maple Syrup Urine Disease Phenylbutyrate, Placebo powder - Maple Syrup Urine Disease | Phase 2 / Phase 3 | Completed | Brendan Lee | 20 | 2013-02-01 | 2019-03-19 |
Glutaric Acidemia I (4) · Ornithine Carbamoyltransferase Deficiency Disease (4) · Argininosuccinic Aciduria (3) · Carbamoyl-Phosphate Synthase I Deficiency Disease (3) · Carnitine-Acylcarnitine Translocase Deficiency (3) · Citrullinemia (3) · Homocystinuria (3) · Hyperargininemia (3) · Medium chain acyl CoA dehydrogenase deficiency (3) · Methylmalonic acidemia (3) · Phenylketonurias (3) · Trifunctional Protein Deficiency With Myopathy And Neuropathy (3) · Tyrosinemias (3) · Urea Cycle Disorders, Inborn (3)
Brain Diseases (9) · Brain Diseases, Metabolic (9) · Brain Diseases, Metabolic, Inborn (9) · Central Nervous System Diseases (9) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (9) · Genetic Diseases, Inborn (9) · Metabolic Diseases (9) · Nervous System Diseases (9) · Nutritional and Metabolic Diseases (9) · Amino Acid Metabolism, Inborn Errors (8) · Metabolism, Inborn Errors (7) · Genetic Diseases, X-Linked (4) · Connective Tissue Diseases (3) · Hyperhomocysteinemia (3) · Mitochondrial Diseases (3)
Aging (1) · Isoleucine (1) · Phenylbutyrates (1) · Surveys and Questionnaires (1) · Valine (1)
Brendan Lee (1) · Centre Hospitalier Universitaire de Liege (1) · Children's Hospital of Philadelphia (1) · Children's National Research Institute (1) · Meta Healthcare Ltd (1) · Oregon Health and Science University (1) · University Children's Hospital, Zurich (1) · Vitaflo International, Ltd (1) · West Kazakhstan Medical University (1)
United States (3) · Belgium (1) · Kazakhstan (1) · Switzerland (1) · United Kingdom (1)
Anita MacDonald (2) · Brendan Lee, M.D., Ph.D. (1) · Dershung Yang, PhD (1) · Gulmira M. Zharmakhanova, MD, PhD (1) · Jessica Gold, MD, PhD (1) · Laurent Servais (1) · Ljubica Caldovic, PhD (1) · Lyazzat M. Syrlybayeva, MD (1) · Martina Huemer (1) · Nicholas Ah Mew, MD (1) · Victoria I. Kononets, MD, MS (1)
R21TR003166 (1), R44HD107766 (1)