324 completed. 324 Not applicable.
| Phase | Trials | Status |
|---|---|---|
| Not applicable | 324 | Completed 324 |
Follow Metabolism, Inborn Errors: new trials by email Literature Review of Metabolism, Inborn Errors trials Research Report on Metabolism, Inborn Errors
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 |
|---|---|---|---|---|---|---|
| EFICAC-TTR Trial: Exercise and Nutritional Supplementation in Transthyretin Cardiac Amyloidosis Home-Based Multicomponent Exercise Program, Microcrystalline Cellulose Control Supplementation, Creatine and HMB Supplementation - Transthyretin Cardiac Amyloidosis | Not applicable | Completed | Universidad de Burgos | 47 | 2026-02-03 | 2026-09-25 |
| Impact of B. Adolescentis iVS-1 Probiotic on Gastrointestinal Outcomes Probiotic, Placebo - Digestive Disease, Gut Health | Not applicable | Completed | Synbiotic Health | 92 | 2026-01-20 | 2026-10-19 |
| Radicle GI Health RDH™: A Study Assessing the Impact of Health and Wellness Products on Gastrointestinal (GI) Health and Related Health Outcomes GI Health Active Product 1, GI Health Product Placebo Control - Gas and Bloating, Diarrhea | Not applicable | Completed | Radicle Science | 500 | 2025-12-16 | 2026-05-28 |
| A Study to Evaluate the Effects of a Butyrate-Polyphenol Formulation on Gut Health and Associated Symptoms Butyrate + Polyphenol Formulation, Placebo - Gut Health | Not applicable | Completed | Supplement Formulators, Inc. | 124 | 2025-10-16 | 2026-08-26 |
| Effect of Different Meal Types Given Before Exercise on Plasma Amino Acid Levels and Metabolic Control Parameters in Classical Phenylketonuria Patients Undergoing Aerobic and Resistance Exercises Aerobic Exercise, Resistance Exercise, Water - Phenylketonuria | Not applicable | Completed | Hacettepe University | 10 | 2025-10-01 | 2026-09-16 |
| PKU EASY Microtabs Plus - Acceptability and Tolerance PKU EASY Microtabs Plus - Phenylketonuria (PKU) | Not applicable | Completed | Galen Limited | 10 | 2025-03-15 | 2025-11-26 |
| UR+AIMS Gout Wearable Skin Uric Acid Monitor Study Uric Acid and Metabolite Monitor System (UR+AIMS) skin patch - Gout | Not applicable | Completed | University of California, Los Angeles | 6 | 2025-03-10 | 2025-11-24 |
| High-Dose Ambroxol in Pediatric Type III Gaucher Disease (GD3) Ambroxol - Gaucher Disease, Type 3 | Not applicable | Completed | Agyany Pharma LTD | 12 | 2025-03-01 | 2026-08-24 |
| Milk Beta Caseins on Digestive Discomfort, Gastric Emptying A1/A2 milk with oral naloxone, A1PF milk with oral naloxone - Lactose Intolerants, Lactose Malabsorption | Not applicable | Completed | Purdue University | 10 | 2025-01-30 | 2026-10-20 |
| Visual (Path)Ways in Multiple Sclerosis - Part II Measurement of retinal vascular density and visual cognition - Multiple Sclerosis, Optic Neuritis, Demyelinating Disease | Not applicable | Completed | University Hospital, Lille | 64 | 2025-01-24 | 2026-05-15 |
| Continuous Glucose Monitoring Alerts, Accuracy, and Patient Outcomes in Adults With Inherited Metabolic Disorders Continuous glucose monitor with threshold and predictive alerts (Dexcom G7), Continuous glucose monitor with threshold-only alerts (Dexcom ONE+) - Glycogen Storage Disease, Congenital Hyperinsulinism (CHI) | Not applicable | Completed | University Hospital Padova | 11 | 2025-01-08 | 2026-05-29 |
| Trial of Rice-Based F-75 in Severe Malnutrition & Persistent Diarrhea in Children (SAM-RICE Study) WHO-recommended standard therapeutic milk used for stabilization phase in SAM), HO-recommended Rice based therapeutic milk used for stabilization phase in SAM) - Severe Acute Malnutrition, Persistent Diarrhea | Not applicable | Completed | Children's Hospital and Institute of Child Health, Multan | 320 | 2024-12-31 | 2025-08-24 |
| MSOT for Assessment of Intestinal Transit Time in Lactose Intolerance Patients gastrointestinal transit time measurement using MSOT and ICG contrast agent - Lactose Intolerance | Not applicable | Completed | University of Erlangen-Nürnberg Medical School | 10 | 2024-11-11 | 2025-05-11 |
| Effect of Abdominal Massage on Gastrointestinal Symptoms After Head and Neck Cancer Surgery Abdominal Massage - Abdominal Massage, Enteral Nutrition | Not applicable | Completed | Maltepe University | 70 | 2024-10-30 | 2026-10-26 |
| Topical Ganciclovir Versus Systemic Aciclovir in the Treatment of Herpetic Keratitis Acyclovir 400 MG, Ganciclovir (GCV) - Herpetic Keratitis | Not applicable | Completed | Al-Azhar University | 20 | 2024-10-01 | 2025-01-03 |
| Effect of a Probiotic Formulation in Adults With Lactose Intolerance and Methanogenic Intestinal Overgrowth Probiotic formulation, Maltodextrin (Placebo) - Lactose Intolerance | Not applicable | Completed | Universidad de Concepcion | 22 | 2024-08-12 | 2026-10-07 |
| The Exploratory Handheld Optical Coherence Tomography Study in Wilson's Disease Diagnostic procedures - Wilson Disease | Not applicable | Completed | Royal Surrey County Hospital NHS Foundation Trust | 10 | 2024-08-02 | 2025-07-01 |
| Lactobreath: A Study to Diagnose Lactose Intolerance Using Breath Markers Lactose solution/Glucose solution - Lactose Intolerance | Not applicable | Completed | ETH Zurich | 112 | 2024-06-24 | 2026-09-15 |
| CO2 Supplement for Treatment of Acute Mountain Sickness Breathing with CO2 during daytime and night at high altitude, Sleep and doing exercise test under inhalation of air - Acute Mountain Sickness, High Altitude | Not applicable | Completed | State Key Laboratory of Respiratory Disease | 46 | 2024-06-07 | 2024-12-18 |
| Cardiac Amyloidosis Discovery Trial Cardiac amyloidosis deep learning model - Cardiac Amyloidosis | Not applicable | Completed | Pierre Elias | 50 | 2024-05-28 | 2025-12-04 |
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Phenylketonurias (33) · Gout (25) · Lactose Intolerance (24) · Multiple Acyl Coenzyme A Dehydrogenase Deficiency (23) · Hyperlipoproteinemia Type II (22) · HIV Infections (18) · Acquired Immunodeficiency Syndrome (17) · Mucopolysaccharidosis IV (16) · Cytomegalovirus Infections (15) · Cytomegalovirus Retinitis (13) · Fabry Disease (13) · Glycogen Storage Disease Type II (12) · Retinitis (12) · Amyloid Neuropathies, Familial (10)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (324) · Nutritional and Metabolic Diseases (324) · Metabolic Diseases (320) · Genetic Diseases, Inborn (319) · Nervous System Diseases (130) · Brain Diseases (112) · Central Nervous System Diseases (112) · Brain Diseases, Metabolic (108) · Brain Diseases, Metabolic, Inborn (108) · Amino Acid Metabolism, Inborn Errors (90) · Carbohydrate Metabolism, Inborn Errors (81) · Lipid Metabolism Disorders (69) · Lipid Metabolism, Inborn Errors (68) · Pathological Conditions, Signs and Symptoms (66)
Ganciclovir (21) · Blood Specimen Collection (9) · Exercise (7) · Cysteamine (4) · Folic Acid (4) · Probiotics (4) · Standard of Care (4) · Epidemiologic Studies (3) · Genetic Testing (3) · High-Energy Shock Waves (3) · Resistance Training (3) · Vitamin B 12 (3) · Zidovudine (3) · Butyrates (2) · Caseins (2)
Vitaflo International, Ltd (13) · Hoffmann-La Roche (11) · Assistance Publique - Hôpitaux de Paris (9) · Baylor College of Medicine (5) · Lille Catholic University (5) · National Center for Research Resources (NCRR) (5) · Centre Hospitalier Universitaire de Nice (4) · Copenhagen University Hospital, Rigshospitalet (4) · Duke University (4) · Hospices Civils de Lyon (4) · PATH (4) · University Hospital, Bordeaux (4) · FDA Office of Orphan Products Development (3) · Laval University (3) · Nantes University Hospital (3)
United States (107) · France (45) · United Kingdom (34) · Canada (17) · Germany (15) · Denmark (13) · Italy (11) · Switzerland (9) · Turkey (Türkiye) (9) · Brazil (8) · Netherlands (8) · Spain (8) · Egypt (4) · Belgium (3) · China (3)
Anita MacDonald (6) · Dennis Savaiano, PhD (2) · Ni-Chung Lee, M.D., Ph.D (2) · Rajavel Elango, PhD (2) · Sandesh CS Nagamani, MD, FACMG (2) · William T Cade, PT, PhD (2) · Yuanming Luo, PhD (2) · Adam Wax, Ph.D. (1) · Adrian Regensburger, PD Dr. med. Dr. rer. biol. hum (1) · Adriana B Carvalho, PhD (1) · Agnes Chen, MD (1) · Alexandre B Cavalcanti, PhD (1) · Aline Marcadenti, PhD (1) · Amalia A Tsiami, PhD (1)
PMID 38485432, PMID 40670924, PMID 11820919, PMID 12485966, PMID 16049943, PMID 162198, PMID 16818923, PMID 17316019, PMID 17384344, PMID 17915573, PMID 19220406, PMID 19833552, PMID 21183586, PMID 21590737, PMID 21963264, PMID 22000478, PMID 22331114, PMID 22543891, PMID 22911730, PMID 23199589, PMID 23430559, PMID 23665863, PMID 24279738, PMID 24359113, PMID 24388732, PMID 24615309, PMID 24899155, PMID 24924293, PMID 25042846, PMID 25101284, PMID 25391139, PMID 25456098, PMID 25527677, PMID 25559009, PMID 25605552, PMID 25803389, PMID 26252852, PMID 26270952, PMID 26352873, PMID 26499033, PMID 26717401, PMID 26779697, PMID 26780631, PMID 26781431, PMID 27014575, PMID 27295193, PMID 27413125, PMID 27458176, PMID 27548405, PMID 27561270
2016 (2), 2018 (1), P30AG064199 (1), P50AR060772 (1), R01CA204585 (1), R01DK088988 (1), R21AR061818 (1), R21AR069880 (1), R21DK132666 (1), R56HL118343 (1), U01HG006487 (1), U19HD077632 (1), U54NS065768 (1), U54RR019484 (1)