Clinical trials: Syndrome, Metabolic
Primary Mitochondrial Disease
OMT-28 in Patients With Primary Mitochondrial Disease (PMD) (PMD-OPTION)
The goal of this clinical trial is to learn about the treatment effects of the investigational new drug OMT-28 in patients with Primary Mitochondrial Disease.
- Trial name
- PMD-OPTION (NCT05972954)
- Testing
- OMT-28
- Where
- 9 sites in Germany, Italy and 1 more
- Volunteers
- 28 people
- Sponsor
- Omeicos Therapeutics GmbH
Hurler Syndrome (MPS I), Hurler-Scheie Syndrome
Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
The goal of this research study is to establish chimerism and avoid graft-versus-host-disease (GVHD) in patients with inherited metabolic disorders.
- Conditions
- Hurler Syndrome (MPS I), Hurler-Scheie Syndrome, Hunter Syndrome (MPS II), Sanfilippo Syndrome (MPS III)
- Trial name
- NCT01372228
- Testing
- hematopoietic stem cell infusion
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Talaris Therapeutics Inc.
Metabolic Syndrome, Type 2 Diabetes
LUPS-Lipids and Glucose Under Prospective Surveillance
Prospective, non-randomized cohort study to analyse genetic variations, dietary patterns, perceived stress and anxiety and early changes in lipid metabolism leading to the metabolic syndrome and type 2 diabetes
- Trial name
- LUPS (NCT01313156)
- Where
- 2 sites in Germany
- Volunteers
- 1,962 people, healthy volunteers welcome
- Sponsor
- Asklepios proresearch
Obesity Hypoventilation Syndrome, Obstructive Sleep Apnea
Simplus and Eson Non-Invasive Ventilation (NIV) Evaluation - Germany
This investigation is designed to evaluate the performance, comfort and ease of use of the Simplus and Eson masks amongst NIV patients who are currently on Bi-level therapy
- Conditions
- Obesity Hypoventilation Syndrome, Obstructive Sleep Apnea, Chronic Obstructive Pulmonary Disease, Neuromuscular Disease
- Trial name
- NCT02726048
- Testing
- Simplus/Eson
- Where
- Germany
- Volunteers
- 4 people
- Sponsor
- Fisher and Paykel Healthcare
Metabolic Syndrome
VIsceral Fat Reduction Assessed by CT-scan On RImonabAnt
Primary objective: To assess the effect of rimonabant on visceral fat area over a period of 12 months when prescribed with a mild hypocaloric diet in abdominally obese patients with metabolic syndrome Secondary objectives: To…
- Trial name
- VICTORIA (NCT00299325)
- Testing
- Rimonabant, Mild hypocaloric diet
- Where
- 9 sites in United States, Canada and 7 more
- Volunteers
- 254 people
- Sponsor
- Sanofi
Diabetes Mellitus
Efficacy of Alogliptin and Pioglitazone in Subjects With Type 2 Diabetes Mellitus
The purpose of the study is to compare the effect of adding alogliptin, once daily (QD), to the ongoing treatment regimen of pioglitazone HCl and metformin in patients with inadequate glycemic control.
- Trial name
- NCT00432276
- Testing
- Alogliptin, Pioglitazone, Metformin
- Where
- 86 sites in United States
- Volunteers
- 803 people
- Sponsor
- Takeda
Werner Syndrome
Phase 2, Open-Label Study to Evaluate the Safety and Tolerability of Progerinin in Werner Syndrome
This is a Phase 2, open-label study to A Phase 2, Open-Label Study to Evaluate the Safety and Tolerability of Progerinin for the Treatment of Bone Mineral Density (BMD) Loss in Subjects with Typical Werner Syndrome.
- Trial name
- NCT05847179
- Testing
- Progerinin
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- PRG Science & Technology Co., Ltd.
Celiac Disease
A Study to Evaluate the Efficacy and Safety of AMG 714 in Adult Patients With Celiac Disease
This study is designed to evaluate the efficacy and safety of AMG 714 for the attenuation of the effects of gluten exposure in adult patients with celiac disease during a gluten challenge.
- Trial name
- NCT02637141
- Testing
- AMG 714, Gluten Challenge
- Where
- 3 sites in Finland
- Volunteers
- 64 people
- Sponsor
- Amgen
Mucopolysaccharidosis Type IIIB
A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)
This is a natural history study for children up to 18 years of age who have been diagnosed with Mucopolysaccharidosis Type IIIB (MPS IIIB, also known as Sanfilippo Syndrome Type B).
- Trial name
- NCT03227042
- Where
- 9 sites in United States, Argentina and 7 more
- Volunteers
- 44 people
- Sponsor
- Allievex Corporation
Hyponatremias, Water Intoxication
"SALT Trial" Study of Ascending Levels of Tolvaptan in Hyponatremia
This study's purpose is to determine whether tolvaptan can safely and effectively return the body's balance of sodium and water toward normal, and to characterize and quantify the potential clinical benefits of this treatment.
- Conditions
- Hyponatremias, Water Intoxication, Inappropriate ADH Syndrome, Water-Electrolyte Imbalances
- Trial name
- NCT00072683
- Testing
- tolvaptan
- Where
- 23 sites in United States
- Volunteers
- 205 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Type 2 Diabetes, Metabolic Syndrome
Study to Evaluate the Safety and Tolerability of PBI-4050 in Type 2 Diabetes Patients With Metabolic Syndrome
This is a Phase 2, open-label, single-arm study of the safety and tolerability of PBI-4050 800 mg daily oral administration in type 2 diabetes patients with metabolic syndrome.
- Trial name
- NCT02562573
- Testing
- PBI4050
- Where
- Canada
- Volunteers
- 24 people
- Sponsor
- Liminal BioSciences Ltd.
Nutritional Support
Interpretation and Optimization of Nutrition in the Intensive Care Units
Despite recommendations, inadequate nutritional intake in intensive care unit (ICU) patients remains frequent and can lead to complications such as infections, increased length of stay, prolonged weaning from ventilation…
- Trial name
- IONIC (NCT06342895)
- Testing
- Nutritional support prescription and delivery monitoring feedback, Usual care
- Where
- France
- Volunteers
- 144 people
- Sponsor
- Dim3
Congenital Adrenal Hyperplasia
Nevanimibe HCl for the Treatment of Classic CAH
This is a multicenter, intra-subject dose-titration open-label study of nevanimibe hydrochloride (HCl) for the treatment of classic congenital adrenal hyperplasia (CAH).
- Trial name
- NCT03669549
- Testing
- Nevanimibe hydrochloride
- Where
- 11 sites in Brazil, Czechia and 3 more
- Volunteers
- 15 people
- Sponsor
- Millendo Therapeutics US, Inc.
Ketosis, Epilepsy
Market Research - Acceptability Study for New MCT Fat Products
This is a market research, observational study to evaluate the tolerability and acceptability of MCT supplements for young children, young people and adults with intractable epilepsy, GLUT-1 or PDHD from 3 years to adulthood.
- Conditions
- Ketosis, Epilepsy, Fatty Acid Oxidation Disorder, Malabsorption
- Trial name
- NCT04309214
- Testing
- MCT fats
- Where
- United Kingdom
- Volunteers
- 12 people
- Sponsor
- Dr. Schär AG / SPA
Mitochondrial Diseases, Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation
The KHENEREXT Study
This is an open-label, multi-centre study in subjects with a genetically confirmed mitochondrial deoxyribonucleic acid (DNA) transfer ribonucleic acid (tRNA)Leu(UUR) m.3243A>G mutation who completed study KH176-202.
- Conditions
- Mitochondrial Diseases, Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation, Maternally Inherited Diabetes and Deafness (MIDD), Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke Like Episodes (MELAS)
- Trial name
- NCT04604548
- Testing
- Oral administration of 100 mg KH176 twice daily
- Where
- 4 sites in Denmark, Germany and 2 more
- Volunteers
- 11 people
- Sponsor
- Khondrion BV
Celiac Disease
A Trial to Assess the Efficacy and Safety of TEV-53408 in Adults With Celiac Disease
The primary efficacy objective of the trial is to assess the ability of TEV-53408 to attenuate gluten-induced enteropathy in adults with celiac disease.
- Trial name
- NCT06807463
- Testing
- TEV-53408
- Where
- 21 sites in United States, Australia and 1 more
- Volunteers
- 50 people
- Sponsor
- Teva Branded Pharmaceutical Products R&D LLC
Mucopolysaccharidosis II
To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Patients
The purpose of this study is to evaluate the safety and efficacy of GC1111 (recombinant human iduronate-w-sulfatase) in Hunter Syndrome (Mucopolysaccharidosis II) patients
- Trial name
- NCT01301898
- Testing
- GC1111_0.5mg/kg, GC1111_1.0mg/kg, Elaprase_0.5mg/kg
- Where
- South Korea
- Volunteers
- 31 people
- Sponsor
- Green Cross Corporation
Primary Immunodeficiencies, Inborn Errors of Metabolism
Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant Diseases
The aim of the trial is to describe the safety and efficacy of intravenous (i.v.) Treosulfan compared to the conventional (myeloablative) dose of i.v.
- Conditions
- Primary Immunodeficiencies, Inborn Errors of Metabolism, Haemoglobinopathies, Bone Marrow Failure Syndromes
- Trial name
- NCT02349906
- Testing
- Treosulfan, Busilvex
- Where
- 21 sites in Czechia, Germany and 2 more
- Volunteers
- 106 people
- Sponsor
- medac GmbH
Mitochondrial Diseases, Pearson Syndrome
A Study to Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-BM-BLD in Pediatric Patients With Pearson Syndrome
Mitochondrial diseases are a genetically heterogeneous group of disorders caused by mutations or deletions in mitochondrial DNA (mtDNA) displaying a wide range of severity and phenotypes.
- Trial name
- NCT03384420
- Testing
- CD34+ cells enriched with MNV-BLD
- Where
- Israel
- Volunteers
- 7 people
- Sponsor
- Minovia Therapeutics Ltd.
Low Bone Mass, Low Bone Mineral Density
Study to Compare the Efficacy and Safety of DenosumAb Versus Placebo in Males With Osteoporosis
The purpose of this study is to assess how effective and safe denosumab is in a population of males with low bone mass at risk of fracture.
- Conditions
- Low Bone Mass, Low Bone Mineral Density, Males With Osteoporosis, Osteopenia
- Trial name
- NCT00980174
- Testing
- 60 mg denosumab
- Volunteers
- 242 people
- Sponsor
- Amgen