Clinical trials: Syndrome, Metabolic
Celiac Disease
AMG 714 Expanded Access Program
Expanded access requests for AMG 714 may be considered for adult patients with biopsy proven Refractory Celiac Disease Type II who have failed all available treatment options and do not have EATL.
- Trial name
- NCT03439475
- Testing
- AMG 714
- Sponsor
- Amgen
Mucopolysaccharidosis Type II, Metabolic Diseases
A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II
This is phase 2/3 study to evaluate the safety, pharmacokinetics, pharmacodynamics, and efficacy of the investigational product GNR-055 in MPS II (Hunter syndrome) patients of different age groups.
- Trial name
- NCT05208281
- Testing
- GNR-055 1.0-2.0-3.0 mg/kg, GNR-055 2.0 mg/kg, GNR-055 3.0 mg/kg
- Where
- 5 sites in Russia
- Volunteers
- 32 people
- Sponsor
- AO GENERIUM
Cardiac Glycogenosis, Cardiomyopathy
ATR 1072 in Participants With PRKAG2 Syndrome
ATR 1072 is an investigational medicine being studied in adults with PRKAG2 Syndrome. This study will evaluate the safety of ATR 1072 and how the body processes the drug.
- Conditions
- Cardiac Glycogenosis, Cardiomyopathy, Hypertrophic, Wolff-Parkinson-White Syndrome
- Trial name
- NCT07738107
- Testing
- ATR 1072
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- Atrium Therapeutics
Healthy Participants
Study to Investigate the Mass Balance, Metabolism, and Excretion of [14C]PF-07321332 in Healthy Male Participants.
This study is to characterize mass balance and further the understanding of human pharmacokinetics, metabolism, and excretion of radiolabeled [14C]PF-07321332 in healthy male participants following oral administration with…
- Trial name
- NCT05178654
- Testing
- [14C]PF-07321332
- Volunteers
- healthy volunteers welcome
- Sponsor
- Pfizer
Diabetes Mellitus, Type II
GOLO Open-Label Pilot 1
The objective of this open-label pilot study is to evaluate the effect of the commercially available GOLO for Life (GFL) weight management program on body weight in a type 2 diabetic patient population after approximately 3…
- Trial name
- NCT03478202
- Testing
- GOLO RELEASE
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- Golo
Thymidine Kinase 2 Deficiency
An Open-Label Study of Continuation Treatment With Combination Pyrimidine Nucleosides in Patients With TK2 Deficiency
This is a Phase 2 prospective open-label treatment study of the safety and efficacy of doxecitine and doxribtimine in study participants with thymidine kinase 2 (TK2) deficiency who participated in the retrospective study…
- Trial name
- Continuation (NCT03845712)
- Testing
- doxecitine and doxribtimine
- Where
- 8 sites in United States, Israel and 1 more
- Volunteers
- 47 people
- Sponsor
- UCB BIOSCIENCES, Inc.
Subjects With Hyperlipidemia, Dyslipidemia and HIV Infection
Safety, Tolerability and Efficacy on Low Density Lipoprotein Cholesterol (LDL-C) of Evolocumab in Participants With Human Immunodeficiency Virus (HIV) and Hyperlipidemia/Mixed Dyslipidemia
The study is divided into 2 parts. The first part of the study will be double-blinded and will last for 24 weeks. During this time, participants will be randomized in a ratio of 2:1 to receive either evolocumab once monthly (QM)…
- Trial name
- NCT02833844
- Testing
- Evolocumab
- Where
- 78 sites in United States, Australia and 13 more
- Volunteers
- 467 people
- Sponsor
- Amgen
Infant, Small for Gestational Age
SURF, Neurodevelopment, Growth Study in SGA Infants
The purpose of this study is to determine whether term SGA infants who are fed a nutrient-enriched pre-term formula will have a higher cognitive score, a more appropriate length-for-age Z-score (leading to a lower prevalence of…
- Trial name
- NCT04360967
- Testing
- Standard Formula, Nutrient-enriched Formula
- Volunteers
- healthy volunteers welcome
- Sponsor
- Société des Produits Nestlé (SPN)
Metabolic Syndrome
Regional Observation of the Metabolic Syndrome
The aim of the study, is to assess the prevalence of metabolic syndrome in Italy on a regional basis, defined according to National Cholesterol Education Program/Adult Treatment Panel III (NCEP/ATPIII) guidelines criteria.
- Trial name
- ORSA (NCT00874952)
- Where
- Italy
- Volunteers
- 1,600 people
- Sponsor
- AstraZeneca
Mucopolysaccharidosis VII, MPS VII
Mucopolysaccharidosis VII Disease Monitoring Program
The objectives of this study are to characterize MPS VII disease presentation and progression and assess long-term effectiveness and safety, including hypersensitivity reactions and immunogenicity of vestronidase alfa.
- Conditions
- Mucopolysaccharidosis VII, MPS VII, MPS 7, Sly Syndrome
- Trial name
- NCT03604835
- Testing
- No Intervention
- Where
- 14 sites in United States, Argentina and 6 more
- Volunteers
- 50 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Congenital Adrenal Hyperplasia
A Ph2b to Evaluate Tildacerfont in the Reduction of Glucocorticoid Steroid Doses in Adult CAH
An investigation of the ability of Tildacerfont to reduce supraphysiologic glucocorticoid dosing in classic Congenital adrenal hyperplasia (CAH) subjects up to 76 weeks of treatment. Optional open label extension up to 240 weeks.
- Trial name
- NCT04544410
- Where
- 43 sites in United States, Australia and 14 more
- Volunteers
- 100 people
- Sponsor
- Spruce Biosciences
Overweight, Obesity
Electrical Vestibular Nerve Stimulation (VeNS) Compared to Sham Control as a Means of Reducing Excess Body Weight
A randomized, double blind sham controlled clinical trial to evaluate the efficacy of vestibular nerve stimulation (VeNS), together with a lifestyle modification program, compared to a sham control with a lifestyle modification…
- Conditions
- Overweight, Obesity, Overweight and Obesity, Overweight or Obesity
- Trial name
- NCT04110717
- Testing
- Vestal Device, Control Device, Hypocaloric Diet
- Where
- Ireland
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Neurovalens Ltd.
Obesity, Hyperglycemia
The Effect of a Bifidobacterium and Polydextrose on Body Fat Mass
Obesity is a major problem worldwide, and it is related to abnormalities in glucose and lipid metabolism. The purpose of this study is to investigate the effect of a dietary supplement containing probiotic (Bifidobacterium…
- Conditions
- Obesity, Hyperglycemia, Insulin Resistance
- Trial name
- MetSProb (NCT01978691)
- Testing
- Bifidobacterium animalis ssp. lactis 420, Polydextrose
- Where
- 4 sites in Finland
- Volunteers
- 225 people, healthy volunteers welcome
- Sponsor
- Danisco
Menopause, Climacteric Syndrome
Effects of Combined Natural Molecules on Metabolic Syndrome in Menopausal Women
The study collected retrospective data from the assumption of combined natural molecules in menopausal women with metabolic syndrome
- Conditions
- Menopause, Climacteric Syndrome, Metabolic Syndrome
- Trial name
- NCT06057896
- Testing
- inositol, phytoestrogens, cocoa polyphenols
- Where
- Italy
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Lo.Li.Pharma s.r.l
Malabsorption
Enteral Nutrition and Amino Acid Absorption
The purpose of this study is to evaluate amino acid absorption with two different type of proteins.
- Trial name
- PEPS (NCT02590120)
- Testing
- Enteral nutrition
- Where
- France
- Volunteers
- 6 people
- Sponsor
- Société des Produits Nestlé (SPN)
Prader-Willi Syndrome
A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone Injection in Patients With Prader-Willi Syndrome
To evaluate the effectiveness of rhGH (Recombinant human growth hormone) injection for improving motor development in patients with PWS.
- Trial name
- NCT03554031
- Testing
- Recombinant Human Growth Hormone (rhGH) Injection
- Where
- 6 sites in China
- Volunteers
- 30 people
- Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
Obesity, Overweight
AMAZE 3: A Research Study Investigating How Well the Medicine NNC0487-0111 Helps People With Excess Body Weight and Obstructive Sleep Apnoea Not Treated With Positive Airway Pressure Lose Weight and Improve Sleep Apnoea
This study is being done to look at the efficacy and safety of NNC0487-0111 in participants with excess body weight and obstructive sleep apnoea not treated with positive airway pressure lose weight and improve sleep apnoea.
- Conditions
- Obesity, Overweight, Obstructive Sleep Apnoea
- Trial name
- AMAZE 3 (NCT07571005)
- Testing
- NNC0487-0111
- Where
- 80 sites in United States, Australia and 8 more
- Volunteers
- 300 people
- Sponsor
- Novo Nordisk A/S
Metabolism and Nutrition Disorder, Obesity
Effect of Liraglutide in Obese Subjects With Moderate or Severe Obstructive Sleep Apnoea: SCALE™ - Sleep Apnoea
This trial is conducted in North America. The aim of the trial is to investigate the effect of liraglutide in obese subjects with sleep apnoea.
- Conditions
- Metabolism and Nutrition Disorder, Obesity, Obstructive Sleep Apnoea
- Trial name
- NCT01557166
- Testing
- liraglutide
- Where
- 42 sites in United States, Canada
- Volunteers
- 359 people
- Sponsor
- Novo Nordisk A/S
Menkes Disease
Long Term Follow-up on Menkes Disease Patients
This study will collect long term follow-up data regarding overall survival and neurological parameters from patients previously identified and/or enrolled in Protocols 09-CH-0059 and 90-CH0149 and Historical Control data on…
- Trial name
- NCT04337684
- Testing
- Long Term Follow-Up
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Sentynl Therapeutics, Inc.
Meulengracht Syndrome, Hyperbilirubinemia
Biomarker for Gilbert Disease (BioGilbert)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Gilbert disease from blood
- Conditions
- Meulengracht Syndrome, Hyperbilirubinemia, Unconjugated Benign Bilirubinemia
- Trial name
- BioGilbert (NCT02712138)
- Where
- 4 sites in Egypt, India and 1 more
- Sponsor
- CENTOGENE GmbH Rostock