Clinical trials: Syndrome, Metabolic
Respiratory Failure, Neuromuscular Disease
Ventilator Monitoring in Early Exacerbation Detection
The aim of this study is to determine whether respiratory disease exacerbations (a sudden worsening of symptoms) can be predicted by variables that are monitored by non-invasive ventilators (small machines that assist breathing)…
- Conditions
- Respiratory Failure, Neuromuscular Disease, Chest Wall Disorder, Chronic Obstructive Pulmonary Disease
- Trial name
- NCT01644162
- Testing
- iVAPS ventilation
- Where
- United Kingdom
- Volunteers
- 30 people
- Sponsor
- ResMed
Diabetes
Effect of Glucose on QTc Interval in Type 1 Diabetes
High blood glucose levels (hyperglycaemia) and Moxifloxacin (a commonly used antibiotic) have both been shown independently to affect heart activity in healthy volunteers as recorded by ECG. i.e.
- Trial name
- NCT01984827
- Testing
- Moxifloxacin, hyper-glycaemic clamp
- Where
- United Kingdom
- Volunteers
- 24 people
- Sponsor
- Richmond Research Institute
Prader-Willi Syndrome
Phase 3 Study of Intranasal Carbetocin (LV-101) in Patients With Prader-Willi Syndrome
This Phase 3 study is designed to test the effectiveness of intranasal carbetocin (LV-101) in participants with Prader-Willi syndrome (PWS). Carbetocin is an oxytocin analog (a man-made chemical that is like oxytocin).
- Trial name
- CARE-PWS (NCT03649477)
- Testing
- 3.2 mg intranasal carbetocin, 9.6 mg intranasal carbetocin
- Where
- 24 sites in United States, Australia and 1 more
- Volunteers
- 130 people
- Sponsor
- Levo Therapeutics, Inc.
Lysosomal Storage Disease, Lung Diseases
Biomarker for Maroteaux-Lamy Disease (BioMaroteaux)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Maroteaux-Lamy disease from blood
- Conditions
- Lysosomal Storage Disease, Lung Diseases, Obstructive Sleep Apnoea, Macroglossia
- Trial name
- BioMaroteaux (NCT01458613)
- Where
- 5 sites in Egypt, Germany and 2 more
- Sponsor
- CENTOGENE GmbH Rostock
Type 2 Diabetes Mellitus, Metabolic Syndrome
Study to Evaluate Safety, Efficacy of PBI-4050 & Its Effect on Relevant Biomarkers in T2DM Patients With Metabolic Syndrome
This is a Phase 2, multi-center, double-blind, placebo-controlled study of the safety and effect of PBI-4050 at doses ranging from 400 mg to 1200 mg on relevant biomarkers in subjects with inadequately-controlled T2DMS on stable…
- Trial name
- NCT03081598
- Testing
- PBI-4050
- Where
- 11 sites in Canada
- Volunteers
- 104 people
- Sponsor
- Liminal BioSciences Ltd.
Frail Elderly Syndrome
A Machine Learning Algorithm to Predict Health Clinical Situations in Primary Healthcare for Frail Older Adults.
Introduction: We developed a machine learning algorithm to predict the risk of emergency hospitalization within the new 7 to 14 days with a good predictive performance (AUC=0.85).
- Trial name
- NCT06013709
- Testing
- PRESAE CARE
- Volunteers
- 1,478 people
- Sponsor
- Presage
Mitochondrial Diseases
EPI-743 for Mitochondrial Respiratory Chain Diseases
This study will evaluate the safety and efficacy of EPI-743 in participants with severe mitochondrial respiratory chain diseases who are considered to be within 90 days of end-of-life care.
- Trial name
- NCT01370447
- Testing
- EPI-743
- Where
- 16 sites in United States
- Sponsor
- PTC Therapeutics
Mucopolysaccharidosis II
A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome
This is a multicenter, multiregional, open-label study to assess the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant enzyme…
- Trial name
- NCT04251026
- Testing
- tividenofusp alfa
- Where
- 7 sites in United States, Canada and 2 more
- Volunteers
- 47 people
- Sponsor
- Denali Therapeutics Inc.
Hyperlipidemia, Low-density-lipoprotein-type
Combined Effects of Bioactive Compounds in Lipid Profile
The aim of this study is to demonstrate whether, along with dietary recommendations, Armolipid Plus ® can improve the profile of patients with elevated plasma LDL-C acting as a change of lifestyle therapy (TLC) according to the…
- Conditions
- Hyperlipidemia, Low-density-lipoprotein-type, Elevated Triglycerides
- Trial name
- ARM-PLUS-LDL (NCT01562080)
- Testing
- Armolipid Plus
- Where
- Spain
- Volunteers
- 118 people
- Sponsor
- Rottapharm Spain
Gastric Outlet Obstruction, Small Bowel Obstruction
GT Metabolic Magnet System in Adults With Gastrointestinal Disorders
Evaluate the performance and safety of the GT Metabolic Magnet System for the creation of side-to-side compression anastomosis in the stomach and/or small bowel in conditions requiring an anastomosis as part of the underlying…
- Conditions
- Gastric Outlet Obstruction, Small Bowel Obstruction, Superior Mesenteric Artery Syndrome
- Trial name
- NCT07317076
- Testing
- Magnet System
- Where
- Canada
- Volunteers
- 35 people
- Sponsor
- GT Metabolic Solutions, Inc.
Obesity Hypoventilation Syndrome
AVAPS-AE Efficacy Study
The purpose of this study is to evaluate the feasibility of using of the Average Volume Assured Pressure Support (AVAPS-AE) mode versus Continuous Positive Airway Pressure (CPAP) and bilevel pressure support ventilation (PSV)…
- Trial name
- NCT01368614
- Testing
- AVAPS-AE Mode of Therapy, Respironics OnmiLab BiPAP S mode, Respironics OmniLab Advanced CPAP mode
- Where
- 2 sites in United States
- Volunteers
- 90 people
- Sponsor
- Philips Respironics
Mucopolysaccharidosis I
Long Term Follow-Up for RGX-111
RGX-111-5101 is a long-term follow up study that evaluates the long-term safety and efficacy of RGX-111 in participants who have received RGX-111 (a gene therapy intended to deliver a functional copy of the alpha-L-iduronidase…
- Trial name
- NCT06103487
- Testing
- No Intervention
- Where
- 2 sites in United States, Brazil
- Volunteers
- 21 people
- Sponsor
- REGENXBIO Inc.
Hereditary Periodic Fever Syndromes, Cryopyrin-associated Periodic Syndromes (CAPS)
Ilaris NIS in Korea
This is a study to evaluate safety and effectiveness of Ilaris in adult and pediatric patients receiving the drug in a clinical setting for any of the following indications, Hereditary Periodic Fever Syndromes…
- Conditions
- Hereditary Periodic Fever Syndromes, Cryopyrin-associated Periodic Syndromes (CAPS), Colchicine Resistance Familial Mediterranean Fever (crFMF), TNF Receptor Associated Periodic Syndrome (TRAPS)
- Trial name
- REASSURE (NCT06838143)
- Testing
- Ilaris
- Where
- South Korea
- Volunteers
- 25 people
- Sponsor
- Novartis Pharmaceuticals
Celiac Disease
Safety and Systemic Exposure Study of BL-7010 in Well-Controlled Celiac Patients.
The purpose of this study is to evaluate the safety of different amounts of BL-7010 in single oral administration and in repeated oral administration to well-controlled celiac patients.
- Trial name
- NCT01990885
- Testing
- BL-7010
- Where
- 2 sites in Finland
- Volunteers
- 40 people
- Sponsor
- BioLineRx, Ltd.
Familial Chylomicronemia Syndrome (FCS)
A Randomized, Double-blind, Placebo Controlled Study to Assess Efficacy, Safety and Tolerability of LCQ908 in Subjects With Familial Chylomicronemia Syndrome
The purpose of this study is to determine whether LCQ908 is effective and safe in lowering triglycerides in subjects with Familial Chylomicronemia Syndrome (FCS) (Hyperlipoproteinemia [HLP] type I).
- Trial name
- NCT01514461
- Testing
- LCQ908
- Where
- 14 sites in United States, Canada and 6 more
- Volunteers
- 45 people
- Sponsor
- Novartis Pharmaceuticals
Sepsis
A Study To Compare The Effects Of The Infusion Of A Sepsis Drug Between Healthy Adults And Adults With Damaged Livers
This compound, GR270773, is a lipid based compound being developed to treat Sepsis. Lipids are broken down in the body by the liver.
- Trial name
- NCT00158769
- Testing
- Intravenous GR270773 - Phospholipid emulsion
- Where
- Slovakia
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Cockayne Syndrome
Pharmacokinetics and Safety Study of Single and Multiple Oral Doses Prodarsan™ in Patients With Cockayne Syndrome
This study is to compare the exposure of orally administered Prodarsan to the intravenous dosed Osmitrol (10% solution) in Cockayne Syndrome (CS) patients.
- Trial name
- NCT01142154
- Testing
- Prodarsan
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- DNage B.V.
Body Weight Changes, Metabolic Syndrome
Evaluation the Effect of AKK Formula on Intestinal Microbiota Regulation and Body Composition
The AKK formula, a prebiotic blend, can effectively promote the proliferation of A. muciniphila. In this study, we attempt to explore the clinical efficacy of AKK formula for A. muciniphila proliferation and weight management.
- Trial name
- NCT06132269
- Testing
- AKK formula prebiotic sachet
- Where
- Taiwan
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- TCI Co., Ltd.
Congenital Adrenal Hyperplasia
Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Crinecerfont in Participants With Classic Congenital Adrenal Hyperplasia (CAH) Who Are Less Than 4 Years Old
The main objective of this study is to assess the safety and tolerability of crinecerfont in pediatric participants 3 months to <4 years of age with CAH.
- Trial name
- NCT07536269
- Testing
- Crinecerfont
- Where
- 11 sites in United States
- Volunteers
- 20 people
- Sponsor
- Neurocrine Switzerland GmbH
Congenital Adrenal Hyperplasia, CAH - Congenital Adrenal Hyperplasia
Study to Evaluate the Safety and Efficacy of SPR001 in Subjects With Classic Congenital Adrenal Hyperplasia
This is a Phase 2 study of SPR001 for the treatment of classic CAH that will provide 12 weeks of open-label treatment to eligible subjects.
- Conditions
- Congenital Adrenal Hyperplasia, CAH - Congenital Adrenal Hyperplasia, CAH - 21-Hydroxylase Deficiency
- Trial name
- NCT03687242
- Testing
- SPR001
- Where
- 8 sites in United States
- Volunteers
- 11 people
- Sponsor
- Spruce Biosciences