Clinical trials: Syndrome, Metabolic
Mucopolysaccharidosis II, Hunter Syndrome
A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS Ⅱ
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of intracerebroventricular GC1123 in patients with MPS Ⅱ who have central nervous system involvement and are receiving…
- Trial name
- NCT05422482
- Testing
- GC1123
- Where
- 3 sites in South Korea
- Volunteers
- 12 people
- Sponsor
- GC Biopharma Corp
Cytomegalovirus Retinitis, HIV Infections
Phase III Ganciclovir +/- rGM-CSF for AIDS-Related CMV Retinitis
To determine whether co-administration of sargramostim (granulocyte-macrophage colony-stimulating factor; GM-CSF) improves tolerance to ganciclovir in patients previously intolerant because of neutropenia defined as an absolute…
- Trial name
- NCT00002070
- Testing
- Sargramostim, Ganciclovir
- Where
- 14 sites in United States
- Sponsor
- Schering-Plough
RDS of Prematurity, Surfactant Protein B Deficiency
Administration of Surfactant Through an Instillation Device Infasurf® (Calfactant) in Neonates- A Pilot Study
A pilot study to explore and determine feasibility and safety of the administration of surfactant through a new supraglottic device in patients from 500 to 5000 grams with RDS.
- Conditions
- RDS of Prematurity, Surfactant Protein B Deficiency, Chronic Lung Disease
- Trial name
- NCT04056741
- Testing
- surfactant airway device
- Where
- 4 sites in United States
- Volunteers
- 55 people
- Sponsor
- ONY
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.
DiGeorge Anomaly, Complete DiGeorge Anomaly
Phase I/II Thymus Transplantation With Immunosuppression #950
The study purpose is to determine if cultured thymus tissue implantation (CTTI) (previously described as transplantation) with tailored immunosuppression based on the recipient's pre-implantation T cell population is a safe and…
- Conditions
- DiGeorge Anomaly, Complete DiGeorge Anomaly, Complete Atypical DiGeorge Anomaly, Complete DiGeorge Syndrome
- Trial name
- #950 (NCT00579527)
- Testing
- Cultured Thymus Tissue for Implantation (CTTI), Cultured Thymus Tissue Implantation and Parental Parathyroid Transplantation, Blood Draw
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Sumitomo Pharma Switzerland GmbH
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
Dyslipidemia, Atherosclerosis
Safety, Pharmacokinetic Study of RVX000222 in Healthy Subjects and Subjects With Low HDL Cholesterol
The purpose of this study is to investigate an oral formulation of RVX000222 for safety, pharmacokinetic and efficacy in healthy subjects.
- Conditions
- Dyslipidemia, Atherosclerosis, Acute Coronary Syndrome, Cardiovascular Disease
- Trial name
- NCT00768274
- Testing
- RVX000222
- Where
- United States
- Volunteers
- 72 people, healthy volunteers welcome
- Sponsor
- Resverlogix Corp
Respiratory Insufficiency, Obesity Hypoventilation Syndrome
Juno Perth Clinical Trial
This study is to evaluate the efficacy of a new therapy (Automatic Expiratory Positive Airway Pressure with intelligent Volume Assured Pressure Support (AutoEPAP iVAPS)) designed to treat respiratory insufficiency, respiratory…
- Conditions
- Respiratory Insufficiency, Obesity Hypoventilation Syndrome, Chronic Obstructive Pulmonary Disease (COPD), Neuromuscular Disease
- Trial name
- NCT02317042
- Testing
- Juno
- Where
- 2 sites in Australia
- Volunteers
- 25 people
- Sponsor
- ResMed
Celiac Disease
A Study to Evaluate Gluten Challenge on Immune Responses in Subjects With Celiac Disease
This is a model development, open label, no therapeutic treatment, three sequential group, short term-gluten challenge study in subjects with celiac disease. Immune responses are evaluated following gluten challenge.
- Trial name
- NCT03521180
- Where
- 2 sites in United States
- Volunteers
- 5 people
- Sponsor
- Celgene
Menopause
The Effects of a Novel Probiotic Supplement on Menopausal Symptoms and Bone Health
This Phase II study is designed to evaluate the potential effects of a novel probiotic supplement on the severity of global menopause symptoms (e.g., psychological, somatic, urogenital symptoms) and on bone health in…
- Trial name
- MPS (NCT04001088)
- Testing
- Probiotic
- Where
- 5 sites in United States
- Volunteers
- 144 people, healthy volunteers welcome
- Sponsor
- Lallemand Health Solutions
Mild Cognitive Impairment
A Retrospective Analysis of Cerefolin NAC® in Cognitively Impaired Patients
This retrospective analysis of de-identified data from 700 charts of consecutive patients from this patient population is to investigate the prevalence of hyperhomocysteinemia and associated metabolic abnormalities in this…
- Trial name
- NCT01594541
- Where
- United States
- Volunteers
- 700 people
- Sponsor
- Pamlab, Inc.
Lesch-Nyhan Disease, Self-injurious Behavior
Ecopipam Treatment of Self-Injurious Behavior in Subjects With Lesch-Nyhan Disease
The purpose of this research study is to gather scientific information about the effectiveness and safety of the study drug, Ecopipam (PSYRX 101), for the treatment of self-injurious behaviors when compared with the effectiveness…
- Trial name
- NCT01751802
- Testing
- Ecopipam
- Where
- 2 sites in United States, Spain
- Volunteers
- 9 people
- Sponsor
- Emalex Biosciences Inc.
Hyponatremia, Syndrome of Inappropriate ADH (SIADH) Secretion
An Observational Study to Describe the Adherence to the SEOM Algorithm for the Treatment of Hyponatraemia in Spain
In 2014, the Spanish Sociedad Española de Oncología Médica (SEOM) algorithm for the treatment of hyponatraemia secondary to to syndrome of inappropriate antidiuretic hormone (SIADH) in oncology patients was developed.
- Trial name
- ALGA (NCT02548845)
- Where
- 4 sites in Spain
- Volunteers
- 70 people
- Sponsor
- Otsuka Pharmaceutical Europe Ltd
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
Coeliac Disease, Celiac Disease
A Phase 2a/b Study of the Efficacy and Safety of Subcutaneous Amlitelimab in Adults With Nonresponsive Celiac Disease
This is a Phase 2a/b, randomized, double-blind, placebo-controlled, parallel-group, 6-arm study to evaluate the efficacy and safety of amlitelimab in adult participants with non-responsive celiac disease (NRCD) who are on a…
- Trial name
- NCT06557772
- Testing
- Amlitelimab, SIGE, Gluten-free product (GFP)
- Where
- 118 sites in United States, Argentina and 19 more
- Volunteers
- 229 people
- Sponsor
- Sanofi
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
Hypertension, Metabolic Syndrome
Effect of Valsartan on Lipid Subfractions in Hypertensive Patients With Metabolic Syndrome
The purpose of this study is to evaluate potential effects of valsartan on lipid subfractions and other parameters in lipid metabolism in patients with essential hypertension and metabolic syndrome.
- Trial name
- NCT00394745
- Testing
- Valsartan
- Where
- 2 sites in Germany, Switzerland
- Volunteers
- 45 people
- Sponsor
- Novartis
Lactose Intolerance
A Non-inferiority, Multicenter and Randomized, Multiple-Dose Study About a Treatment to Hypolactasia
The primary objective is to determine the compared clinical efficacy of Lactase Eurofarma (test drug), showing non-inferiority to Lactaid® (comparative drug) in the supportive treatment of lactose intolerance and to assess the…
- Trial name
- LAILAII (NCT01145586)
- Testing
- Lactase Oral Tablets
- Where
- 8 sites in Brazil
- Volunteers
- 140 people
- Sponsor
- Eurofarma Laboratorios S.A.
MELAS, LHON
A Dose-escalating Clinical Trial With KH176
Mitochondrial Diseases are rare progressive, multi-system, often early fatal disorders affecting both children and adults. KH176 is a novel chemical entity currently under development for the treatment of inherited mitochondrial…
- Conditions
- MELAS, LHON, Leigh Syndrome, Mitochondrial Disease
- Trial name
- NCT02544217
- Testing
- KH176
- Where
- Belgium
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Khondrion BV
Celiac Disease, Intestinal Disease
Bioequivalence Study of Nexvax2 in Subjects With Celiac Disease
A randomized, double-blind, placebo-controlled clinical study in non-homozygous human leukocyte antigen (HLA)-DQ.2.5+ adults with celiac disease (CeD).
- Conditions
- Celiac Disease, Intestinal Disease, Malabsorption Syndromes, Gastrointestinal Disease
- Trial name
- NCT03543540
- Testing
- Nexvax2
- Where
- 3 sites in Australia
- Volunteers
- 14 people
- Sponsor
- ImmusanT, Inc.