Clinical trials: Lipid Disorder
Obesity, Prediabetic State
The Study Aims to Investigate the Effects of Oral Inositols on Insulin-resistance in Children With Obesity.
The study aims to investigate the effects of oral inositols on insulin-resistance and metabolic variables (triglycerides, total cholesterol, HDL cholesterol) in children aged 8-12 years with obesity and insulin-resistance.
- Conditions
- Obesity, Prediabetic State, Dyslipidemias, Insulin Resistance
- Trial name
- ONIRICO (NCT05701813)
- Testing
- INOSITOLS, CELLULOSE
- Volunteers
- 56 people
- Sponsor
- Azienda Ospedaliera Universitaria Integrata Verona
Niemann-Pick Diseases
Induced Pluripotent Stem Cells for Niemann Pick Disease
Establishment of individualized human cellular disease models based on induced pluripotent stem cells that reflect the broad heterogeneous phenotypic spectrum of Niemann Pick disease
- Trial name
- IPSNPABC (NCT03883750)
- Where
- Pakistan
- Volunteers
- 40 people
- Sponsor
- CENTOGENE GmbH Rostock
Diabetes Mellitus Type 2
Internet Intervention to Improve Rural Diabetes Care
Rural Diabetes Online Care (RDOC) will develop an Internet-based intervention for rural primary care physicians, focusing on improving care for adult patients with diabetes.
- Trial name
- NCT00403091
- Testing
- Internet Intervention, Control
- Where
- 2 sites in United States
- Volunteers
- 205 people, healthy volunteers welcome
- Sponsor
- University of Alabama at Birmingham
Mucopolysaccharidoses, Progressive Pseudorheumatoid Dysplasia
Pediatric Arthropathy Beyond Inflammation: Clinical Spectrum and Diagnostic Approach at Assiut University Children Hospital
To identify the most common underlying causes of non-inflammatory arthropathy in children presenting to Assiut University Children Hospital for through clinical evaluation, laboratory testing, and imaging, in order to improve…
- Conditions
- Mucopolysaccharidoses, Progressive Pseudorheumatoid Dysplasia, Farber Disease, Osteogenesis Imperfecta
- Trial name
- NCT07173010
- Volunteers
- 35 people
- Sponsor
- Assiut University
Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis
Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD.
- Conditions
- Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies
- Trial name
- NCT02231710
- Testing
- BPX-501 and Rimiducid
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Bellicum Pharmaceuticals
Lysosomal Acid Lipase Deficiency, Wolman Disease
A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype
This is a Natural History study to characterize key aspects of the clinical course of lysosomal acid lipase (LAL) deficiency/Wolman phenotype in patients.
- Trial name
- NCT01358370
- Where
- 14 sites in United States, Canada and 3 more
- Volunteers
- 40 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Gaucher Disease, Type I, Gaucher Disease, Type III
Gaucherite - A Study to Stratify Gaucher Disease
The purpose of this research is to review data already collected and to collect new data from adults and children in England with Gaucher Disease to determine clinical factors which predict severity and response to therapy of…
- Trial name
- Gaucherite (NCT03240653)
- Testing
- Stratified response to Enzyme Therapy
- Where
- 8 sites in United Kingdom
- Volunteers
- 250 people
- Sponsor
- Cambridge University Hospitals NHS Foundation Trust
Lipid Profile, Neonates
Omega-3 on Lipid Profile and Serum Leptin Level n Neonates With Intrauterine Growth Restriction
The aim of our study will demonstrate the effect of omega 3 supplementation on serum lipid profile level and leptin level in neonates with Intrauterine Growth Restriction( IUGR)
- Conditions
- Lipid Profile, Neonates, Intrauterine Growth Restriction
- Trial name
- NCT05242107
- Testing
- Omega-3 supplement group, Full feeding without receiving any supplementation group
- Where
- Egypt
- Volunteers
- 70 people
- Sponsor
- Tanta University
Late Infantile Metachromatic Leukodystrophy
Long-term Metazym Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
This is a single center, open-label study of patients with late infantile MLD. All patients were previous treated 26 weeks in the phase I trial (EudraCT number: 2006-005341-11, NCT00418561).
- Trial name
- NCT00633139
- Testing
- Recombinant human Arylsulfatase A (rhASA)
- Where
- Denmark
- Volunteers
- 13 people
- Sponsor
- Shire
Bone Marrow Failure Syndrome, Severe Aplastic Anemia
CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation
This study's goal is to determine the frequency and severity of acute graft versus host disease, to evaluate incidence of primary and secondary graft rejection, to assess event free survival and overall survival, to determine the…
- Conditions
- Bone Marrow Failure Syndrome, Severe Aplastic Anemia, Severe Congenital Neutropenia, Amegakaryocytic Thrombocytopenia
- Trial name
- NCT01966367
- Testing
- CD34 Stem Cell Selection Therapy
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- Diane George
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.
Hypercholesterolemia
Study of Awareness and Detection of Familial Hypercholesterolemia
The CASCADE-FH Registry is a national, multi-center initiative that will track the therapy, clinical outcomes, and patient-reported outcomes over time.
- Trial name
- CASCADE-FH (NCT01960244)
- Where
- United States
- Volunteers
- 5,000 people
- Sponsor
- The Familial Hypercholesterolemia Foundation
GM1 Gangliosidosis, GM1 Gangliosidosis, Type I
Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis
PBGM01 is a gene therapy for GM1 gangliosidosis intended to deliver a functional copy of the GLB1 gene to the brain and peripheral tissues.
- Conditions
- GM1 Gangliosidosis, GM1 Gangliosidosis, Type I, GM1 Gangliosidosis, Type 2, Beta-Galactosidase-1 (GLB1) Deficiency
- Trial name
- Imagine-1 (NCT04713475)
- Testing
- PBGM01
- Where
- 7 sites in United States, Brazil and 2 more
- Volunteers
- 26 people
- Sponsor
- Gemma Biotherapeutics
Hypertriglyceridemia, Pediatric Obesity
Effect of Omega-3 Fatty Acids Supplementation on Hypertriglyceridemia in Pediatric Patients With Obesity.
The primary objective was to evaluate the safety and efficacy the 3 grams per day of omega-3 in adolescents with obesity and hypertriglyceridemia ( ≥ 150 mg/dl and ≤ 1000 mg/dl) for 12 weeks, compared with placebo.
- Trial name
- NCT03216057
- Testing
- Omega-3 fatty acid 3 grams per day
- Volunteers
- 168 people
- Sponsor
- Hospital Infantil de Mexico Federico Gomez
Gestational Diabetes, Hyperlipidemia
Lipid Metabolism in Gestational Diabetes
The purpose of this pilot study is to determine if, during pregnancy, women with GDM have an exaggerated lipid response compared to non-gestational diabetics.
- Trial name
- NCT00534105
- Testing
- Total Cholesterol, Triglyceride, HDL
- Where
- United States
- Volunteers
- 106 people
- Sponsor
- Baystate Medical Center
Cardiovascular Disease (CVD), Hypertension
Veteran Peer Coaches Optimizing and Advancing Cardiac Health
The purpose of this study is to test if having a Veteran peer health coach will improve blood pressure control among Veterans with high blood pressure and at least one other Cardiovascular disease (CVD) risk factor.
- Conditions
- Cardiovascular Disease (CVD), Hypertension, Hyperlipemia, Obesity
- Trial name
- Vet-COACH (NCT02697422)
- Testing
- Community-based peer health coach intervention
- Where
- United States
- Volunteers
- 264 people
- Sponsor
- VA Office of Research and Development
Niemann-Pick Disease, Type C
N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)
This is a multinational, multicenter, open-label, rater-blinded prospective Phase II study which will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of Niemann-Pick type C disease (NPC).
- Trial name
- NCT03759639
- Testing
- IB1001
- Where
- 9 sites in United States, Germany and 3 more
- Volunteers
- 33 people
- Sponsor
- IntraBio Inc
Abetalipoproteinemia
Vitamin Replacement in Abetalipoproteinemia
This study will determine whether short term intravenous infusion of vitamins A and E in patients with abetalipoproteinemia can reverse disease symptoms in these patients.
- Trial name
- NCT00004574
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Acid Sphingomyelinase Deficiency (ASMD)
Data Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in France
Primary Objective: To describe the lung, spleen and liver outcomes of olipudase alfa Secondary Objectives: To describe the patient's characteristics To describe conditions of olipudase alfa use To describe safety data related to…
- Trial name
- OPERA (NCT05359276)
- Testing
- Olipudase alfa
- Where
- France
- Volunteers
- 40 people
- Sponsor
- Sanofi
Fabry Disease
Enzyme Replacement Therapy in Fabry Disease
Fabry disease is an X-linked rare metabolic disease, caused by a deficient activity of the hydrolase α-Galactosidase A, and characterized by a progressive and systematic deposition of glycosphingolipids in many organs.
- Trial name
- NCT00149318
- Testing
- Biochemical analyses.
- Where
- Italy
- Volunteers
- 2 people
- Sponsor
- Mario Negri Institute for Pharmacological Research
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