Clinical trials: Lipid Disorder
Hypobetalipoproteinemias
Study of the Absorption of Vitamin E Water-soluble Form (Pegylated) in the Familial Hypocholesterolemia With Chylomicron Retention
To evaluate the kinetics of intestinal absorption of vitamin E water-soluble form from the classical lipid-soluble form in a population of patients with intestinal malabsorption (hypocholesterolemias family by retention of…
- Trial name
- VEDROP (NCT01457690)
- Testing
- Tocofersolan, tocopherol alpha, Tocofersolan and tocopherol alpha
- Where
- France
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- Hospices Civils de Lyon
Familial Hypercholesterolaemia
Computerized Decision Support for Identification and Management of Familial Hypercholesterolemia
The goal of this clinical trial is to learn if a computer alert can aid clinicians in identifying patients with a genetic type of high cholesterol, called Familial Hypercholesterolemia.
- Trial name
- FH-ALERT (NCT06743659)
- Testing
- Alert-based computerized decision support
- Where
- United States
- Volunteers
- 450 people
- Sponsor
- Brigham and Women's Hospital
Type 2 Gaucher Disease
Convection-Enhanced Delivery of Glucocerebrosidase to Treat Type 2 Gaucher Disease
This study will use an experimental method of delivering the enzyme glucocerebrosidase directly into the brain of a patient with Gaucher disease to treat disease symptoms.
- Trial name
- NCT00244582
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Gaucher Disease, Gaucher Disease Type 1
Study of Velaglucerase Beta ERT in Children With Gaucher Disease
This is a Phase 3, multicenter, open-label, single-arm study to evaluate the safety, efficacy, PK, PD, and immunogenicity of velaglucerase beta in untreated children with GD1 or GD3 for 39 weeks.
- Conditions
- Gaucher Disease, Gaucher Disease Type 1, Gaucher Disease Type 3
- Trial name
- NCT07847411
- Testing
- Velaglucerase beta
- Where
- China
- Volunteers
- 10 people
- Sponsor
- CANbridge Life Sciences Ltd.
Late Infantile Neuronal Ceroid Lipofuscinosis, Batten Disease
Collection of Cerebrospinal Fluid in Healthy Children
The purpose of this study is to obtain cerebrospinal fluid from healthy children already undergoing a lumbar puncture procedure at New York Presbyterian Hospital, to act as healthy controls when compared to children with late…
- Trial name
- NCT01698229
- Testing
- Group 1
- Where
- United States
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Weill Medical College of Cornell University
Neuronal Ceroid Lipofuscinosis
Italian NCL Registry: a Registry for NCL as an Integration Tool for Future Therapeutic Strategies
The goal is to create a solid and harmonious disease registry of patient affected by neuronal ceroid lipofuscinosis (NCLs) that facilitates the collection and management of patients' data over time encouraging the research and…
- Trial name
- NCT06844877
- Where
- Italy
- Volunteers
- 50 people
- Sponsor
- IRCCS Fondazione Stella Maris
Lipomatosis, Multiple Symmetrical
Evaluation of Safety and Efficiency of Metreleptin Treatment for Patients With Multiple Symmetric Lipomatosis (MSL)
Participants (homozygous MFN2 [gene that provides instructions to produce the Mitofusin 2 protein] R707W) will be treated with Metreleptin, and effects on body composition, metabolic parameters and safety will be assessed over a…
- Trial name
- NCT05351164
- Testing
- Metreleptin
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- University of Michigan
Hyperlipidemia, Familial Combined, Coronary Disease
Understanding the Genetic Basis of Familial Combined Hyperlipidemia in Mexican Individuals
Familial combined hyperlipidemia (FCHL) is an inherited disorder characterized by elevated levels of cholesterol and triglycerides; it often occurs in Mexican individuals with coronary heart disease (CHD).
- Trial name
- NCT00365235
- Where
- Mexico
- Volunteers
- 998 people, healthy volunteers welcome
- Sponsor
- University of California, Los Angeles
Inherited Metabolic Disorders (IMD)
MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)
This study is designed to assess the safety and efficacy of using MGTA-456 in patients with Inherited Metabolic Disorders (IMD) undergoing stem cell transplantation.
- Trial name
- NCT03406962
- Testing
- MGTA-456
- Where
- 2 sites in United States
- Volunteers
- 8 people
- Sponsor
- Magenta Therapeutics, Inc.
Cerebrotendinous Xanthomatosis (CTX)
Cerebrotendinous Xanthomatosis (CTX) Prevalence Study
Iiopathic bilateral cataracts occur in many children with Cerebrotendinous Xanthomatosis (CTX) so the opportunity to use contact with health care providers in connection with pediatric cataract evaluation and treatment is a…
- Trial name
- NCT02638220
- Where
- 44 sites in United States
- Volunteers
- 442 people
- Sponsor
- Travere Therapeutics, Inc.
Cardiovascular Outcome, Kalemia Control
Fiber Intake in an End Stage Renal Disease (ESRD) Population Followed Over 2 Years
Our research involves the detailed description of the content of the diet of x dialysis patients. For our purpose we used a newly developed "food frequency questionnaire".
- Conditions
- Cardiovascular Outcome, Kalemia Control, Lipid Profile
- Trial name
- NCT00874380
- Testing
- Increasing total fiber intake in patient's diet
- Where
- United States
- Volunteers
- 70 people
- Sponsor
- Northwell Health
Non-Alcoholic Fatty Liver Disease
Gemcabene for the Treatment of Pediatric NAFLD
This is a multicenter, prospective, open-label, Phase 2, proof of concept study to test preliminary efficacy and safety of gemcabene in children with established nonalcoholic fatty liver disease (NAFLD) incompletely treated by…
- Trial name
- NCT03436420
- Testing
- Gemcabene
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Emory University
Fabry Disease
Treatment Protocol of Replagal for Patients With Fabry Disease
The study will evaluate the safety and efficacy of Replagal® (agalsidase alfa) at a dose of 0.2 mg/kg infused intravenously (IV) over 40 minutes, every other week.
- Trial name
- NCT01031173
- Testing
- agalsidase alfa
- Where
- 25 sites in United States
- Sponsor
- Shire
Bone Marrow Failure Syndrome, Congenital Amegakaryocytic Thrombocytopenia
Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)
This phase II trial tests whether treosulfan, fludarabine, and rabbit antithymocyte globulin (rATG) work when given before a blood or bone marrow transplant (conditioning regimen) to cause fewer complications for patients with…
- Conditions
- Bone Marrow Failure Syndrome, Congenital Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Hereditary Sideroblastic Anemia
- Trial name
- NCT04965597
- Testing
- Treosulfan, Fludarabine Phosphate, Tacrolimus
- Where
- 24 sites in United States
- Volunteers
- 40 people
- Sponsor
- Fred Hutchinson Cancer Center
Diabetes, Hypertension
Pharmacy Interventions to Improve Chronic Disease Medication Refill
Compare the effectiveness of 3 strategies by pharmacists to decrease the time to refill of prescriptions for common chronic diseases (diabetes, hypertension, hyperlipidemia, heart failure, depression, psychoses).
- Conditions
- Diabetes, Hypertension, Hyperlipidemia, Heart Diseases
- Trial name
- NCT00469365
- Testing
- pharmacist contact with the patient via telephone, Pharmacist contact with the patient's physician via fax, Usual care
- Where
- United States
- Volunteers
- 3,048 people
- Sponsor
- Medical University of South Carolina
Blood Clots, Infection and Inflammatory Reaction
Cobas® Lumira Collection of Venous and Capillary Blood Samples for the Research, Optimization and Calibration of New Diagnostic Devices
This study is a blood sample collection study, collecting venous and capillary blood samples from adult patients in the UK, with a range of health conditions.
- Conditions
- Blood Clots, Infection and Inflammatory Reaction, Heart Disease, Kidney Disease
- Trial name
- EVOLVE (NCT07378514)
- Testing
- Blood samples are obtained and pseudonymized, prior to use for the development, optimization and calibration of diagnostic tests
- Volunteers
- 30,000 people
- Sponsor
- LumiraDx UK Limited
Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML)
CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant
The purpose of this study is to learn more about the effects of (classification determinant) CD34+ stem cell selection on graft versus host disease (GVHD) in children, adolescents, and young adults.
- Conditions
- Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome (MDS), Juvenile Myelomonocytic Leukemia (JMML)
- Trial name
- NCT02061800
- Testing
- CliniMACS CD34+ Reagent System, Thiotepa, Cyclophosphamide
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Diane George
Fabry Disease, Healthy
PET Scans in Normal Volunteers and Patients With Fabry Disease
This study will determine the rate of sugar metabolism in the brain of patients with Fabry disease, a genetic disease of abnormal lipid metabolism.
- Trial name
- NCT00005111
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Childhood Obesity, Dyslipidemia
Nutritional Treatment of Overweight Adolescents With Cardiovascular Risk Factors (PowerUp)
This study is designed to evaluate the efficacy of two diets, a low glycemic load diet and a low saturated fat diet, in the treatment of adolescents with some heart disease risk factors associated with being overweight, such as…
- Conditions
- Childhood Obesity, Dyslipidemia, Insulin Resistance, Hypertension
- Trial name
- NCT00477477
- Testing
- Diet
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- Boston Children's Hospital
Neurological Regression, Myoclonus
Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders
Study description: This is a natural history study that will evaluate any patient with enzyme or DNA confirmed GM1 or GM2 gangliosidosis, sialidosis or galactosialidosis.
- Conditions
- Neurological Regression, Myoclonus, Cherry Red Spot, Brain Atrophy
- Trial name
- NCT00029965
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
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