Clinical trials: Lipid Metabolism, Inborn Errors
Fabry Disease, Left Ventricular Hypertrophy
Viennese Prevalence Study of Anderson-Fabry Disease
The prevalence of Anderson - Fabry disease in patients with left ventricular hypertrophy is unclear. The investigators will examine urine - α - Galactosidase activity and globotriaosylceramide isoforms in these patients.
- Trial name
- VIEPAF (NCT00871611)
- Where
- Austria
- Volunteers
- 4,000 people
- Sponsor
- Medical University of Vienna
Hypercholesterolemia, Familial Hypercholesterolemia
A Clinical Trial to Assess the Long Term Safety and Tolerability of MK-0653H in Japanese Participants With Hypercholesterolemia (MK-0653H-833)
The study will assess the safety and tolerability of Ezetimibe 10 mg+ Rosuvastatin 2.5 mg and Ezetimibe 10 mg+ Rosuvastatin 5.0 mg for up to 52 weeks in Japanese participants with hypercholesterolemia uncontrolled with…
- Trial name
- NCT02748057
- Testing
- Ezetimibe, Rosuvastatin
- Volunteers
- 135 people
- Sponsor
- Organon and Co
Familial Hypercholesterolemia, Adherence, Medication
The Use of Mobile Health Technology and Behavioral Economics to Encourage Adherence in Adolescents
Familial hypercholesterolemia (FH) affects over one million Americans and increases the risk of cardiovascular disease (CVD) by as much as 20-fold.
- Trial name
- NCT04458766
- Testing
- Monetary incentives
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Boston Children's Hospital
Lipodystrophy, Familial Partial
Familial Partial Lipodystrophy Study
Studying patients with rare adipose tissue disorders may help the investigators to better understand the pathophysiology of diabetes and dyslipidemia in relation to adiposity, and thus have an enormous impact on public health.
- Trial name
- FPL (NCT02858830)
- Testing
- High fat mixed meal
- Where
- United States
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Mayo Clinic
Familial Hypercholesterolemia
a Prospective Pilot Study of Screening Out Rate and Clinical Management of Familial Hypercholesterolemia
1. Primary Objective To estimate the prevalence of clinical diagnosed familial hypercholesterolemia, as well as the clinical characteristics and current treatment, with applying China recent issued FH screening protocol in pilot…
- Trial name
- NCT03398954
- Where
- China
- Volunteers
- 1,128 people
- Sponsor
- China Cardiovascular Association
Cerebrotendinous Xanthomatosis
Phase II Study of Cholesterol- and Cholestanol-Free Diet, Lovastatin, and Chenodeoxycholic Acid for Cerebrotendinous Xanthomatosis
OBJECTIVES: I. Assess the biosynthesis of cholesterol and cholestanol, and measure the turnover of individual sterols and bile acids in patients with cerebrotendinous xanthomatosis before and after a cholesterol- and…
- Trial name
- NCT00004346
- Testing
- chenodeoxycholic acid, lovastatin
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- National Center for Research Resources (NCRR)
Homozygous Familial Hypercholesterolaemia (HoFH)
Efficacy and Safety of Lomitapide in Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH)
This is a single arm, open label, multi centre phase III study to evaluate the efficacy and long term safety of lomitapide in paediatric patients with HoFH receiving stable lipid lowering therapy (LLT) (including lipoprotein…
- Trial name
- NCT04681170
- Testing
- Lomitapide
- Where
- 12 sites in Germany, Israel and 4 more
- Volunteers
- 46 people
- Sponsor
- Amryt Pharma
Familial Hypercholesterolemia
A Study of the Safety and Efficacy of Patients With Familial Hypercholesterolaemia Taking Colesevelam as add-on Therapy to Their Existing Medication
This study is designed to assess whether colesevelam given as third line treatment added to a maximal tolerated and stable dose of a statin and ezetimibe is able to further decrease the level of LDL cholesterol in a safe and…
- Trial name
- NCT00655265
- Testing
- Colesevelam hydrochloride film-coated tablets
- Where
- 8 sites in France, Germany and 3 more
- Volunteers
- 86 people
- Sponsor
- Genzyme, a Sanofi Company
Hyperlipemia, Carbohydrate Inducible
Milk as a Recovery Beverage After Exercise for Improving Metabolic Health
Fasting blood fat levels (triglycerides) are often used to assess risk of heart disease but the level of fats in the blood after a meal is actually a stronger risk factor.
- Trial name
- NCT03154216
- Testing
- Exercise, Drink
- Where
- Canada
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- University of Saskatchewan
Sphingomyelin Lipidosis
A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
The primary objective of this study was to obtain data regarding the safety of olipudase alfa in participants with acid sphingomyelinase deficiency (ASMD) who were exposed to long term treatment with olipudase alfa.
- Trial name
- NCT02004704
- Testing
- GZ402665
- Where
- 9 sites in United States, Belgium and 5 more
- Volunteers
- 25 people
- Sponsor
- Genzyme, a Sanofi Company
Familial LCAT Deficiency
Intravenous ACP-501 for Familial LCAT Deficiency (rhLCAT)
Background: High density lipoprotein (HDL), or good cholesterol, moves cholesterol from the artery walls back to the liver. A blood enzyme known as LCAT maintains HDL levels and helps it remove cholesterol from the body.
- Trial name
- NCT04737720
- Testing
- Recombinant LCAT
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Smith-Lemli-Opitz Syndrome
Simvastatin Therapy in Smith-Lemli-Opitz Syndrome
This study will evaluate the safety and effectiveness of simvastatin in treating children with Smith-Lemli-Opitz syndrome (SLOS).
- Trial name
- NCT00064792
- Testing
- Simvastatin Susp., OraPlus
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- Forbes Porter, M.D.
Fabry's Disease
Analysis of the Nervous System in Patients With Fabry's Disease
Fabry's disease a genetic disorder (X-linked recessive) due to the absence of the enzyme ceramidetrihexosidase. The disease is characterized by abnormal collections of glycolipids in cells (histiocytes) within blood vessel walls…
- Trial name
- NCT00001491
- Where
- United States
- Volunteers
- 325 people, healthy volunteers welcome
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Fabry Disease
Lipidomics and Functional Analyses of Platelets in Fabry Disease
This study aims to evaluate whether platelets are biochemically and functionally altered in Fabry disease (FD) and therefore possibly implicated in FD manifestations such as cerebrovascular events.
- Trial name
- NCT02649660
- Where
- 2 sites in Switzerland
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Spital Linth
Fed
Bioequivalence Study of Lisinopril and Hydrochlorothiazide Tablets (20+25) mg Under Fed Conditions
This is a randomized, open Label, balanced, two-treatment, two-period, two-sequence, single dose, cross over pivotal study. The purpose of this study is to assess the bioequivalence between Test Product and the corresponding…
- Trial name
- NCT01827878
- Testing
- Lisinopril and Hydrochlorothiazide Tablets (20+25) mg, Zestoretic® 20/25 lisinopril/hydrochlorothiazide Tablets
- Where
- India
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- IPCA Laboratories Ltd.
Lipoprotein Lipase Deficiency, Familial, Hyperlipoproteinemia Type I
Pilot Study To Assess CAT-2003 in Patients With Chylomicronemia
The purpose of this study is to assess safety and efficacy of CAT-2003 in patients with chylomicronemia. The study will evaluate the effects of CAT-2003 on fasting total and chylomicron triglyceride levels, as well as…
- Conditions
- Lipoprotein Lipase Deficiency, Familial, Hyperlipoproteinemia Type I, Chylomicronemia, Familial, Hypertriglyceridemia
- Trial name
- NCT02098278
- Testing
- CAT-2003
- Where
- 2 sites in Canada
- Volunteers
- 13 people
- Sponsor
- Catabasis Pharmaceuticals
Niemann-Pick Type C Disease, GM1 Gangliosidosis
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of Niemann-Pick type C disease and in…
- Conditions
- Niemann-Pick Type C Disease, GM1 Gangliosidosis, GM2 Gangliosidosis
- Trial name
- NCT07054515
- Testing
- AZ-3102
- Where
- 39 sites in United States, Argentina and 15 more
- Volunteers
- 147 people
- Sponsor
- Azafaros B.V.
Parkinson's Disease, Gaucher's Disease
Intravenous N-acetylcysteine for the Treatment of Gaucher's Disease and Parkinson's Disease
The investigators are interested in determining if the investigators are able to detect changes in brain chemistry using Magnetic Resonance Spectroscopy (MRS) in individuals with Parkinson's disease (PD), those with Gaucher's…
- Trial name
- NCT01427517
- Testing
- N-acetylcysteine
- Where
- United States
- Volunteers
- 9 people, healthy volunteers welcome
- Sponsor
- University of Minnesota
Hyperhomocysteinemia, Hyperlipoproteinemias
Evaluating the Impact of B Vitamin Supplementation (Soloways™) on Homocysteine and LDL-C Levels in Patients With MTHFR, MTR, and MTRR Polymorphisms.
This randomized, double-blind, placebo-controlled trial will evaluate the impact of methylfolate, pyridoxal-5'-phosphate (P5P), and methylcobalamin supplementation on homocysteine and LDL-C levels in individuals with low to…
- Trial name
- VITAGEN-H (NCT06163443)
- Testing
- Vitamin B
- Where
- Russia
- Volunteers
- 54 people
- Sponsor
- S.LAB (SOLOWAYS)
Hypercholesterolemia
Screening for Hypercholesterolemia in Children Using Dried Blood Spot
Purpose: Heterozygous Familial Hypercholesterolemia (HeFH) is a common genetic disease responsible for premature atherosclerosis.
- Trial name
- CHOLESPOT (NCT05191355)
- Testing
- blood test
- Where
- 2 sites in France
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Hospices Civils de Lyon
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