Clinical trials: Cholesterol
Smith-Lemli-Opitz Syndrome
Treatment of the Cholesterol Defect in Smith-Lemli-Opitz Syndrome
The purpose of this study is to determine whether supplementation with an oil-based cholesterol suspension will correct the biochemical abnormalities in cholesterol and its precursors in individuals with the Smith-Lemli-Opitz…
- Trial name
- NCT00272844
- Testing
- crystalline cholesterol oil-based suspension
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- Boston Children's Hospital
Hypercholesterolemia
An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia
Primary Objective: To evaluate the efficacy of alirocumab (75 or 150 milligrams [mg] depending on body weight [BW]), administered every 2 weeks (Q2W), on low-density lipoprotein cholesterol (LDL-C) levels at Week 12 of treatment…
- Trial name
- NCT03510715
- Testing
- Alirocumab SAR236553 (REGN727), Atorvastatin, Simvastatin
- Where
- 10 sites in Brazil, Canada and 8 more
- Volunteers
- 18 people
- Sponsor
- Sanofi
Lysosomal Acid Lipase Deficiency, Wolman Disease
Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
This was an open-label, repeat-dose, intra-participant dose-escalation study of SBC-102 (sebelipase alfa) in children with growth failure due to lysosomal acid lipase (LAL) Deficiency.
- Trial name
- NCT01371825
- Testing
- Sebelipase alfa (SBC-102)
- Where
- 7 sites in United States, Egypt and 3 more
- Volunteers
- 9 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Adrenoleukodystrophy, Metachromatic Leukodystrophy
HSCT for High Risk Inherited Inborn Errors
Hematopoietic stem cell transplantation has proven effective therapy for individuals with adrenoleukodystrophy (ALD), metachromatic leukodystrophy (MLD) or globoid cell leukodystrophy (GLD, or Krabbe disease).
- Conditions
- Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Tay Sachs Disease
- Trial name
- NCT00383448
- Testing
- Clofarabine, Total body Irradiation, Melphalan
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Lysosomal Acid Lipase Deficiency
Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency
This Phase 3 study evaluated the efficacy and safety of 1 milligram/kilogram (mg/kg) intravenous (IV) infusions of SBC-102 (sebelipase alfa) administered every other week (qow) in participants with late onset lysosomal acid…
- Trial name
- ARISE (NCT01757184)
- Testing
- Sebelipase Alfa
- Where
- 40 sites in United States, Argentina and 13 more
- Volunteers
- 66 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hypercholesterolemia
Effect of Ezetimibe Treatment on Low-density Lipoprotein Cholesterol (LDL-C) Levels in Participants With Coronary Heart Disease (CHD) Already Treated With a Statin (MK-0653A-205 AM1)
This non-interventional longitudinal study is a follow-up of the Austrian Cholesterol Screening and Treatment project (ACT I), which assessed the cholesterol status, including achievement of the target levels applicable at that…
- Trial name
- ACT II (NCT01381679)
- Testing
- Ezetimibe
- Volunteers
- 1,682 people
- Sponsor
- Organon and Co
Adrenoleukodystrophy, Metachromatic Leukodystrophy
Stem Cell Transplant for Inborn Errors of Metabolism
The purpose of this study is to determine the safety and engraftment of donor hematopoietic cells using this conditioning regimen in patients undergoing a hematopoietic (blood forming) cell transplant for an inherited metabolic…
- Conditions
- Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Gaucher's Disease
- Trial name
- NCT00176904
- Testing
- Stem Cell Transplant, Busulfan, Cyclophosphamide, Antithymocyte Globulin
- Where
- United States
- Volunteers
- 135 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Smith-Lemli-Opitz Syndrome
SLOS: The Effect of Simvastatin in Patients Receiving Cholesterol Supplementation
The purpose of this study is to determine if simvastatin improves development and behavior in patients with Smith Lemli-Opitz syndrome (SLOS) receiving dietary cholesterol supplementation.
- Trial name
- NCT01434745
- Testing
- Simvastatin, Lactose
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Oregon Health and Science University
HIV Infections, Hyperlipidemia
IMPAACT P1063: Safety and Effectiveness of Atorvastatin in HIV Infected Children and Adolescents With Hyperlipidemia
Treatment of HIV with combination antiretroviral regimens frequently results in the suppression of HIV viral load, significant immune recovery, and delayed disease progression.
- Trial name
- NCT00663234
- Testing
- Atorvastatin
- Where
- 11 sites in United States
- Volunteers
- 28 people
- Sponsor
- International Maternal Pediatric Adolescent AIDS Clinical Trials Group
Smith-Lemli-Opitz Syndrome
Short-term Behavioral Effects of Cholesterol Therapy in Smith-Lemli-Opitz Syndrome
This 10-week study will evaluate and compare behavior changes in children with Smith-Lemli-Opitz syndrome (SLOS) who are taking cholesterol supplementation versus those who are not on cholesterol supplementation.
- Trial name
- NCT00114634
- Testing
- Egg yolk preparation with cholesterol, Egg substitute, without cholesterol
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Niemann-Pick Disease, Type C
Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine
Background: Niemann-Pick disease type C (NPC) is a genetic disorder that results in progressive loss of nervous system function by affecting the membranes of nerve cells. There is no known cure for NPC.
- Trial name
- NCT00975689
- Testing
- N-Acetyl Cysteine
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Hypercholesterolemia
A Clinical Study in Children With Heterozygous Familial Hypercholesterolemia (HeFH) Aged 6 to 17 Treated Once Daily With Bempedoic Acid Oral Dosing (CLEAR Path 1)
Multiple-dose study to measure pharmacokinetics, pharmacodynamics and safety of bempedoic acid in pediatric participants 6 to 17 years of age with HeFH.
- Trial name
- CLEAR Path 1 (NCT05694260)
- Testing
- Bempedoic acid
- Where
- 24 sites in United States, Canada and 4 more
- Volunteers
- 31 people
- Sponsor
- Esperion Therapeutics, Inc.
15q Duplication Syndrome, CDKL5 Deficiency Disease
A Multicenter, Open-label, Pilot Study of Soticlestat (TAK-935/OV935) in Participants With 15Q Duplication Syndrome (Dup 15q) or Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder (ARCADE STUDY)
The purpose of this study is to investigate the effect of soticlestat on the frequency of motor seizures for participants with Dup15q or CDD during the Maintenance Period.
- Trial name
- NCT03694275
- Testing
- Soticlestat
- Where
- 8 sites in United States
- Volunteers
- 20 people
- Sponsor
- Takeda
Hypercholesterolemia
Efficacy and Safety of Colesevelam in Pediatric Patients With Genetic High Cholesterol
This study will evaluate the lipid-lowering effect and safety of colesevelam therapy administered to heterozygous familial pediatric patients 10 through 17 years of age who are on a stable dose of a pediatric-approved statin…
- Trial name
- NCT00145574
- Testing
- colesevelam HCl
- Where
- 25 sites in United States, Australia and 6 more
- Volunteers
- 194 people
- Sponsor
- Daiichi Sankyo
Subarachnoid Hemorrhage
Antiepileptic Drugs and Vascular Risk Markers
The purpose of this study is to determine if certain seizure medications raise levels of cholesterol and other blood components which could increase the risk of heart attacks and strokes.
- Trial name
- NCT00774306
- Testing
- phenytoin, valproate, levetiracetam
- Volunteers
- 52 people
- Sponsor
- Thomas Jefferson University
Familial Hyperlipidemia
Familial Hyperlipidemia Family Registry
If a child from the Healthy Hearts and Minds education and screening program is found to have a genetic marker for familial hyperlipidemia (FH), immediate family members are offered genetic testing at no charge to identify those…
- Trial name
- NCT05814419
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Poudre Valley Health System
Hypercholesterolemia
A Patient-Spouse Intervention for Self-Managing High Cholesterol
We examined the effect of a patient-spouse intervention to lower LDL-C by increasing patient treatment adherence. A randomized controlled trial compared a one-year, telephone-based patient-spouse intervention to usual care.
- Trial name
- CouPLES (NCT00321789)
- Testing
- spouse-assisted intervention
- Where
- United States
- Volunteers
- 255 people, healthy volunteers welcome
- Sponsor
- US Department of Veterans Affairs
Hurler's Syndrome, Maroteaux-Lamy Syndrome
Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism
The primary objective of this clinical trial is to evaluate the ability to achieve and sustain donor engraftment in patients with lysosomal and peroxisomal inborn errors of metabolism undergoing hematopoietic stem cell…
- Conditions
- Hurler's Syndrome, Maroteaux-Lamy Syndrome, Sly Syndrome, Alpha Mannosidosis
- Trial name
- NCT00668564
- Testing
- Stem Cell Transplantation, Cyclophosphamide, Campath-1H
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Heterozygous Familial Hypercholesterolemia
Trial Assessing Efficacy, Safety and Tolerability of Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) Inhibition in Paediatric Subjects With Genetic Low-Density Lipoprotein (LDL) Disorders
A study to assess safety and efficacy of evolocumab (AMG-145) in paediatric subjects aged 10-17 years diagnosed with heterozygous familial hypercholesterolemia.
- Trial name
- HAUSER-RCT (NCT02392559)
- Testing
- Evolocumab
- Where
- 66 sites in United States, Australia and 24 more
- Volunteers
- 158 people
- Sponsor
- Amgen
Type 1 Diabetes, Type 2 Diabetes
Nurse Telephone Management of Cholesterol in Diabetes
The current project is evaluating the effect of a nurse-administered phone care in diabetes to improve access to care and healthcare delivery.
- Trial name
- NATCHOS (NCT00950963)
- Testing
- Phone Counseling, Standard Clinical Care
- Where
- United States
- Volunteers
- 762 people
- Sponsor
- Denver Health and Hospital Authority