Clinical trials: Lipid
Type II Gaucher Disease
An Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher Disease
This exploratory trial is to prove the tolerability and safety of VGN-R08b to treat infants with type II Gaucher disease.
- Trial name
- NCT06272149
- Testing
- VGN-R08b
- Where
- China
- Volunteers
- 6 people
- Sponsor
- Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Resistance Training
Resistance Exercise Training in Children.
The purpose of this study is examine the effects of 8 weeks (3 days/week) of resistance exercise training in children 8 to 10 years of age on muscle strength, activation, and lipids of the vastus lateralis.
- Trial name
- NCT06078358
- Testing
- Resistance exercise training
- Where
- 4 sites in United States
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- University of Kansas Medical Center
Leukemia
Use of Lipid Emulsion or Nanoemulsion of Propofol on Children Undergoing Ambulatory Invasive Procedures.
The purpose of this study is to compare the safety and efficacy of the of propofol nanoemulsion and lipid emulsion for sedation in non-invasive ambulatory procedures.
- Trial name
- NCT01326078
- Testing
- propofol
- Sponsor
- Cristália Produtos Químicos Farmacêuticos Ltda.
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
GM1 Gangliosidosis
Interviews and Video Capture in Patients With GM1 Gangliosidosis
GM1 gangliosidosis is a rare disease for which there is a limited understanding of disease progression and meaningful outcome measures.
- Trial name
- NCT04310163
- Testing
- Natural history
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- LYSOGENE
GM2 Gangliosidosis, Niemann-Pick Type C Disease
A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease
This open-label study aims to gather long-term safety, tolerability, PK, biomarker, and clinical efficacy data relating to daily administration of Nizubaglustat in participants previously enrolled in the Phase 2 RAINBOW study…
- Trial name
- PRISMA (NCT07399704)
- Testing
- AZ-3102
- Where
- 3 sites in Brazil
- Volunteers
- 21 people
- Sponsor
- Azafaros B.V.
Shwachman Syndrome, Fanconi's Anemia
Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes
OBJECTIVES: I. Assess the efficacy of recombinant human granulocyte colony-stimulating factor (G-CSF) in raising the absolute neutrophil count, platelet count, and hemoglobin level in patients with inherited bone marrow failure…
- Conditions
- Shwachman Syndrome, Fanconi's Anemia, Dyskeratosis Congenita, Thrombocytopenia
- Trial name
- NCT00004787
- Testing
- filgrastim
- Volunteers
- 20 people
- Sponsor
- National Center for Research Resources (NCRR)
Fabry Disease
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents with Fabry Disease.
- Trial name
- FLY (NCT06328608)
- Testing
- PRX-102 1 mg/kg every two weeks
- Where
- 12 sites in United States, Austria and 4 more
- Volunteers
- 22 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Cardiovascular Diseases, Dyslipidemias
NUTS for the Prevention of Cardiovascular Disease in Chinese Adults
This study evaluates the feasibility of walnuts supplementation among population of high CVD risks over a period of 6 months. 70 participants will be controls receiving non-edible gifts.
- Trial name
- NUTS (NCT04037943)
- Testing
- Walnuts 30 grams, Walnuts 60 grams
- Where
- China
- Volunteers
- 210 people
- Sponsor
- The George Institute for Global Health, China
Gaucher Disease
Efficacy and Safety of Eliglustat in Chinese Pediatric Patients With Gaucher Disease Type 1 and Type 3
Primary Objective: Evaluate the efficacy and safety of eliglustat in Chinese pediatric patients (≥12 to <18 years old) with Gaucher disease type 1 and type 3.
- Trial name
- NCT06523517
- Testing
- Eliglustat Tartrate Capsules
- Where
- China
- Volunteers
- 5 people
- Sponsor
- Peking Union Medical College Hospital
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Severe Hypertriglyceridemia
A Phase II Study to Evaluate the Efficacy and Safety of of DR10624 in Subjects With Severe Hypertriglyceridemia
DR10624 is an Fc fusion protein tri-agonist with balanced glucagon-like peptide-1 receptor (GLP-1R)/glucagon receptor (GCGR)/ fibroblast growth factor 21 receptor (FGF21R) agonizing activities.
- Trial name
- NCT06555640
- Testing
- DR10624 Injection
- Where
- 35 sites in China
- Volunteers
- 79 people
- Sponsor
- Zhejiang Doer Biologics Co., Ltd.
Obesity, Hypercholesterolemia
Plant-Based, American Heart Assoc. or Mediterranean Diets In 9-18 yo With BMI >95%, Cholesterol >169 and Their Parents
The purpose of this research is to investigate the effects of a PB no- added-fat and the AHA and MED in obese (BMI >95%) children ages 9-18 with hypercholesterolemia (>169 mg/dl) and a parent or guardian.
- Conditions
- Obesity, Hypercholesterolemia, Cardiovascular Disease
- Trial name
- NCT02857543
- Testing
- Plant-Based Diet, American Heart Association Diet, Mediterranean Diet
- Where
- United States
- Volunteers
- 180 people
- Sponsor
- The Cleveland Clinic
Thrombocytopenia, Metachromatic Leukodystrophy
Stem Cell Transplantation (SCT) for Genetic Diseases
OBJECTIVES: I. Ascertain whether stem cell transplantation (SCT) is an effective method by which missing or dysfunctional enzymes can be replaced in patients with various inborn errors of metabolism. II.
- Conditions
- Thrombocytopenia, Metachromatic Leukodystrophy, Fanconi's Anemia, Thalassemia Major
- Trial name
- NCT00004378
- Testing
- Stem Cell Transplantation
- Where
- United States
- Sponsor
- National Center for Research Resources (NCRR)
Basal Cell Carcinoma, Keratosis Palmaris et Plantaris
The Evaluation of Oral Acitretin in the Treatment of Psoriasis, Cutaneous Disorders of Keratinization, Multiple Basal Cell Carcinomas and Other Retinoid Responsive Diseases
This is a continuing study which evaluates the long-term safety and efficacy of oral acitretin in an open manner in the treatment of psoriasis, cutaneous disorders of keratinization, multiple basal cell carcinomas and other…
- Conditions
- Basal Cell Carcinoma, Keratosis Palmaris et Plantaris, Psoriasis
- Trial name
- NCT00005660
- Where
- United States
- Volunteers
- 130 people
- Sponsor
- National Cancer Institute (NCI)
NAFLD
Effects of Tomato Products in Children With NAFLD
CONTROLLED TRIAL ON THE EFFECT OF TOMATO PRODUCTS IN OBESE CHILDREN WITH NON ALCOHOLIC FATTY LIVER DISEASE Aim of the study To evaluate the effect of the addition of a daily dose of lycopene enriched tomato sauce on the progress…
- Trial name
- NCT03463967
- Testing
- Lycopene-enriched tomato juice, Energy-restricted diet
- Where
- Italy
- Volunteers
- 61 people
- Sponsor
- Federico II University
Hypercholesterolemia, Hypertension
Effects of Rosiglitazone on Blood Vessels in Patients With High Blood Pressure and High Cholesterol
Cells in the lining of blood vessels produce various substances that cause the vessels to dilate (relax) and constrict (tighten), thereby regulating blood flow.
- Trial name
- NCT00006071
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Homozygous Familial Hypercholesterolemia
Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia
This study will evaluate the efficacy and safety of zodasiran subcutaneous (sc) injection in participants 12 to <18 years of age with genetically or clinically diagnosed homozygous familial hypercholesterolemia (HoFH) and…
- Trial name
- SPRUCE (NCT07473843)
- Testing
- Zodasiran
- Where
- 3 sites in United States, Denmark and 1 more
- Volunteers
- 10 people
- Sponsor
- Arrowhead Pharmaceuticals
Niemann-Pick Diseases, Acid Sphingomyelinase Deficiency
A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD)
US, multicenter, cohort, open label observational study with primary data collection. Ancillary protocol-specified procedures to address the study objectives (eg, assessment of ADA) may be considered outside the standard of care…
- Trial name
- NCT06192576
- Testing
- Olipudase alfa
- Where
- 6 sites in United States
- Volunteers
- 10 people
- Sponsor
- Sanofi
Fabry Disease
Podocyturia - Predictor of Renal Dysfunction in Fabry Nephropathy
In patients with Fabry disease, this research study explores the presence of podocytes in their urine as a potential non-invasive biomarker for baseline kidney disease; and explores changes in the quantity of podocytes in their…
- Trial name
- NCT02994303
- Where
- 3 sites in United States
- Volunteers
- 58 people
- Sponsor
- University of Washington
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