Clinical trials: Hyperlipoproteinemia Type 1
Familial Chylomicronemia Syndrome (FCS)
CS-121 APOC3 Base Editing in FCS
This is an open-label, single-arm, dose-escalation Phase I clinical trial to evaluate the safety, tolerability, pharmacodynamics (PD), and pharmacokinetics (PK) of CS-121, an in vivo base editing therapy delivered by lipid…
- Trial name
- NCT07176923
- Testing
- CS-121
- Where
- China
- Volunteers
- 15 people
- Sponsor
- CorrectSequence Therapeutics Co., Ltd
Lipoprotein Lipase Deficiency, Inborn Error of Lipid Metabolism
Biomarker for Homozygous Familial Hypercholesterolemia (BioHoFH)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Homozygous familial Hypercholesterolemia from blood
- Conditions
- Lipoprotein Lipase Deficiency, Inborn Error of Lipid Metabolism, Corneal Arcus
- Trial name
- BioHoFH (NCT03198897)
- Where
- 4 sites in Germany, India and 1 more
- Sponsor
- CENTOGENE GmbH Rostock
Familial Hypercholesterolemia - Heterozygous
Study to Evaluate Efficacy and Safety of Inclisiran in Children With Heterozygous Familial Hypercholesterolemia
This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 6 to <12 years) with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein…
- Trial name
- ORION-20 (NCT06597019)
- Testing
- Inclisiran
- Where
- 66 sites in United States, Argentina and 21 more
- Volunteers
- 60 people
- Sponsor
- Novartis Pharmaceuticals
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
Elevated LDL-Cholesterol (LDL-C)
Trial to Evaluate Safety and Tolerability of ALN-PCS02 in Subjects With Elevated LDL-Cholesterol (LDL-C)
The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of ALN-PCS02 in subjects with Elevated LDL-Cholesterol (LDL-C).
- Trial name
- NCT01437059
- Testing
- ALN-PCS02, Sterile Normal Saline (0.9% NaCl)
- Where
- 2 sites in United Kingdom
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals
Lipoprotein Lipase Deficiency, Familial Hyperlipoproteinemia Type 1
Glybera Registry, Lipoprotein Lipase Deficient (LPLD) Patients
Lipoprotein lipase deficiency (LPLD) is a rare autosomal recessive disorder, characterized by loss-of function mutations in the LPL gene, leading to the inability to produce functionally active lipoprotein lipase (LPL).
- Conditions
- Lipoprotein Lipase Deficiency, Familial Hyperlipoproteinemia Type 1, Familial Hyperchylomicronemia
- Trial name
- GENIALL (NCT03293810)
- Testing
- Observational study
- Where
- Germany
- Volunteers
- 16 people
- Sponsor
- UniQure Biopharma B.V.
Familial Chylomicronemia Syndrome (FCS)
A Randomized, Double-blind, Placebo Controlled Study to Assess Efficacy, Safety and Tolerability of LCQ908 in Subjects With Familial Chylomicronemia Syndrome
The purpose of this study is to determine whether LCQ908 is effective and safe in lowering triglycerides in subjects with Familial Chylomicronemia Syndrome (FCS) (Hyperlipoproteinemia [HLP] type I).
- Trial name
- NCT01514461
- Testing
- LCQ908
- Where
- 14 sites in United States, Canada and 6 more
- Volunteers
- 45 people
- Sponsor
- Novartis Pharmaceuticals
Immunosuppression, Hyperlipidemias
Cholesterol and Fatty Acid Synthesis in Islet and Liver Transplant Patients and Effect of Dietary Intervention
The purpose is to investigate possible mechanisms of increased blood lipid levels following transplantation, and also to see if a comprehensive dietary intervention is effective in reducing blood lipid levels in post-transplant…
- Conditions
- Immunosuppression, Hyperlipidemias, Diabetes Mellitus, Type 1
- Trial name
- NCT00947635
- Testing
- Dietary intervention - supplements
- Where
- Canada
- Volunteers
- 46 people, healthy volunteers welcome
- Sponsor
- University of Alberta
LPL Deficiency
Alipogene Tiparvovec for the Treatment of LPLD Patients
The aim of the study is to provide further confirmatory evidence of clinical benefit in LPLD patients treated with alipogene tiparvovec by assessing both the "clinical response" (as defined by a range of parameters), and "the…
- Trial name
- NCT02904772
- Testing
- alipogene tiparvovec, Prednisolone, Cyclosporins
- Where
- 2 sites in United States, Canada
- Sponsor
- UniQure Biopharma B.V.
Familial Chylomicronemia Syndrome
A Phase 3 Study of ARO-APOC3 / VSA001 / SAR449124 (Plozasiran) in Chinese Adults With Familial Chylomicronemia Syndrome
This is a randomized, double-blinded, placebo controlled, two periods phase 3 clinical study. The primary objective of the study was to evaluate the efficacy and safety of Plozasiran injection in Chinese adults with familial…
- Trial name
- NCT05902598
- Testing
- Plozasiran
- Where
- China
- Volunteers
- 37 people
- Sponsor
- Visirna Therapeutics HK Limited
LDL Hyperlipoproteinemia, Low-Density-Lipoprotein-Type [LDL] Hyperlipoproteinemia
Evaluating the Impact of Omega-3 Fatty Acid Supplementation (Soloways™) on Lipid Profiles in Adults With PPARG Polymorphisms
In this study, 102 patients will be evenly randomized into two groups: one set to receive omega-3 fatty acids and the other a placebo.
- Conditions
- LDL Hyperlipoproteinemia, Low-Density-Lipoprotein-Type [LDL] Hyperlipoproteinemia, Triglyceride Storage Type I or II Disease
- Trial name
- SOLIPARG (NCT06154408)
- Testing
- omega-3 fatty acids
- Where
- Russia
- Volunteers
- 102 people
- Sponsor
- S.LAB (SOLOWAYS)
Familial Chylomicronemia Syndrome
A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS)
The purpose of this study is to evaluate the effect of olezarsen (formerly known as AKCEA-APOCIII-LRx) on the percent change in fasting triglycerides (TG) from baseline.
- Trial name
- NCT05130450
- Testing
- Olezarsen
- Where
- 27 sites in United States, Canada and 9 more
- Volunteers
- 60 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Type 1 Diabetes, Familial Hypercholesterolemia
Pediatric Population Screening for Type 1 Diabetes and Familial Hypercholesterolemia in Lower Saxony, Germany
This is a Pilot/ feasibility study for a population-based Screening in all children 2 to 6 years in Lower Saxony, Germany during the compulsory (U7 (from the age of 2) U7a, U8, U9) routine check-ups and any voluntary visits to…
- Trial name
- Fr1dolin (NCT02750527)
- Volunteers
- 13,049 people, healthy volunteers welcome
- Sponsor
- Kinderkrankenhaus auf der Bult
Antiphospholipid (aPL)-Positive, Rheumatic Arthritis
MENA/Levant Epidemiological, Registry of Immunological & Clinical Traits (MERIT)
The primary objective of this multicenter observational registry is to characterize the clinical, epidemiological, and biological profiles of individuals of Middle Eastern, North African, and Levantine ancestry, including…
- Conditions
- Antiphospholipid (aPL)-Positive, Rheumatic Arthritis, Celiac Disease, Focal Segmental Glomerulosclerosis (FSGS)
- Trial name
- MERIT (NCT07892651)
- Where
- Jordan
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- Axiom Bioregistry
Lipoprotein Lipase Deficiency
Lipoprotein Lipase Enzyme Activity Assay Validation and Clinical Assessment
This study plans to learn more about measuring Lipoprotein lipase (LPL) activity in humans. LPL is an enzyme in the breakdown of certain types of fats into smaller parts.
- Trial name
- NCT02656095
- Testing
- Heparin
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- University of Colorado, Denver
Hypertriglyceridemia, Familial Chylomicronemia Syndrome
Study of RN0361in Adult Healthy Subjects and Adult Hypertriglyceridemic Subjects
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and immunogenicity of single doses of RN0361 in Adult healthy subjects and Adult Hypertriglyceridemic Subjects.
- Trial name
- NCT06471543
- Testing
- RN0361
- Where
- 10 sites in United States, Australia
- Volunteers
- 108 people, healthy volunteers welcome
- Sponsor
- Ikaria Bioscience Pty Ltd
Familial Chylomicronemia Syndrome
Olezarsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)
The purpose of the Expanded Access Program is to provide pre-approval access of olezarsen to eligible patients with Familial Chylomicronemia Syndrome (FCS).
- Trial name
- NCT06360237
- Testing
- Olezarsen
- Where
- United States
- Sponsor
- Ionis Pharmaceuticals, Inc.
Hyperlipoproteinemia Type I
Duration of Effect of Alipogene Tiparvovec Treatment, Which Was Administered in Other Studies
LPL (Lipoprotein Lipase) is an enzyme which plays an important role in the elimination of triglycerides (fat) and the clearance of dietary fat particles known as chylomicrons (CM) in the blood.
- Trial name
- NCT01447901
- Where
- Canada
- Volunteers
- 19 people, healthy volunteers welcome
- Sponsor
- Amsterdam Molecular Therapeutics
Familial Chylomicronemia
Study of ARO-APOC3 (Plozasiran) in Adults With Familial Chylomicronemia Syndrome (FCS)
The purpose of AROAPOC3-3001 is to evaluate the efficacy and safety of ARO-APOC3 (plozasiran) in adult participants with familial chylomicronemia syndrome (FCS).
- Trial name
- PALISADE (NCT05089084)
- Testing
- Plozasiran
- Where
- 58 sites in United States, Argentina and 19 more
- Volunteers
- 75 people
- Sponsor
- Arrowhead Pharmaceuticals
Familial Chylomicronemia Syndrome, Multifactorial Chylomicronemia Syndrome
Medical Complications in Familial and Multifactorial Chylomicronaemia Syndromes
A retrospective, systematic study of reimbursed healthcare costs over a 10 year period in patients suffering from Familial Chylomicronaemia Syndromes (FCS) or Multifactorial Chylomicronaemia Syndromes (MCS) in order to establish…
- Trial name
- ESTHYM (NCT03912181)
- Where
- France
- Volunteers
- 140 people
- Sponsor
- Hospices Civils de Lyon