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Hyperlipoproteinemia Type I clinical trials

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NCT ID: NCT05902598 Not yet recruiting - Clinical trials for Familial Chylomicronemia Syndrome

A Phase 3 Study of VSA001 in Chinese Adults With Familial Chylomicronemia Syndrome

Start date: July 1, 2023
Phase: Phase 3
Study type: Interventional

This is a randomized, double-blinded, placebo controlled, two periods phase 3 clinical study. The primary objective of the study is to evaluate the efficacy and safety of VSA001 injection in Chinese adults with familial chylomicronemia syndrome (FCS). A total of approximately 30 participants will be enrolled in the study.

NCT ID: NCT05687474 Recruiting - Cystic Fibrosis Clinical Trials

Baby Detect : Genomic Newborn Screening

Start date: September 1, 2022
Phase:
Study type: Observational

Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions. With a simple blood test, rare genetic conditions can be easily detected, and the early start of transformative treatment will help avoid severe disabilities and increase the quality of life. Baby Detect Project is an innovative NBS program using a panel of target sequencing that aims to identify 126 treatable severe early onset genetic diseases at birth caused by 361 genes. The list of diseases has been established in close collaboration with the Paediatricians of the University Hospital in Liege. The investigators use dedicated dried blood spots collected between the first day and 28 days of life of babies, after a consent sign by parents.

NCT ID: NCT05185843 Active, not recruiting - Clinical trials for Familial Chylomicronemia Syndrome

A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemia Syndrome (FCS) Previously Treated With Volanesorsen

Start date: February 25, 2022
Phase: Phase 3
Study type: Interventional

The purpose of the study is to evaluate the safety, tolerability, pharmacokinetic (PK) and pharmacodynamic (PD) effects of olezarsen (formerly known as AKCEA -APOCIII-LRX) in participants with FCS previously treated with volanesorsen.

NCT ID: NCT05130450 Active, not recruiting - Clinical trials for Familial Chylomicronemia Syndrome

A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS)

Start date: November 18, 2021
Phase: Phase 3
Study type: Interventional

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.

NCT ID: NCT05089084 Active, not recruiting - Clinical trials for Familial Chylomicronemia

Study of ARO-APOC3 in Adults With Familial Chylomicronemia Syndrome (FCS)

PALISADE
Start date: January 11, 2022
Phase: Phase 3
Study type: Interventional

The purpose or AROAPOC3-3001 is to evaluate the efficacy and safety of ARO-APOC3 in adult participants with familial chylomicronemia syndrome (FCS). Participants who have met all eligibility criteria will be randomized to receive 4 doses of ARO-APOC3 or matching placebo administered subcutaneously. Participants who complete the randomized period will continue in a 2-year open-label extension period where all participants will receive ARO-APOC3.

NCT ID: NCT04568434 Completed - Clinical trials for Familial Chylomicronemia Syndrome

A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS)

BALANCE
Start date: November 16, 2020
Phase: Phase 3
Study type: Interventional

The purpose of the study is to evaluate the efficacy of Olezarsen as compared to placebo on the percent change in fasting triglycerides (TG) from baseline.

NCT ID: NCT04227678 Recruiting - Clinical trials for Lipoprotein Lipase Deficiency

Postprandial Fatty Acid Metabolism in Subjects With Lipoprotein Lipase Deficiency

AGL12
Start date: December 9, 2019
Phase: N/A
Study type: Interventional

Lipoprotein lipase (LPL) is an enzyme that plays an important role in removing triglycerides (TG) (molecules that transport dietary fat) from the blood. Patients with LPL deficiency (LPLD) display during their whole life very high plasma TG levels often associated with episodes of postprandial abdominal pain, malaise, blurred vision, dizziness (hyperchylomicronemia syndrome) that may lead to recurrent pancreatitis episodes. Because of their very slow clearance in blood of their chylomicron-TG, these patients need to severely restrict their dietary fat intake to avoid these complications. Fortunately, novel treatments are being developed to circumvent LPL deficiency (LPLD) metabolic effect on chylomicron-TG clearance. However, there is no data on how LPLD affect organ-specific dietary fatty acid metabolism nor how the novel therapeutic agents may change this metabolism. For example, it is currently not understood how subjects with LPLD store their DFA into adipose tissues and whether they are able to use DFA as a fuel to sustain their cardiac metabolism, as healthy individuals do. This study aims to better understand theses two questions.

NCT ID: NCT04223908 Completed - Clinical trials for Familial Chylomicronemia Syndrome

InFocus France Epidemiological Study of Health Burden in Major Hypertriglyceridemia

InFocus
Start date: September 19, 2018
Phase:
Study type: Observational

FCS and MCS patients recruited from 7 academic reference centers were invited to answer a paper or a web questionnaire. Questions encompassed demographics, physical, cognitive and mental symptoms, health care circuit, past and current disease management, satisfaction regarding healthcare providers and impact on daily life.

NCT ID: NCT03912181 Completed - Clinical trials for Familial Chylomicronemia Syndrome

Medical Complications in Familial and Multifactorial Chylomicronaemia Syndromes

ESTHYM
Start date: March 1, 2018
Phase:
Study type: Observational

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 the relative healthcare burden of both syndromes by linking the Hospices Civils de Lyon (HCL) registry of FCS or MCS patients and data obtained from FCS or MCS patients followed in Paris, Nantes and Lyon to the French National Health System (NHS) healthcare claims database, the Système National d'Information Inter-Régimes de l'Assurance Maladie (SNIIR-AM). A probabilistic approach will be used to link databases. This linkage will be based on the following variables: age, gender, date of discharge of any hospitalization, date of any imaging procedure. This study will help to describe, in real life, the management of severe hyperglyceridaemia in France. In addition, the descriptive results will help obtain a better understanding of the patients suffering from this disease, the burden of the disease and the healthcare consumption linked to this disease. Even if this consumption of care has been relatively unexplored until this point, it is not negligible. The potential of merging genomics and claims data for cardiovascular research could help to identify ways to optimize disease

NCT ID: NCT03783377 Completed - Clinical trials for Hypertriglyceridemia

Study of ARO-APOC3 in Healthy Volunteers, Hypertriglyceridemic Patients and Patients With Familial Chylomicronemia Syndrome (FCS)

Start date: March 8, 2019
Phase: Phase 1
Study type: Interventional

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple doses of ARO-APOC3 in healthy adult volunteers and in patients with severe hypertriglyceridemia and familial chylomicronemia syndrome (FCS).