Clinical trials: Lipoprotein Lipase Deficiency
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
Familial Lipoprotein Lipase Deficiency
Safety and Efficacy in LPL-Deficient Subjects of AMT-011, an Adeno-Associated Viral Vector Expressing Human Lipoprotein Lipase [S447X]
LPLD is a rare autosomal recessive disorder, characterized by the presence of marked chylomicronemia and hence hypertriglyceridemia.
- Trial name
- NCT01109498
- Testing
- Alipogene Tiparvovec (AMT-011), Human LPL [S447X], Mycophenolate mofetil, cyclosporine
- Where
- Canada
- Volunteers
- 14 people
- Sponsor
- Amsterdam Molecular Therapeutics
Hypertriglyceridemia, Familial Chylomicronemia
Study of ARO-APOC3 in Healthy Volunteers, Hypertriglyceridemic Patients and Patients With Familial Chylomicronemia Syndrome (FCS)
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…
- Trial name
- NCT03783377
- Testing
- ARO-APOC3, sterile normal saline (0.9% NaCl)
- Where
- 10 sites in Australia, Canada and 1 more
- Volunteers
- 112 people, healthy volunteers welcome
- Sponsor
- Arrowhead Pharmaceuticals
Familial Chylomicronemia Syndrome, Lipoprotein Lipase Deficiency
Phase 2 Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Chylomicronemia Syndrome (FCS)
This is a single center, open-label study to evaluate the efficacy of AKCEA-ANGPTL3-LRx for reduction of triglyceride (TG) levels in participants with FCS.
- Conditions
- Familial Chylomicronemia Syndrome, Lipoprotein Lipase Deficiency, Hyperlipoproteinemia Type 1
- Trial name
- NCT03360747
- Testing
- AKCEA-ANGPTL3-LRx
- Where
- Canada
- Volunteers
- 3 people
- Sponsor
- Akcea Therapeutics
Hyperlipoproteinemia Type I, Hypertriglyceridemia
Orlistat for the Treatment of Type I Hyperlipoproteinemia
Patients with Type I Hyperlipoproteinemia (T1HLP) have a rare form of hypertriglyceridemia marked by significant chylomicronemia and recurrent episodes of acute pancreatitis.
- Trial name
- T1HLP (NCT02767531)
- Testing
- Orlistat
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- University of Texas Southwestern Medical Center
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 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.
Familial Chylomicronemia
Volanesorsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)
The purpose of this program is to provide expanded access to volanesorsen for up to 100 Patients with Familial Chylomicronemia Syndrome (FCS).
- Trial name
- NCT03544060
- Testing
- Volanesorsen
- Sponsor
- Akcea Therapeutics
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
Type 1 Hyperlipoproteinemia
Phase 2 Study of Orlistat and SLx-4090 for the Treatment of Type 1 Hyperlipoproteinemia
Funding Source - FDA OOPD This study is being done to find out whether an investigational (not approved by FDA ) drug called SLx-4090 or Orlistat (FDA approved medication for weight loss) when given alone or in combination can…
- Trial name
- NCT01675154
- Testing
- Orlistat, Slx-4090
- Where
- 2 sites in United States
- Volunteers
- 5 people
- Sponsor
- University of Texas Southwestern Medical Center
Familial Chylomicronemia Syndrome
A Study of Olezarsen for the Treatment of Familial Chylomicronemia Syndrome (FCS) in Pediatric Participants
The primary purpose of the study is to evaluate the efficacy of olezarsen administered by subcutaneous injection to pediatric participants with FCS.
- Trial name
- NCT07727538
- Testing
- Olezarsen
- Where
- 5 sites in United States
- Volunteers
- 12 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
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
Familial Chylomicronemia Syndrome, Multifactorial Chylomicronemia Syndrome
InFocus France Epidemiological Study of Health Burden in Major Hypertriglyceridemia
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…
- Trial name
- InFocus (NCT04223908)
- Testing
- observational : questionnaries for assessment of health burden
- Where
- France
- Volunteers
- 52 people
- Sponsor
- New French Society of Atherosclerosis
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.
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.
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
Familial Lipoprotein Lipase Deficiency
Efficacy and Safety of Human Lipoprotein Lipase (LPL)[S447X] Expressed by an Adeno-Associated Viral Vector in LPL-deficient Subjects
This trial is designed to expand the currently available data on the safety and efficacy of alipogene tiparvovec treatment in lipoprotein lipase deficiency (LPLD) and to further the understanding of possible mechanisms of action…
- Trial name
- NCT00891306
- Testing
- Alipogene Tiparvovec (AMT-011), Human LPL [S447X], mycophenolate mofetil, cyclosporine
- Where
- 2 sites in Canada
- Volunteers
- 5 people
- Sponsor
- Amsterdam Molecular Therapeutics
Familial Chylomicronemia Syndrome (FCS) (HLP Type I)
Extension to a Randomized, Double-blind, Placebo Controlled Study of LCQ908 in Subjects With Familial Chylomicronemia Syndrome.
This study was to determine long-term safety and tolerability, and continued efficacy in lowering triglycerides of LCQ908 in subjects with Familial Chylomicronemia Syndrome (FCS) (HLP type I).
- Trial name
- NCT01589237
- Testing
- LCQ908
- Where
- 10 sites in United States, Canada and 5 more
- Volunteers
- 38 people
- Sponsor
- Novartis Pharmaceuticals
Familial Chylomicronemia Syndrome
A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS)
The purpose of the study was to evaluate the efficacy of olezarsen as compared to placebo on the percent change in fasting triglycerides (TG) from baseline.
- Trial name
- BALANCE (NCT04568434)
- Testing
- Olezarsen
- Where
- 47 sites in United States, Canada and 9 more
- Volunteers
- 66 people
- 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