Clinical trials: Lipoprotein Lipase Deficiency
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.
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
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
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
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.
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
Hyperchylomicronemia
CS-121 APOC3 Base Editing in Children and Adolescents With Hyperchylomicronemia
This is a Prospective, Single-center, Open-label, Single-arm Clinical Study to Evaluate the Safety and Efficacy of CS-121, an In Vivo Base Editing Therapy Delivered by Lipid Nanoparticles Targeting APOC3, in Children and…
- Trial name
- NCT07371767
- Testing
- CS-121
- Where
- China
- Volunteers
- 15 people
- Sponsor
- Shanghai Jiao Tong University School of Medicine
Familial Chylomicronemia, High Risk Severe Hypertriglyceridemia (SHTG)
Treatment Protocol of Plozasiran in Adults With High-Risk Severe Hypertriglyceridemia (SHTG) and in Adults and Adolescents With FCS
This is a treatment program for the use of plozasiran in adults (AROAPOC3-EAP-002) and adolescents (AROAPOC3-EAP-003) with familial chylomicronemia syndrome (FCS) as well as in adults (AROAPOC3-EAP-004) with high risk severe…
- Trial name
- NCT06796426
- Testing
- Plozasiran
- Where
- United States
- Sponsor
- Arrowhead Pharmaceuticals