Clinical trials: Lipoprotein Lipase Deficiency, open to volunteers
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
Postprandial Fatty Acid Metabolism in Subjects With Lipoprotein Lipase Deficiency
Lipoprotein lipase (LPL) is an enzyme that plays an important role in removing triglycerides (TG) (molecules that transport dietary fat) from the blood.
- Trial name
- AGL12 (NCT04227678)
- Testing
- Heparin, liquid meal
- Where
- Canada
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Université de Sherbrooke
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 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
- 6 sites in United States
- Volunteers
- 12 people
- Sponsor
- Ionis Pharmaceuticals, Inc.