Trial reportLipids in health and disease2015
Effect of the DGAT1 inhibitor pradigastat on triglyceride and apoB48 levels in patients with familial chylomicronemia syndrome.
Trial report in Lipids in health and disease, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT01146522 (A Multiple-dose, Parallel Group Study to Assess Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of LCQ908 in Patients With Severe Hypertriglyceridemia and Chylomicronemia), which is not on this map. Cited by 48 papers, 1 of them a synthesis that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Multiple-dose, Parallel Group Study to Assess Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of LCQ908 in Patients With Severe Hypertriglyceridemia and Chylomicronemia (Phenotypes I and V)
Who cites it
48 citing papers in PubMed, 1 synthesis or guideline pooled it, 108 citations in OpenAlex.
- Efficacy and Safety of Volanesorsen (ISIS 304801): the Evidence from Phase 2 and 3 Clinical Trials.Current atherosclerosis reports · 2020Pooled it
- The Crimean-Congo haemorrhagic fever virus hijacks the liver lipid metabolic pathway for virion production.Emerging microbes & infections · 2026Article
- Review
- DGAT1 Inhibition Induces Ferroptosis and Enhances Cancer Immunotherapy Efficacy.Cancer research · 2026Article
- Lipid droplet-associated proteins: Roles in cardiovascular diseases.Journal of lipid research · 2026Review
- Targeting dormant cancer cells: ferroptosis as a precision therapeutic strategy.Cellular & molecular biology letters · 2026Review
- Metformin induces ferroptosis associated with lipidomic remodeling in AML.Blood advances · 2026Article
- Lipid metabolic reprogramming in clear cell renal cell carcinoma: focus on high-density lipoprotein-related pathways.Oncology reviews · 2026Review
- Targeting Triglycerides: The Rise of Apolipoprotein C3 and Angiopoietin-Like Protein 3 Inhibitors.American journal of cardiovascular drugs : drugs, devices, and other interventions · 2025Review
- Pharmacological Treatment of MASLD: Contemporary Treatment and Future Perspectives.International journal of molecular sciences · 2025Review
- An overview of persistent chylomicronemia: much more than meets the eye.Current opinion in endocrinology, diabetes, and obesity · 2025Review
- A New Insight on Atherosclerosis Mechanism and Lipid-Lowering Drugs.Reviews in cardiovascular medicine · 2025Review
- Review
- The Essence of Lipoproteins in Cardiovascular Health and Diseases Treated by Photodynamic Therapy.Biomedicines · 2024Review
- The role of DGAT1 and DGAT2 in regulating tumor cell growth and their potential clinical implications.Journal of translational medicine · 2024Review
- Long-Term Treatment of Lipoprotein Lipase Deficiency with Medium-Chain Triglyceride-Enriched Diet: A Case Series.Nutrients · 2023Article
- Severe Hypertriglyceridaemia and Chylomicronaemia Syndrome-Causes, Clinical Presentation, and Therapeutic Options.Metabolites · 2023Review
- Current Understanding on the Role of Lipids in Macrophages and Associated Diseases.International journal of molecular sciences · 2022Review
- When fat meets the gut-focus on intestinal lipid handling in metabolic health and disease.EMBO molecular medicine · 2022Review
- Inflammation and atherosclerosis: signaling pathways and therapeutic intervention.Signal transduction and targeted therapy · 2022Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors at 3 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundFamilial chylomicronemia syndrome (FCS) is a rare lipid disease caused by complete lipoprotein lipase (LPL) deficiency resulting in fasting chylomicronemia and severe hypertriglyceridemia. Inhibition of diacylglycerol acyltransferase 1 (DGAT1), which mediates chylomicron triglyceride (TG) synthesis, is an attractive strategy to reduce TG levels in FCS. In this study we assessed the safety, tolerability and TG-lowering efficacy of the DGAT1 inhibitor pradigastat in patients with FCS.
methodsSix FCS patients were enrolled in an open-label clinical study. Following a 1-week very low fat diet run-in period patients underwent baseline lipid assessments, including a low fat meal tolerance test. Patients then underwent three consecutive 21 day treatment periods (pradigastat at 20, 40 & 10 mg, respectively). Treatment periods were separated by washout periods of ≥4 weeks. Fasting TG levels were assessed weekly through the treatment periods. Postprandial TGs, ApoB48 and lipoprotein lipid content were also monitored.
resultsFollowing once daily oral dosing, steady-state exposure was reached by Day 14. There was an approximately dose proportional increase in pradigastat exposure at studied doses. Pradigastat was associated with a 41% (20 mg) and 70% (40 mg) reduction in fasting triglyceride over 21 days of treatment. The reduction in fasting TG was almost entirely accounted for by a reduction in chylomicron TG. Pradigastat treatment also led to substantial reductions in postprandial TG as well as apo48 (both fasting and postprandial). Pradigastat was safe and well tolerated, with only mild, transient gastrointestinal adverse events.
conclusionThe novel DGAT1 inhibitor pradigastat substantially reduces plasma TG levels in FCS patients, and may be a promising new treatment for this orphan disease.
trial registrationClinicalTrials.gov identifier NCT01146522 .
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.